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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-002619-24 | EudraCT Number |
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This study will evaluate whether IV sildenafil can reduce the time on inhaled nitric oxide treatment and reduce the failure rate of available treatments for persistent pulmonary hypertension of the newborn.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| placebo | Placebo Comparator | iv placebo of normal saline or 10% dextrose |
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| sildenafil | Experimental | Active study drug |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| placebo | Drug | IV placebo or 0.9% sodium chloride or 10% dextrose. Infusion rate based on weight. |
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| Measure | Description | Time Frame |
|---|---|---|
| Time on Inhaled Nitric Oxide (iNO) Treatment After Initiation of Intravenous (IV) Study Drug For Participants Without Treatment Failure | Time in days, on iNO treatment, for participants without iNO treatment failure, was calculated 14 days from the initiation of IV study drug or hospital discharge, whichever occurred first. iNO treatment failure was defined as need for additional treatment targeting PPHN, need for extra corporeal membrane oxygenation (ECMO), or death during the study. | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
| Treatment Failure Rate | Treatment failure rate was defined as percentage of participants who needed additional treatment targeting PPHN, needed ECMO, or died during the study. | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
| Measure | Description | Time Frame |
|---|---|---|
| Time From Initiation of Intravenous (IV) Study Drug to Final Weaning of Mechanical Ventilation | Time in days, from initiation of IV study drug to final weaning of mechanical ventilation among participants achieving final weaning of mechanical ventilation for PPHN was evaluated. Kaplan-Meier method was used for estimation. For participants with mechanical ventilation beyond 336 hours (14 days) from initiation of IV study drug, data was censored at 14 days. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| University of California Davis Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34052232 | Derived | Pierce CM, Zhang MH, Jonsson B, Iorga D, Cheruvu N, Balagtas CC, Steinhorn RH. Efficacy and Safety of IV Sildenafil in the Treatment of Newborn Infants with, or at Risk of, Persistent Pulmonary Hypertension of the Newborn (PPHN): A Multicenter, Randomized, Placebo-Controlled Trial. J Pediatr. 2021 Oct;237:154-161.e3. doi: 10.1016/j.jpeds.2021.05.051. Epub 2021 May 27. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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Neonates with persistent pulmonary hypertension of the newborn (PPHN) or hypoxic respiratory failure (HRF) and at risk of PPHN who were receiving inhaled nitric oxide (iNO) treatment were evaluated in this study.
This study was planned in two parts Part A (double-blind phase) and Part B (long-term, non-intervention phase).
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| ID | Title | Description |
|---|---|---|
| FG000 | IV Sildenafil | Part A: Participants received sildenafil intravenously based on their body weight at a loading dose of 0.1 milligrams per kg (mg/kg) for 30 minutes on Day 1 followed by a maintenance dose of 0.03 milligrams per kg per hour (mg/kg/hr), for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 16, 2015 | Oct 16, 2019 |
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| iv sildenafil | Drug | loading dose of 0.1 mg/kg over 30 minutes followed by maintenance dose of 0.03 mg/kg/h. To infuse minimum 48 hours and maximum of 14 days. |
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| 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
| Time From Initiation of Intravenous (IV) Study Drug to First Treatment Failure | Time in days, from initiation of IV study drug to first treatment failure (defined as need for additional treatment targeting PPHN, need for ECMO, or death) for participants with treatment failure was evaluated. Kaplan-Meier method was used for estimation. For participants without treatment failure by the endpoint assessment date, data was censored at the endpoint assessment date. | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
| Percentage of Participants With Individual Components of Treatment Failure | Percentage of participants with individual components of treatment failure (need to start additional treatment targeting PPHN, need to start ECMO, or death) were evaluated. Some participants could have had multiple qualifying events for treatment failure. | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
| Change From Baseline in Oxygenation Index (OI) at Hours 6, 12 and 24 Post-Infusion | Oxygenation index was calculated as the product of fraction of inspired oxygen (FiO2) and mean airway pressure divided by partial pressure of oxygen dissolved in arterial blood (PaO2) [(FiO2*mean airway pressure)/PaO2] measured in centimeter of water per millimeter of mercury (cmH2O/mmHg). FiO2 is the measure of oxygen concentration that is breathed. Mean airway pressure is defined as an average of the airway pressure throughout the respiratory cycle. PaO2 is the measure of oxygen level dissolved in the arterial blood. | Baseline, Hours 6, 12 and 24 after start of infusion |
| Change From Baseline in Differential Saturation at Hours 6, 12 and 24 Post-Infusion | Differential oxygenation saturation is a simple way to detect the right-to left shunting at ductus arteriosus using 2 pulse oximeters. It is the difference between pre-ductal and post-ductal sites pulse oxygen saturation (SpO2). Where, pre-duct refers to right upper extremity and post-duct refers to lower limb. Oxygenation saturation is measured as percentage of hemoglobin binding sites occupied by oxygen in the blood. | Baseline, Hours 6, 12 and 24 after start of infusion |
| Change From Baseline in Ratio of Partial Pressure of Oxygen in Arterial Blood to Fraction of Inspired Oxygen (P/F) at Hours 6, 12 and 24 | The ratio of partial pressure of arterial oxygen to fraction of inspired oxygen is a ratio between the oxygen level in the arterial blood and the oxygen concentration that is breathed. It helps to determine the degree of any problems with how the lungs transfer oxygen to the blood. | Baseline, Hours 6, 12 and 24 after start of infusion |
| Maximum Plasma Concentration (Cmax) of Sildenafil and Its Metabolite | Cmax was obtained for Sildenafil and its major metabolite UK-103,320. | Loading dose, Day 1: prior to the start of infusion, 5, 30 minutes after end of loading infusion; Maintenance dose: 48 to 72, 96 to 120 hours during infusion and immediately prior to end of maintenance infusion (up to maximum on Day 14) |
| Total Plasma Clearance (CL) of Sildenafil and Its Metabolite | CL is volume of the body fluid/ plasma from which the drug or the metabolite is completely removed per unit time. CL was obtained for Sildenafil and its major metabolite UK-103,320. | Loading dose: prior to the start of infusion, 5, 30 minutes after end of loading infusion on Day 1; Maintenance dose: between 48 to 72, 96 to 120 hours during infusion and immediately prior to end of infusion on Day 1 |
