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A multicenter, double blind, randomized, single dose, active-controlled study to investigate the efficacy and safety of synthetic surfactant (CHF 5633) in comparison to porcine surfactant (Poractant alfa, Curosurf ®) in the treatment of preterm neonates with respiratory distress syndrome. Main objectives of this study are to investigate the short term efficacy profile of CHF 5633 vs. porcine surfactant (Poractant Alfa, Curosurf®) in terms of reduced oxygen requirement and ventilatory support and to evaluate the mid-term efficacy profile in terms of reduced incidence of bronchopulmonary dysplasia (BPD) and mortality/BPD rate at 36 weeks post menstrual age (PMA), mortality rate at 28 days and 36 weeks PMA, RDS-associated mortality through 14 days of age and other major co-morbidities of prematurity.
Inclusion criteria are: Written parental informed consent, inborn preterm neonates of either sex with a gestational age of 24+0 weeks up to 29+6 weeks, clinical course consistent with RDS, requirement of endotracheal surfactant administration within 24 hours from birth, fraction of inspired oxygen (FiO2) ≥0.30 for babies 24+0 to 26+6 weeks and FiO2 ≥0.35 for babies 27+0 to 29+6 weeks to maintain arterial oxygen saturation by pulse oximetry (SpO2) between 88-95%.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CHF5633 | Experimental | Single dose within 24 hours from birth |
|
| Poractant alfa | Active Comparator | Single dose within 24 hours from birth |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CHF5633 | Drug | Rescue treatment (if needed) |
| |
| Poractant alfa |
| Measure | Description | Time Frame |
|---|---|---|
| Oxygen Requirement and Ventilatory Support -- SpO2/FiO2 Ratio | SpO2/FiO2 ratio The oxygen requirement and ventilatory support were assessed through arterial oxygen saturation, measured by pulse oximetry (SpO2 [%]) and ventilator settings, by measuring fraction of inspired oxygen (FiO2[%]) and SpO2/FiO2. Results are shown as change from baseline, summarized at post-treatment timepoints. Definitions: SpO2=Arterial Oxygen saturation by pulse oximetry; FiO2=Fraction of inspired oxygen; Baseline=The last pre-dose measurement taken on Day -1. | Post-treatment Day 1: 30 min, at 1h, 3h, 6h, 12h, 18h, 24 h; Day 2, 3, 5, and 7 |
| Fraction of Inspired Oxygen (FiO2) (Percent) During the First 24 h and up to Day 7 | Fraction of inspired oxygen (FiO2) (percent) during the first 24 h and up to Day 7. Fraction of inspired oxygen (FiO2 [percent]). Results are shown as change from baseline, summarized at post-treatment time points. Definitions: FiO2=Fraction of inspired oxygen (percent); Baseline=The last pre-dose measurement taken on Day -1; | Post-treatment Day 1: 30 min, at 1h, 3h, 6h, 12h, 18h, 24 h; Day 2, 3, 5, and 7 |
| Number of Patients With Bronchopulmonary Dysplasia and Mortality | Bronchopulmonary dysplasia and mortality. Results summarize the following items: Number of patients who died and the number of patients who had bronchopulmonary dysplasia (BPD) were assessed by treatment, at 36 weeks post menstrual age (PMA). Number of patients who died by Day 28 post-natal age (PNA). Number of patients with respiratory distress syndrome (RDS)-associated mortality by Day 14 post-natal age (PNA). Definitions: BPD=Bronchopulmonary dysplasia; Mortality/BPD incidence=The incidence of neonates dead within 36-week PMA or alive at 36-week PMA with a diagnosis of BPD; PMA=Post menstrual age; PNA=Post-natal age; RDS=Respiratory distress syndrome; | 36 weeks post menstrual age, Day 14 Post-Natal Age, Day 28 Post-Natal Age |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients With Normal Breathing (Room Air) Within 24 Hours | Normal breathing (room air) within 24 hours The number of patients with at least one reading of FiO2 equal to 21% (i.e. corresponding to room air for oxygen concentration) within 24 hours from first dose of surfactant was evaluated. | Post-treatment up to 24 h |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Rangasamy Ramanathan, MD | Division of Neonatology, Department of Pediatrics, LAC+USC Medical Center and Good Samaritan Hospital, Keck School of Medicine of USC, Los Angeles, CA, USA | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of South Alabama - USA Children's and Women's Hospital | Mobile | Alabama | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32553868 | Result | Ramanathan R, Biniwale M, Sekar K, Hanna N, Golombek S, Bhatia J, Naylor M, Fabbri L, Varoli G, Santoro D, Del Buono D, Piccinno A, Dammann CE. Synthetic Surfactant CHF5633 Compared with Poractant Alfa in the Treatment of Neonatal Respiratory Distress Syndrome: A Multicenter, Double-Blind, Randomized, Controlled Clinical Trial. J Pediatr. 2020 Oct;225:90-96.e1. doi: 10.1016/j.jpeds.2020.06.024. Epub 2020 Jun 14. |
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| ID | Title | Description |
|---|---|---|
| FG000 | CHF5633 | Single dose within 24 hours from birth CHF5633: Rescue treatment (if needed) |