| Central Volume of Distribution (Vc) of Sildenafil and Its Metabolite | Vc is the hypothetical volume into which a drug or a metabolite initially distributes upon administration. It was determined by using a population-based analysis, non-linear mixed-effects modeling (NONMEM), version 7.4.0. Vc was calculated for Sildenafil and its major metabolite, UK-103,320. | Loading dose: prior to the start of infusion, 5, 30 minutes after end of loading infusion on Day 1; Maintenance dose: between 48 to 72, 96 to 120 hours during infusion and immediately prior to end of infusion on Day 1 |
| Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was any untoward medical occurrence in a subject who received study drug without regard to possibility of causal relationship. A SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. Treatment-emergent are events between first infusion of study drug and up to 31 days after end of study drug infusion (up to 45 days) that were absent before treatment or that worsened relative to pretreatment state. AEs included both SAEs and non-SAEs. | Baseline up to 31 days after end of study drug infusion (up to 45 days) |
| Number of Treatment-Emergent Adverse Events (AEs) According to Severity | AE: untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. SAE: AE resulting in any of the following outcomes: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. Treatment-emergent are events between first infusion of study drug and up to 31 days after end of study drug infusion (up to 45 days) that were absent before treatment or that worsened relative to pretreatment state. AEs included both SAEs and non-SAEs. Severity criteria: mild=did not interfere with subject's usual function; moderate=interfered to some extent with participant's usual function and severe=interfered significantly with participant's usual function. Missing baseline severities were imputed as mild. | Baseline up to 31 days after end of study drug infusion (up to 45 days) |
| Number of Participants With Laboratory Abnormalities | Criteria for laboratory values: Hematology: hemoglobin, hematocrit, red blood cell count <0.8*lower limit of normal (LLN), platelets<0.5*LLN, >1.75*upper limit of normal (ULN), white blood cells count <0.6*LLN, >1.5*ULN; Liver function: total and direct bilirubin >1.5*ULN, aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase >3.0*ULN, total protein <0.8*LLN, >1.2*ULN; Renal function: blood urea nitrogen, creatinine >1.3*ULN; Electrolytes: sodium <0.95*LLN, >1.05*ULN, potassium, chloride, calcium, bicarbonate (venous) <0.9*LLN, >1.1*ULN. | Up to 14 days from initiation of study drug infusion |
| Part B: Composite Scores of Cognitive, Language, and Motor Developmental Progress of Participants as Assessed by Bayley Scales of Infant and Toddler Development Third Edition (Bayley-III) | Bayley-III assesses infant and toddler development across five domains: cognitive, language, motor, social-emotional (SE), and adaptive behavior (AB). Assessments of the cognitive, language, and motor domains conducted using items administered to the child; assessments of the SE and AB domains conducted using the primary caregiver's responses to a questionnaire. Score ranges: cognitive scale 0-91, language scale 0-97 and motor scale 0-132, where higher scores indicated better cognitive function, communication and motor skills respectively. Raw scores of cognitive, language and motor domains were converted to composite scores. Composite scores of cognitive, language and motor developmental scales ranged from a scale of 40 to 160, where higher score indicated stronger skills and abilities. In this outcome measure composite scores for infants and toddlers were reported at month 12 and 24. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Composite Scores of Social-Emotional and Adaptive Behavior Questionnaire as Assessed by Bayley Scales of Infant and Toddler Development Third Edition (Bayley-III) | The Bayley-III assesses infant and toddler development across five domains: cognitive, language, motor, social-emotional (SE), and adaptive behavior (AB). Assessments of the cognitive, language, and motor domains conducted using items administered to the child; assessments of the SE and AB domains conducted using the primary caregiver's responses to a questionnaire. The questionnaire comprises the SE scale (35 items) and the AB scale (241 items). Raw scores of SE and AB were converted to composite scores. Composite scores for SE and AB scale ranged from 40 to 160, where higher scores indicated better social-emotional skills and adaptive behavior in child. In this outcome measure composite scores for parent/caregiver were reported at month 24. | Month 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Number of Participants With Eye Movement Disorders as Assessed by Eye Examination | Standard age-appropriate ophthalmological examinations were used to assess eye movement disorders (presence of amblyopia, strabismus, and nystagmus) at month 12 and 24. In this outcome measure, data have been reported for right and left eye separately. Rows according to eye movement disorder categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Visual Acuity of Verbal Participants as Assessed by Ophthalmological Assessment | Standard age-appropriate ophthalmological examinations were used to assess visual acuity (performed differently for children able of verbal interaction) through visual acuity chart (VAC) quantitative, counting finger (CF), hand motion (HM), light perception (LP), no light perception (NLP) and missing at month 12 and 24. In this outcome measure, data have been reported for right and left eye separately. Rows according to visual acuity categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Visual Acuity of Non-Verbal Participants as Assessed by Ophthalmological Assessment | Standard age-appropriate ophthalmological examinations were used to assess visual acuity (performed differently for children unable of verbal interaction) through fixates and follows (included central, steady and maintained), light perception (wince to light), no light perception, and missing at month 12 and 24. In this outcome measure, data have been reported for right and left eye separately. Rows according to visual acuity categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Visual Acuity of Verbal Participants as Assessed by LogMAR Through Visual Acuity Chart | Standard age-appropriate ophthalmological examinations were used to assess visual acuity (performed differently for children able of verbal interaction) at month 12 and 24. Visual acuity (VA) of verbal children was assessed for each eye using the Snellen method, where logarithm of minimum angle of resolution (logMAR) units were derived from the Snellen ratios. Participants had to read letters from the chart at a distance of 20 feet/6 meter or 4 meter. VA (Snellen ratio) = distance between the chart and participant, divided by distance at which participant was able to see/read chart without impairment; expressed as decimal, logMAR = log10 (1/decimal VA). In this outcome measure, data have been reported for right and left eye separately. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Visual Status of Participants With Abnormality as Assessed by Eye Examination of the Anterior and Posterior Segments | Standard age-appropriate ophthalmological examinations were used to assess examination of anterior and posterior chamber for abnormality in lids, conjunctiva, cornea, anterior chamber, lens, iris, pupil, extraocular muscle movement and eye movements at month 12 and 24. In this outcome measure, data have been reported for right and left eye separately. Rows according to visual status categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Behavior Hearing Assessment Through Pure Tone Audiometry Test | Audiological evaluations of participants were recorded and reported using behavior hearing assessment through pure tone audiometry test which includes participants with normal, abnormal, incomplete/inconclusive behavior at month 12 and 24. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Bone Conduction Through Pure Tone Audiometry Test | Audiological evaluations of participants were recorded and reported by bone conduction assessment through pure tone audiometry test which included participants with sensorineural hearing loss, conductive hearing loss, mixed hearing loss, neural, and unspecified at month 12 and 24. Rows according to bone conduction categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Air Conduction Via Phones/Headphones Through Pure Tone Audiometry Test | Audiological evaluations of participants were recorded and reported by air conduction via phones/headphones through pure tone audiometry test which included participants with hearing loss ranged from less than or equal to (<=) 20 decibel hearing loss (DB HL), 21-40 DB HL, 41-70 DB HL, 71-90 DB HL, greater than (>) 90 DB HL or no response, and missing at frequencies ranged from 500 Hertz (Hz) to 8000 Hz at month 12 and 24. In this outcome measure, data have been reported for right and left ear separately. Rows according to air conduction categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Air Conduction Via Soundfield Through Pure Tone Audiometry Test | Audiological evaluations of participants were recorded and reported by air conduction via soundfield through pure tone audiometry test which included participants with hearing loss ranged from <=20 DB HL, 21-40 DB HL, 41-70 DB HL, 71-90 DB HL, >90 DB HL or no response, and missing at frequencies ranged from 500 Hz to 4000 Hz at month 12 and 24. Rows according to air conduction categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Tympanometry Assessment (Peak Pressure) Through Immittance Audiometry Test | Audiological evaluations of participants were recorded and reported by tympanometry assessment through immittance audiometry test which included participants with peak pressure signs (+) and (-) at month 12 and 24. In this outcome measure, data have been reported for right and left ear separately. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Tympanometry Assessment (Static Acoustic Admittance) Through Immittance Audiometry Test | Audiological evaluations of participants were recorded and reported by tympanometry assessment through immittance audiometry test which included participants with static acoustic admittance at month 12 and 24. In this outcome measure, data have been reported for right and left ear separately. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Ipsilateral Stapedial Reflex Through Immittance Audiometry Test | Audiological evaluations of participants were recorded and reported by ipsilateral stapedial reflex through immittance audiometry test which included participants with presence of ipsilateral stapedial reflex at frequencies ranged from 500 Hz to 2000 Hz at month 12 and 24. Ipsilateral stapedial reflex measures are used to assess the neural pathway surrounding the stapedial reflex, which occurs in response to a loud sound (70 to 90 decibel above threshold). In this outcome measure, data have been reported for right and left ear separately. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Transient Evoked Emission Through Otoacoustic Emissions Assessment | Audiological evaluations of participants were recorded and reported by transient evoked emission through otoacoustic emissions assessment which included participants with presence of transient evoked emissions from frequencies 1000 Hz to 4000 Hz at month 12 and 24. In this outcome measure, data have been reported for right and left ear separately. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Audiological Status of Participants as Assessed by Distort Product Through Otoacoustic Emissions Assessment | Audiological evaluations of participants were recorded and reported by distort product through otoacoustic emissions assessment which included participants with presence of distort product at frequencies ranged from 2000 Hz to 8000 Hz at month 12 and 24. Distortion-product otoacoustic emissions (DPOAEs) are generated in the cochlea in response to two tones of a given frequency and sound pressure level presented in the ear canal. Distort product otoacoustic emissions are an objective indicator of normally functioning cochlea outer hair cells. In this outcome measure, data have been reported for right and left ear separately. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and Deaths | An AE was any untoward medical occurrence in a participant who received study medication without regard to possibility of causal relationship to it. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/ incapacity; congenital anomaly. AEs included both serious and all non-serious AEs. | up to 24 months after end of study treatment in Part A (maximum up to 26 months) |
| Part B: Neurological Progress of Participants as Assessed by the Neurology Optimality Score | The Hammersmith Infant Neurological Examination (HINE) was a standard scoring examination to assess development of cranial nerve; posture; movement; tone; and reflexes and reaction. HINE exam global score is a sum of subset (cranial nerve, posture, movement, tone, reflexes and reactions) scores, ranged from 0 to 78, where higher score represents better outcome. Here, the HINE global scores were reported at month 12 and 24. | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
| Sacramento |
| California |
| 95817 |
| United States |
| University of California Davis | Sacramento | California | 95817 | United States |
| University of California Davis Medical Center | Sacramento | California | 95825 | United States |
| Children´s National Medical Center | Washington D.C. | District of Columbia | 20010 | United States |
| Indiana University Health Methodist Hospital | Indianapolis | Indiana | 46202 | United States |
| Riley Hospital for Children at IU Health | Indianapolis | Indiana | 46202 | United States |
| Sydney and Lois Eskenazi Hospital | Indianapolis | Indiana | 46202 | United States |
| Children's Mercy Hospitals & Clinics | Kansas City | Missouri | 64108 | United States |
| Duke University Medical Center (DUMC) | Durham | North Carolina | 27710 | United States |
| Fairview Hospital | Cleveland | Ohio | 44111 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| OU Follow-Up Program, PREMIEr Clinic, Children's Hospital | Oklahoma City | Oklahoma | 73104 | United States |
| OU Neonatal Intensive Care Unit at Children's Hospital | Oklahoma City | Oklahoma | 73104 | United States |
| The University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | United States |
| Henry Zarrow Neonatal Intensive Care Unit, Children's Hospital at Saint Francis | Tulsa | Oklahoma | 74136 | United States |
| Warren Cancer Research Foundation | Tulsa | Oklahoma | 74136 | United States |
| Vanderbilt Children's Hospital | Nashville | Tennessee | 37232 | United States |
| Seattle Children's Research Institute | Seattle | Washington | 98101 | United States |
| Seattle Childrens Hospital | Seattle | Washington | 98105 | United States |