| FG001 | Poractant Alfa | Single dose within 24 hours from birth Poractant alfa: Rescue treatment (if needed) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Jan 31, 2018 | Apr 9, 2021 |
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| Drug |
Rescue treatment (if needed) |
|
| Number of Patients With the Need for Re-dosing (Use of Rescue Surfactant) |
Number of patients with the need for re-dosing (use of rescue surfactant). The number of patients requiring at least one surfactant rescue dose (i.e. re-dosing with the study drug) at any time during the study was evaluated. |
| Day 1 to Day 7 |
| Time to Reach Normal Breathing (Room Air) Within 24 Hours | Time to reach normal breathing (room air) within 24 hours. The median time to reach normal breathing (room air) within 24 hours from first dose of surfactant was evaluated. These are the patients who contributed to the results in the outcome measure 'Normal Breathing (room air) within 24 hours'. | Post-treatment Day 1: up to 24 h |
| Concentration of Biomarkers of Inflammation in Tracheal Aspirates | The inflammatory status of the patients was assessed (in a subgroup of babies who required endotracheal intubation for mechanical ventilation, when feasible). This was performed by measuring the concentration of specific biomarkers of inflammation in tracheal aspirates. The biomarkers measured were: Interleukin 1β, Interleukin 6, Interleukin 8, Myeloperoxidase, and Tumor Necrosis Factor-Alpha. The total protein content in tracheal aspirates was measured as an endogenous marker of dilution to calculate the extent to which epithelial lining fluid (ELF) was diluted during the tracheal aspirate procedure. To adjust for variation during the collection of tracheal aspirates, the measured cytokines values were normalized to the total protein. Results are presented as change from baseline in pg/mg total protein and were evaluated by descriptive statistics. Definition: Baseline=The last pre-dose measurement taken on Day -1; | Post-treatment Day 1 (24 h), Day 2 (48 h) |
| Immunogenicity: Assessment of Antibodies to Surfactant Protein B (SP-B) Analogue (CHF 5736.03) and to Surfactant Protein C (SP-C) Analogue (CHF 4902.03) | Immunogenicity was assessed by measuring antibodies to SP-B analogue (CHF 5736.03) and to SP-C analogue (CHF 4902.03), contained in CHF 5633. Results are expressed as the titre (i.e. serum dilution) at which the sample had an absorbance of 0.069 (background) for SP-C Analogue (CHF 4902.03) CHF or an absorbance of 0.05 for SP-B Analogue (CHF 5736.03) in a microplate reader. The positive control serum was diluted in buffer solution and the maximum binding for the positive control was determined at dilutions < 1/12.5 for SPC and <1/100 for SPB. Test samples for immunogenicity were analyzed by using negative and positive controls. By definition, titer <12.5 for CHF-4902.03 and <100 for CHF-5736.02 show that the test serum had an absorbance equal to background at the same dilution at which the positive control had the maximum binding, implying absence of antibodies. | At approximately 5 weeks after the administration of study drug (with a range from 3 to 6 weeks). |
| LAC + USC Medical Center, Keck School of Medicine |
| Los Angeles |
| California |
| United States |
| UC Irvine Medical Center | Orange | California | United States |
| Sharp Mary Birch Hospital | San Diego | California | United States |
| Connecticut Children's Medical Center | Hartford | Connecticut | United States |
| Plantation General Hospital (Sheridan Clinical Research, Inc.) | Plantation | Florida | United States |
| Jatinder Bhatia | Augusta | Georgia | United States |
| Indiana University School of Medicine | Indianapolis | Indiana | United States |
| Memorial Hospital of South Bend | South Bend | Indiana | United States |
| University of Louisville Research Foundation, Inc. | Louisville | Kentucky | United States |
| Floating Hospital for Children at Tufts Medical Center | Boston | Massachusetts | 02111 | United States |
| Baystate Children's Hospital / Baystate Medical Center | Springfield | Massachusetts | United States |
| Winthrop University Hospital | Mineola | New York | United States |
| Kings County Hospital Center | New York | New York | United States |
| Sergio G. Golombek | Valhalla | New York | United States |
| Martha Naylor | Greenville | North Carolina | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | United States |
| Case Western Reserve University | Cleveland | Ohio | United States |
| Krishnamurthy Sekar | Oklahoma City | Oklahoma | United States |
| Hahnemann University Hospital | Philadelphia | Pennsylvania | United States |
| Texas Tech University Health Sciences Center | El Paso | Texas | United States |
| MultiCare Institute for Research & Innovation | Tacoma | Washington | United States |
| West Virginia University | Morgantown | West Virginia | United States |
| COMPLETED |
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| NOT COMPLETED |
|
|
Premature infants were screened according to study inclusion and exclusion criteria. Overall, 123 patients were randomized (6 patients were withdrawn before treatment start).