| Universitair Ziekenhuis Antwerpen | Edegem | 2650 | Belgium |
| UZ Gent | Ghent | 9000 | Belgium |
| CHUL du CHU de Quebec | Québec | Quebec | G1V 4G2 | Canada |
| Aarhus Universitetshospital, Skejby | Aarhus N | 8200 | Denmark |
| Neonatalklinikken Rigshospitalet, 5024 | Copenhagen Ø | 2100 | Denmark |
| Centre Hospitalier et Regional de Lille - Hopital Jeanne de Flandre | Lille | 59037 | France |
| Centre Hospitalier et Régional de Lille, | Lille | 59037 | France |
| Hôpital de la Conception Assistance Publique-Hôpitaux de Marseille | Marseille | 13385 | France |
| CHU Robert Debré | Paris | 75019 | France |
| Hopital NECKER - Enfants Malades | Paris | 75743 | France |
| University Hospital of Leipzig | Leipzig | 04103 | Germany |
| Neonatologia Fondazione IRCCS Policlinico San Matteo | Pavia | 27100 | Italy |
| Radboud University Nijmegen Medical Centre | Nijmegen | 6525 GA | Netherlands |
| Erasmus MC, Sophia Children's hospital | Rotterdam | 3015 CN | Netherlands |
| Haukeland University Hospital | Bergen | Haukeland | 5000 | Norway |
| Hospital Sant Joan de Deu | Esplugues de Llobregat | Barcelona / Spain | 08950 | Spain |
| Hospital General Universitario Gregorio Marañon | Madrid | 28007 | Spain |
| Karolinska University Hospital | Stockholm | 171 76 | Sweden |
| St. Michael's Hospital | Bristol | BS2 8EG | United Kingdom |
| Leicester Royal Infirmary | Leicester | LE1 5WW | United Kingdom |
| Glenfield Hospital, University Hospitals of Leicester NHS Trust | Leicester | LE3 9QP | United Kingdom |
| Great Ormond Street Hospital NHS Foundation Trust | London | WC1N 3JH | United Kingdom |
| FG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
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| NOT COMPLETED |
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| Part B |
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Safety population included all participants treated with study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | IV Sildenafil | Part A: Participants received sildenafil intravenously based on their body weight at a loading dose of 0.1 milligrams per kg (mg/kg) for 30 minutes on Day 1 followed by a maintenance dose of 0.03 milligrams per kg per hour (mg/kg/hr), for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
| BG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Continuous | Mean | Standard Deviation | days |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Time on Inhaled Nitric Oxide (iNO) Treatment After Initiation of Intravenous (IV) Study Drug For Participants Without Treatment Failure | Time in days, on iNO treatment, for participants without iNO treatment failure, was calculated 14 days from the initiation of IV study drug or hospital discharge, whichever occurred first. iNO treatment failure was defined as need for additional treatment targeting PPHN, need for extra corporeal membrane oxygenation (ECMO), or death during the study. | The intent-to-treat population (ITT) included all randomized participants treated with study treatment. Here "Overall number of participants analyzed" signifies number of participants without iNO treatment failure. | Posted | Least Squares Mean | 95% Confidence Interval | days | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
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| Primary | Treatment Failure Rate | Treatment failure rate was defined as percentage of participants who needed additional treatment targeting PPHN, needed ECMO, or died during the study. | The ITT population included all randomized participants treated with study treatment. | Posted | Number | 95% Confidence Interval | percentage of participants | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
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| Secondary | Time From Initiation of Intravenous (IV) Study Drug to Final Weaning of Mechanical Ventilation | Time in days, from initiation of IV study drug to final weaning of mechanical ventilation among participants achieving final weaning of mechanical ventilation for PPHN was evaluated. Kaplan-Meier method was used for estimation. For participants with mechanical ventilation beyond 336 hours (14 days) from initiation of IV study drug, data was censored at 14 days. | The ITT population included all randomized participants treated with study treatment. | Posted | Median | 95% Confidence Interval | days | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
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| Secondary | Time From Initiation of Intravenous (IV) Study Drug to First Treatment Failure | Time in days, from initiation of IV study drug to first treatment failure (defined as need for additional treatment targeting PPHN, need for ECMO, or death) for participants with treatment failure was evaluated. Kaplan-Meier method was used for estimation. For participants without treatment failure by the endpoint assessment date, data was censored at the endpoint assessment date. | The ITT population included all randomized participants treated with study treatment. | Posted | Median | 95% Confidence Interval | days | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
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| Secondary | Percentage of Participants With Individual Components of Treatment Failure | Percentage of participants with individual components of treatment failure (need to start additional treatment targeting PPHN, need to start ECMO, or death) were evaluated. Some participants could have had multiple qualifying events for treatment failure. | The ITT population included all randomized participants treated with study treatment. | Posted | Number | 95% Confidence Interval | percentage of participants | 14 days from the initiation of IV study drug or hospital discharge, whichever occurs first (maximum of 14 days) |
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| Secondary | Change From Baseline in Oxygenation Index (OI) at Hours 6, 12 and 24 Post-Infusion | Oxygenation index was calculated as the product of fraction of inspired oxygen (FiO2) and mean airway pressure divided by partial pressure of oxygen dissolved in arterial blood (PaO2) [(FiO2*mean airway pressure)/PaO2] measured in centimeter of water per millimeter of mercury (cmH2O/mmHg). FiO2 is the measure of oxygen concentration that is breathed. Mean airway pressure is defined as an average of the airway pressure throughout the respiratory cycle. PaO2 is the measure of oxygen level dissolved in the arterial blood. | The ITT population included all randomized participants treated with study treatment. Here, 'Number analyzed' = Participants evaluable for this outcome measure at specified rows. | Posted | Least Squares Mean | 95% Confidence Interval | cmH2O/mmHg | Baseline, Hours 6, 12 and 24 after start of infusion |
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| Secondary | Change From Baseline in Differential Saturation at Hours 6, 12 and 24 Post-Infusion | Differential oxygenation saturation is a simple way to detect the right-to left shunting at ductus arteriosus using 2 pulse oximeters. It is the difference between pre-ductal and post-ductal sites pulse oxygen saturation (SpO2). Where, pre-duct refers to right upper extremity and post-duct refers to lower limb. Oxygenation saturation is measured as percentage of hemoglobin binding sites occupied by oxygen in the blood. | The ITT population included all randomized participants treated with study treatment. Here, 'Number analyzed' = Participants evaluable for this outcome measure at specified rows. | Posted | Least Squares Mean | 95% Confidence Interval | percentage of hemoglobin | Baseline, Hours 6, 12 and 24 after start of infusion |
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| Secondary | Change From Baseline in Ratio of Partial Pressure of Oxygen in Arterial Blood to Fraction of Inspired Oxygen (P/F) at Hours 6, 12 and 24 | The ratio of partial pressure of arterial oxygen to fraction of inspired oxygen is a ratio between the oxygen level in the arterial blood and the oxygen concentration that is breathed. It helps to determine the degree of any problems with how the lungs transfer oxygen to the blood. | The ITT population included all randomized participants treated with study treatment. Here, 'Number analyzed' = Participants evaluable for this outcome measure at specified rows. | Posted | Least Squares Mean | 95% Confidence Interval | ratio | Baseline, Hours 6, 12 and 24 after start of infusion |
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| Secondary | Maximum Plasma Concentration (Cmax) of Sildenafil and Its Metabolite | Cmax was obtained for Sildenafil and its major metabolite UK-103,320. | Pharmacokinetic (PK) analysis set included all participants randomized and treated who had at least 1 concentration during whole treatment period. | Posted | Mean | Standard Deviation | nanogram per milliliter | Loading dose, Day 1: prior to the start of infusion, 5, 30 minutes after end of loading infusion; Maintenance dose: 48 to 72, 96 to 120 hours during infusion and immediately prior to end of maintenance infusion (up to maximum on Day 14) |
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| Secondary | Total Plasma Clearance (CL) of Sildenafil and Its Metabolite | CL is volume of the body fluid/ plasma from which the drug or the metabolite is completely removed per unit time. CL was obtained for Sildenafil and its major metabolite UK-103,320. | PK analysis set included all participants randomized and treated who had at least 1 concentration during whole treatment period. | Posted | Mean | Standard Deviation | Liters/hour | Loading dose: prior to the start of infusion, 5, 30 minutes after end of loading infusion on Day 1; Maintenance dose: between 48 to 72, 96 to 120 hours during infusion and immediately prior to end of infusion on Day 1 |
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| Secondary | Central Volume of Distribution (Vc) of Sildenafil and Its Metabolite | Vc is the hypothetical volume into which a drug or a metabolite initially distributes upon administration. It was determined by using a population-based analysis, non-linear mixed-effects modeling (NONMEM), version 7.4.0. Vc was calculated for Sildenafil and its major metabolite, UK-103,320. | PK analysis set included all participants randomized and treated who had at least 1 concentration during whole treatment period. | Posted | Mean | Standard Deviation | Liters | Loading dose: prior to the start of infusion, 5, 30 minutes after end of loading infusion on Day 1; Maintenance dose: between 48 to 72, 96 to 120 hours during infusion and immediately prior to end of infusion on Day 1 |
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| Secondary | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was any untoward medical occurrence in a subject who received study drug without regard to possibility of causal relationship. A SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. Treatment-emergent are events between first infusion of study drug and up to 31 days after end of study drug infusion (up to 45 days) that were absent before treatment or that worsened relative to pretreatment state. AEs included both SAEs and non-SAEs. | The safety population included all participants treated with study treatment. | Posted | Count of Participants | Participants | Baseline up to 31 days after end of study drug infusion (up to 45 days) |
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| Secondary | Number of Treatment-Emergent Adverse Events (AEs) According to Severity | AE: untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. SAE: AE resulting in any of the following outcomes: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. Treatment-emergent are events between first infusion of study drug and up to 31 days after end of study drug infusion (up to 45 days) that were absent before treatment or that worsened relative to pretreatment state. AEs included both SAEs and non-SAEs. Severity criteria: mild=did not interfere with subject's usual function; moderate=interfered to some extent with participant's usual function and severe=interfered significantly with participant's usual function. Missing baseline severities were imputed as mild. | The safety population included all participants treated with study treatment. | Posted | Number | events | Baseline up to 31 days after end of study drug infusion (up to 45 days) |
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| Secondary | Number of Participants With Laboratory Abnormalities | Criteria for laboratory values: Hematology: hemoglobin, hematocrit, red blood cell count <0.8*lower limit of normal (LLN), platelets<0.5*LLN, >1.75*upper limit of normal (ULN), white blood cells count <0.6*LLN, >1.5*ULN; Liver function: total and direct bilirubin >1.5*ULN, aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase >3.0*ULN, total protein <0.8*LLN, >1.2*ULN; Renal function: blood urea nitrogen, creatinine >1.3*ULN; Electrolytes: sodium <0.95*LLN, >1.05*ULN, potassium, chloride, calcium, bicarbonate (venous) <0.9*LLN, >1.1*ULN. | The safety population included all participants treated with study treatment. Here "Overall number of participants analyzed" signifies participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | Up to 14 days from initiation of study drug infusion |
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| Secondary | Part B: Composite Scores of Cognitive, Language, and Motor Developmental Progress of Participants as Assessed by Bayley Scales of Infant and Toddler Development Third Edition (Bayley-III) | Bayley-III assesses infant and toddler development across five domains: cognitive, language, motor, social-emotional (SE), and adaptive behavior (AB). Assessments of the cognitive, language, and motor domains conducted using items administered to the child; assessments of the SE and AB domains conducted using the primary caregiver's responses to a questionnaire. Score ranges: cognitive scale 0-91, language scale 0-97 and motor scale 0-132, where higher scores indicated better cognitive function, communication and motor skills respectively. Raw scores of cognitive, language and motor domains were converted to composite scores. Composite scores of cognitive, language and motor developmental scales ranged from a scale of 40 to 160, where higher score indicated stronger skills and abilities. In this outcome measure composite scores for infants and toddlers were reported at month 12 and 24. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Mean | Standard Deviation | units on a scale | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Composite Scores of Social-Emotional and Adaptive Behavior Questionnaire as Assessed by Bayley Scales of Infant and Toddler Development Third Edition (Bayley-III) | The Bayley-III assesses infant and toddler development across five domains: cognitive, language, motor, social-emotional (SE), and adaptive behavior (AB). Assessments of the cognitive, language, and motor domains conducted using items administered to the child; assessments of the SE and AB domains conducted using the primary caregiver's responses to a questionnaire. The questionnaire comprises the SE scale (35 items) and the AB scale (241 items). Raw scores of SE and AB were converted to composite scores. Composite scores for SE and AB scale ranged from 40 to 160, where higher scores indicated better social-emotional skills and adaptive behavior in child. In this outcome measure composite scores for parent/caregiver were reported at month 24. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | units on a scale | Month 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Number of Participants With Eye Movement Disorders as Assessed by Eye Examination | Standard age-appropriate ophthalmological examinations were used to assess eye movement disorders (presence of amblyopia, strabismus, and nystagmus) at month 12 and 24. In this outcome measure, data have been reported for right and left eye separately. Rows according to eye movement disorder categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Visual Acuity of Verbal Participants as Assessed by Ophthalmological Assessment | Standard age-appropriate ophthalmological examinations were used to assess visual acuity (performed differently for children able of verbal interaction) through visual acuity chart (VAC) quantitative, counting finger (CF), hand motion (HM), light perception (LP), no light perception (NLP) and missing at month 12 and 24. In this outcome measure, data have been reported for right and left eye separately. Rows according to visual acuity categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Visual Acuity of Non-Verbal Participants as Assessed by Ophthalmological Assessment | Standard age-appropriate ophthalmological examinations were used to assess visual acuity (performed differently for children unable of verbal interaction) through fixates and follows (included central, steady and maintained), light perception (wince to light), no light perception, and missing at month 12 and 24. In this outcome measure, data have been reported for right and left eye separately. Rows according to visual acuity categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Visual Acuity of Verbal Participants as Assessed by LogMAR Through Visual Acuity Chart | Standard age-appropriate ophthalmological examinations were used to assess visual acuity (performed differently for children able of verbal interaction) at month 12 and 24. Visual acuity (VA) of verbal children was assessed for each eye using the Snellen method, where logarithm of minimum angle of resolution (logMAR) units were derived from the Snellen ratios. Participants had to read letters from the chart at a distance of 20 feet/6 meter or 4 meter. VA (Snellen ratio) = distance between the chart and participant, divided by distance at which participant was able to see/read chart without impairment; expressed as decimal, logMAR = log10 (1/decimal VA). In this outcome measure, data have been reported for right and left eye separately. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Mean | Standard Deviation | LogMAR | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Visual Status of Participants With Abnormality as Assessed by Eye Examination of the Anterior and Posterior Segments | Standard age-appropriate ophthalmological examinations were used to assess examination of anterior and posterior chamber for abnormality in lids, conjunctiva, cornea, anterior chamber, lens, iris, pupil, extraocular muscle movement and eye movements at month 12 and 24. In this outcome measure, data have been reported for right and left eye separately. Rows according to visual status categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Behavior Hearing Assessment Through Pure Tone Audiometry Test | Audiological evaluations of participants were recorded and reported using behavior hearing assessment through pure tone audiometry test which includes participants with normal, abnormal, incomplete/inconclusive behavior at month 12 and 24. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Bone Conduction Through Pure Tone Audiometry Test | Audiological evaluations of participants were recorded and reported by bone conduction assessment through pure tone audiometry test which included participants with sensorineural hearing loss, conductive hearing loss, mixed hearing loss, neural, and unspecified at month 12 and 24. Rows according to bone conduction categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Air Conduction Via Phones/Headphones Through Pure Tone Audiometry Test | Audiological evaluations of participants were recorded and reported by air conduction via phones/headphones through pure tone audiometry test which included participants with hearing loss ranged from less than or equal to (<=) 20 decibel hearing loss (DB HL), 21-40 DB HL, 41-70 DB HL, 71-90 DB HL, greater than (>) 90 DB HL or no response, and missing at frequencies ranged from 500 Hertz (Hz) to 8000 Hz at month 12 and 24. In this outcome measure, data have been reported for right and left ear separately. Rows according to air conduction categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Air Conduction Via Soundfield Through Pure Tone Audiometry Test | Audiological evaluations of participants were recorded and reported by air conduction via soundfield through pure tone audiometry test which included participants with hearing loss ranged from <=20 DB HL, 21-40 DB HL, 41-70 DB HL, 71-90 DB HL, >90 DB HL or no response, and missing at frequencies ranged from 500 Hz to 4000 Hz at month 12 and 24. Rows according to air conduction categories at specified time points are reported in this outcome measure, only when there was a non-zero data for at least 1 reporting arm. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Tympanometry Assessment (Peak Pressure) Through Immittance Audiometry Test | Audiological evaluations of participants were recorded and reported by tympanometry assessment through immittance audiometry test which included participants with peak pressure signs (+) and (-) at month 12 and 24. In this outcome measure, data have been reported for right and left ear separately. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Mean | Standard Deviation | decapascals | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Tympanometry Assessment (Static Acoustic Admittance) Through Immittance Audiometry Test | Audiological evaluations of participants were recorded and reported by tympanometry assessment through immittance audiometry test which included participants with static acoustic admittance at month 12 and 24. In this outcome measure, data have been reported for right and left ear separately. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Mean | Standard Deviation | millimho | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Ipsilateral Stapedial Reflex Through Immittance Audiometry Test | Audiological evaluations of participants were recorded and reported by ipsilateral stapedial reflex through immittance audiometry test which included participants with presence of ipsilateral stapedial reflex at frequencies ranged from 500 Hz to 2000 Hz at month 12 and 24. Ipsilateral stapedial reflex measures are used to assess the neural pathway surrounding the stapedial reflex, which occurs in response to a loud sound (70 to 90 decibel above threshold). In this outcome measure, data have been reported for right and left ear separately. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Transient Evoked Emission Through Otoacoustic Emissions Assessment | Audiological evaluations of participants were recorded and reported by transient evoked emission through otoacoustic emissions assessment which included participants with presence of transient evoked emissions from frequencies 1000 Hz to 4000 Hz at month 12 and 24. In this outcome measure, data have been reported for right and left ear separately. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Audiological Status of Participants as Assessed by Distort Product Through Otoacoustic Emissions Assessment | Audiological evaluations of participants were recorded and reported by distort product through otoacoustic emissions assessment which included participants with presence of distort product at frequencies ranged from 2000 Hz to 8000 Hz at month 12 and 24. Distortion-product otoacoustic emissions (DPOAEs) are generated in the cochlea in response to two tones of a given frequency and sound pressure level presented in the ear canal. Distort product otoacoustic emissions are an objective indicator of normally functioning cochlea outer hair cells. In this outcome measure, data have been reported for right and left ear separately. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Count of Participants | Participants | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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| Secondary | Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and Deaths | An AE was any untoward medical occurrence in a participant who received study medication without regard to possibility of causal relationship to it. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/ incapacity; congenital anomaly. AEs included both serious and all non-serious AEs. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. | Posted | Count of Participants | Participants | up to 24 months after end of study treatment in Part A (maximum up to 26 months) |
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| Secondary | Part B: Neurological Progress of Participants as Assessed by the Neurology Optimality Score | The Hammersmith Infant Neurological Examination (HINE) was a standard scoring examination to assess development of cranial nerve; posture; movement; tone; and reflexes and reaction. HINE exam global score is a sum of subset (cranial nerve, posture, movement, tone, reflexes and reactions) scores, ranged from 0 to 78, where higher score represents better outcome. Here, the HINE global scores were reported at month 12 and 24. | Part B safety analysis set included all participants enrolled in Part B of the study for data analyses on developmental progress, audiological, neurological and ophthalmological assessment, and safety. Here 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'number analyzed' signifies number of participants evaluable for each specified row. | Posted | Mean | Standard Deviation | units on a scale | Month 12 and 24 after end of study treatment in Part A (Day 1 to 14) |
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Part A: Baseline up to 31 days after end of study drug infusion (up to 45 days); Part B: up to 24 months after end of study treatment in Part A (maximum up to 26 months)
Same event may appear as both an AE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another, or a participant may have experienced both a serious and non-serious event.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
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| EG000 | Part A: IV Sildenafil | Part A: Participants received sildenafil intravenously based on their body weight at a loading dose of 0.1 milligrams per kg (mg/kg) for 30 minutes on Day 1 followed by a maintenance dose of 0.03 milligrams per kg per hour (mg/kg/hr), for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. | 2 | 29 | 7 | 29 | 20 | 29 |
| EG001 | Part A: Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. | 1 | 30 | 2 | 30 | 19 | 30 |
| EG002 | Part B: IV Sildenafil | Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. | 0 | 27 | 9 | 27 | 14 | 27 |
| EG003 | Part B: Placebo | Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. | 2 | 26 | 6 | 26 | 15 | 26 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiomyopathy | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
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| Pulmonary malformation | Congenital, familial and genetic disorders | MedDRA v23.0 | Non-systematic Assessment |
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| Drug withdrawal syndrome | General disorders | MedDRA v23.0 | Non-systematic Assessment |
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| Urosepsis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Myoclonus | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypertensive crisis | Vascular disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Gastroesophageal Reflux | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Bronchiolitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Bronchopneumonia | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Skull fracture | Injury, poisoning and procedural complications | MedDRA v23.0 | Non-systematic Assessment |
| |
| Food allergy | Immune system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Apnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Viral upper respiratory tract infection | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pulmonary hypertension crisis | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Congestive cardiomyopathy | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Congenital heart disease | Congenital, familial and genetic disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Bradycardia neonatal | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Junctional ectopic tachycardia | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Myocardial ischaemia | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Sinus bradycardia | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Persistent foetal circulation | Congenital, familial and genetic disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Eye oedema | Eye disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Periorbital oedema | Eye disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pupil fixed | Eye disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Gastric haemorrhage | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Intestinal perforation | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Disease progression | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Drug withdrawal syndrome | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Drug withdrawal syndrome neonatal | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Generalised oedema | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Infusion site extravasation | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Malaise | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Oedema | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Secretion discharge | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Withdrawal syndrome | General disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Cholestasis | Hepatobiliary disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Jaundice cholestatic | Hepatobiliary disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Nosocomial infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Tracheitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Procedural hypertension | Injury, poisoning and procedural complications | MedDRA v23.0 | Non-systematic Assessment |