Overall, 117 patients received double-blind treatment. The baseline characteristics shown in the table below are based on the intention-to-treat (ITT) population (ITT=All randomized patients who received at least one dose of study medication and with at least one available evaluation of efficacy after the baseline).
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| ID | Title | Description |
|---|---|---|
| BG000 | CHF5633 | Single dose within 24 hours from birth. CHF5633: Rescue treatment (if needed) |
| BG001 | Poractant Alfa | Single dose within 24 hours from birth. Poractant alfa: Rescue treatment (if needed) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | hours |
| |||||||||||||||
| Age, Continuous | Gestational age. | Mean | Standard Deviation | weeks |
| ||||||||||||||
| Age, Customized | Gestational age group | Count of Participants | Participants |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||
| APGAR Score | APGAR Score at birth to summarize the health of a newborn.The Apgar score is based on a total score of 1 to 10. The higher the score, the better the baby is doing after birth. A score of 7, 8, or 9 is normal and is a sign that the newborn is in good health. | Mean | Standard Deviation | units on a scale |
| ||||||||||||||
| Birth weight | Mean | Standard Deviation | gram |
|
| 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 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Oxygen Requirement and Ventilatory Support -- SpO2/FiO2 Ratio | SpO2/FiO2 ratio The oxygen requirement and ventilatory support were assessed through arterial oxygen saturation, measured by pulse oximetry (SpO2 [%]) and ventilator settings, by measuring fraction of inspired oxygen (FiO2[%]) and SpO2/FiO2. Results are shown as change from baseline, summarized at post-treatment timepoints. Definitions: SpO2=Arterial Oxygen saturation by pulse oximetry; FiO2=Fraction of inspired oxygen; Baseline=The last pre-dose measurement taken on Day -1. | Intention-to-Treat Population (ITT). All randomized patients who received at least one dose of study medication and with at least one available evaluation of efficacy after the baseline. | Posted | Least Squares Mean | 95% Confidence Interval | SpO2/FiO2 ratio | Post-treatment Day 1: 30 min, at 1h, 3h, 6h, 12h, 18h, 24 h; Day 2, 3, 5, and 7 |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Fraction of Inspired Oxygen (FiO2) (Percent) During the First 24 h and up to Day 7 | Fraction of inspired oxygen (FiO2) (percent) during the first 24 h and up to Day 7. Fraction of inspired oxygen (FiO2 [percent]). Results are shown as change from baseline, summarized at post-treatment time points. Definitions: FiO2=Fraction of inspired oxygen (percent); Baseline=The last pre-dose measurement taken on Day -1; | Intention-to-Treat Population (ITT). All randomized patients who received at least one dose of study medication and with at least one available evaluation of efficacy after the baseline. | Posted | Least Squares Mean | 95% Confidence Interval | Fraction of inspired oxygen (percent) | Post-treatment Day 1: 30 min, at 1h, 3h, 6h, 12h, 18h, 24 h; Day 2, 3, 5, and 7 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Number of Patients With Bronchopulmonary Dysplasia and Mortality | Bronchopulmonary dysplasia and mortality. Results summarize the following items: Number of patients who died and the number of patients who had bronchopulmonary dysplasia (BPD) were assessed by treatment, at 36 weeks post menstrual age (PMA). Number of patients who died by Day 28 post-natal age (PNA). Number of patients with respiratory distress syndrome (RDS)-associated mortality by Day 14 post-natal age (PNA). Definitions: BPD=Bronchopulmonary dysplasia; Mortality/BPD incidence=The incidence of neonates dead within 36-week PMA or alive at 36-week PMA with a diagnosis of BPD; PMA=Post menstrual age; PNA=Post-natal age; RDS=Respiratory distress syndrome; | Intention-to-Treat Population (ITT). All randomized patients who received at least one dose of study medication and with at least one available evaluation of efficacy after the baseline. | Posted | Count of Participants | Participants | 36 weeks post menstrual age, Day 14 Post-Natal Age, Day 28 Post-Natal Age |
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| Other Pre-specified | Number of Patients With Normal Breathing (Room Air) Within 24 Hours | Normal breathing (room air) within 24 hours The number of patients with at least one reading of FiO2 equal to 21% (i.e. corresponding to room air for oxygen concentration) within 24 hours from first dose of surfactant was evaluated. | Intention-to-Treat Population (ITT). All randomized patients who received at least one dose of study medication and with at least one available evaluation of efficacy after the baseline. | Posted | Count of Participants | Participants | Post-treatment up to 24 h |
|
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| Other Pre-specified | Number of Patients With the Need for Re-dosing (Use of Rescue Surfactant) | Number of patients with the need for re-dosing (use of rescue surfactant). The number of patients requiring at least one surfactant rescue dose (i.e. re-dosing with the study drug) at any time during the study was evaluated. | Intention-to-Treat Population (ITT). All randomized patients who received at least one dose of study medication and with at least one available evaluation of efficacy after the baseline. | Posted | Count of Participants | Participants | Day 1 to Day 7 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Time to Reach Normal Breathing (Room Air) Within 24 Hours | Time to reach normal breathing (room air) within 24 hours. The median time to reach normal breathing (room air) within 24 hours from first dose of surfactant was evaluated. These are the patients who contributed to the results in the outcome measure 'Normal Breathing (room air) within 24 hours'. | Intention-to-Treat Population (ITT). All randomized patients who received at least one dose of study medication and with at least one available evaluation of efficacy after the baseline. | Posted | Median | Full Range | hour | Post-treatment Day 1: up to 24 h |
|
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| Other Pre-specified | Concentration of Biomarkers of Inflammation in Tracheal Aspirates | The inflammatory status of the patients was assessed (in a subgroup of babies who required endotracheal intubation for mechanical ventilation, when feasible). This was performed by measuring the concentration of specific biomarkers of inflammation in tracheal aspirates. The biomarkers measured were: Interleukin 1β, Interleukin 6, Interleukin 8, Myeloperoxidase, and Tumor Necrosis Factor-Alpha. The total protein content in tracheal aspirates was measured as an endogenous marker of dilution to calculate the extent to which epithelial lining fluid (ELF) was diluted during the tracheal aspirate procedure. To adjust for variation during the collection of tracheal aspirates, the measured cytokines values were normalized to the total protein. Results are presented as change from baseline in pg/mg total protein and were evaluated by descriptive statistics. Definition: Baseline=The last pre-dose measurement taken on Day -1; | Intention-to-Treat Population (ITT). All randomized patients who received at least one dose of study medication and with at least one available evaluation of efficacy after the baseline. | Posted | Mean | Standard Deviation | pg/mg total protein | Post-treatment Day 1 (24 h), Day 2 (48 h) |
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| Other Pre-specified | Immunogenicity: Assessment of Antibodies to Surfactant Protein B (SP-B) Analogue (CHF 5736.03) and to Surfactant Protein C (SP-C) Analogue (CHF 4902.03) | Immunogenicity was assessed by measuring antibodies to SP-B analogue (CHF 5736.03) and to SP-C analogue (CHF 4902.03), contained in CHF 5633. Results are expressed as the titre (i.e. serum dilution) at which the sample had an absorbance of 0.069 (background) for SP-C Analogue (CHF 4902.03) CHF or an absorbance of 0.05 for SP-B Analogue (CHF 5736.03) in a microplate reader. The positive control serum was diluted in buffer solution and the maximum binding for the positive control was determined at dilutions < 1/12.5 for SPC and <1/100 for SPB. Test samples for immunogenicity were analyzed by using negative and positive controls. By definition, titer <12.5 for CHF-4902.03 and <100 for CHF-5736.02 show that the test serum had an absorbance equal to background at the same dilution at which the positive control had the maximum binding, implying absence of antibodies. | Safety Population. All randomized patients who took at least one dose of study medication and who had the assessment done on 28 day PNA. | Posted | Number | Titre (dilution with 0.050 absorbance) | At approximately 5 weeks after the administration of study drug (with a range from 3 to 6 weeks). |
|
Adverse events were reported from the time of the informed consent signature* up to the study completion or discontinuation. The study duration was from minutes after birth and up to 36 week post menstrual age (PMA) *The written informed consent was obtained from parents/legal representative (according to local regulation) prior to any study-related procedures.