| |
| Procedural hypotension | Injury, poisoning and procedural complications | MedDRA v23.0 | Non-systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA v23.0 | Non-systematic Assessment |
| |
| Underdose | Injury, poisoning and procedural complications | MedDRA v23.0 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Blood albumin decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Blood calcium decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Blood magnesium decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Blood methaemoglobin present | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Haematocrit decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| PCO2 decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Staphylococcus test positive | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Thyroid function test abnormal | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Acidosis | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Alkalosis hypochloraemic | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Feeding intolerance | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Fluid retention | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hyperchloraemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypochloraemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypoproteinaemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Brain injury | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Cerebral ischaemia | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypertonia | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Motor dysfunction | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Vocal cord paralysis | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Selective eating disorder | Psychiatric disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Oliguria | Renal and urinary disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Oedema genital | Reproductive system and breast disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hiccups | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Neonatal asphyxia | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pulmonary air leakage | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pulmonary interstitial emphysema syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Respiratory tract oedema | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Stridor | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Haemodynamic instability | Vascular disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Cerebral palsy | Congenital, familial and genetic disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Plagiocephaly | Congenital, familial and genetic disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Conductive deafness | Ear and labyrinth disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hypermetropia | Eye disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Strabismus | Eye disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Acute sinusitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Bronchiolitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Herpangina | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Roseola | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Post procedural hypotension | Injury, poisoning and procedural complications | MedDRA v23.0 | Non-systematic Assessment |
| |
| Adenovirus test positive | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Blood pyruvic acid increased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Gastric residual assessment | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Haemoglobin abnormal | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Heart rate decreased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Heart rate increased | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Prothrombin time prolonged | Investigations | MedDRA v23.0 | Non-systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Weight gain poor | Metabolism and nutrition disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Intra-abdominal haemangioma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v23.0 | Non-systematic Assessment |
| |
| Phrenic nerve paralysis | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Psychomotor skills impaired | Nervous system disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Drug dependence | Psychiatric disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Chronic respiratory disease | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Respiratory acidosis | Respiratory, thoracic and mediastinal disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA v23.0 | Non-systematic Assessment |
| |
| Thrombosis | Vascular disorders | MedDRA v23.0 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 8007181021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan: Redacted SAP Part A of study | Jan 8, 2019 | Oct 16, 2019 | SAP_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan: Redacted SAP Part B of Study | Mar 16, 2015 | Jun 1, 2021 | SAP_002.pdf |
| ID | Term |
|---|---|
| D010547 | Persistent Fetal Circulation Syndrome |
| ID | Term |
|---|---|
| D006976 | Hypertension, Pulmonary |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D007232 | Infant, Newborn, Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| D000068677 | Sildenafil Citrate |
| ID | Term |
|---|---|
| D013449 | Sulfonamides |
| D000577 | Amides |
| D009930 | Organic Chemicals |
| D013450 | Sulfones |
| D013457 | Sulfur Compounds |
| D010879 | Piperazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D011687 | Purines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
Not provided
Not provided
| Lost to Follow-up |
|
| Death |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
|
|
|
|
|
|
|
|
Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
|
|
|
|
|
|
|
|
|
|
|
| Placebo |
Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 |
| Placebo |
Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
|
|
Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
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Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
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| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
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Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
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| OG001 | Placebo | Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
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Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
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| OG001 |
| Placebo |
Participants received placebo (0.9 % normal saline or dextrose 10%) at a rate matched to sildenafil infusion, intravenously, based on participant's weight, for a minimum of 2 days and maximum of 14 days. Infusion continuation was upon investigator discretion in view of participants' safety and well-being. Part B: Participants who started Part A (not necessarily completed Part A) and who were eligible and consented, continued to be followed up in part B of the study. |
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