Analysis of adverse events (AEs) was performed on treatment emergent AEs (TEAEs).
Safety population=All randomized subjects who received at least one dose of study treatment, was used for the evaluation of adverse events.
An Independent Safety Monitoring Board (ISMB) was established on an ongoing basis to ensure the safety of study patients, with regard to the incidence of major adverse outcomes after the administration of the study drug (such as serious adverse events [SAEs]).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | CHF5633 | Single dose within 24 hours from birth CHF5633: Rescue treatment (if needed) | 4 | 59 | 20 | 59 | 59 | 59 |
| EG001 | Poractant Alfa | Single dose within 24 hours from birth Poractant alfa: Rescue treatment (if needed) | 7 | 58 | 20 | 58 | 58 | 58 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Disseminated intravascular coagulation in newborn | Blood and lymphatic system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Bradycardia neonatal | Cardiac disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Amniotic band syndrome | Congenital, familial and genetic disorders | MedDRA version 18.0. | Systematic Assessment |
| |
| Cerebellar hypoplasia | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Congenital cytomegalovirus infection | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Developmental glaucoma | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Patent ductus arteriosus | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Retinopathy of prematurity | Eye disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal intestinal perforation | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Necrotising enterocolitis neonatal | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Pneumoperitoneum | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal pneumonia | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Serratia bacteraemia | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Sepsis neonatal | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Urinary tract infection neonatal | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Feeding disorder neonatal | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Intraventricular haemorrhage neonatal | Nervous system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Cerebral haemorrhage neonatal | Nervous system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Cerebellar haemorrhage | Nervous system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Oliguria | Renal and urinary disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Renal failure neonatal | Renal and urinary disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Bronchopulmonary dysplasia | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Atelectasis neonatal | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Tracheal stenosis | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal hypotension | Vascular disorders | MedDRA version 18.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia neonatal | Blood and lymphatic system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Bandaemia | Blood and lymphatic system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neutropenia neonatal | Blood and lymphatic system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Thrombocytopenia neonatal | Blood and lymphatic system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Bradycardia neonatal | Cardiac disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal tachycardia | Cardiac disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Tricuspid valve incompetence | Cardiac disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Adrenal insufficiency neonatal | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Atrial septal defect | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hydrocele | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Patent ductus arteriosus | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Sickle cell trait | Congenital, familial and genetic disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Transient hypothyroxinaemia of prematurity | Endocrine disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Retinopathy of prematurity | Eye disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Gastrointestinal hypomotility | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Gastrointestinal motility disorder | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Peripheral oedema neonatal | General disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hyperbilirubinaemia neonatal | Hepatobiliary disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Jaundice cholestatic | Hepatobiliary disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Bacterial disease carrier | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal pneumonia | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Sepsis neonatal | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Tracheitis | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Urinary tract infection neonatal | Infections and infestations | MedDRA version 18.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA version 18.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA version 18.0 | Systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA version 18.0 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA version 18.0 | Systematic Assessment |
| |
| Chest X-ray abnormal | Investigations | MedDRA version 18.0 | Systematic Assessment |
| |
| Alkalosis hypochloraemic | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Feeding disorder neonatal | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Feeding disorder of infancy or early childhood | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypermagnesaemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypoglycaemia neonatal | Investigations | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Metabolic alkalosis | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal hyponatraemia | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Poor feeding infant | Metabolism and nutrition disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Haemangioma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 18.0 | Systematic Assessment |
| |
| Cerebral ventricle dilatation | Nervous system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Convulsion neonatal | Nervous system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Intraventricular haemorrhage neonatal | Nervous system disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Jaundice neonatal | Pregnancy, puerperium and perinatal conditions | MedDRA version 18.0 | Systematic Assessment |
| |
| Leukopenia neonatal | Pregnancy, puerperium and perinatal conditions | MedDRA version 18.0 | Systematic Assessment |
| |
| Agitation neonatal | Psychiatric disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Apnoea neonatal | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Atelectasis neonatal | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Bronchopulmonary dysplasia | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypercapnia | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal respiratory acidosis | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Pulmonary interstitial emphysema syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Pulmonary oedema neonatal | Respiratory, thoracic and mediastinal disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Hypertension neonatal | Vascular disorders | MedDRA version 18.0 | Systematic Assessment |
| |
| Neonatal hypotension | Vascular disorders | MedDRA version 18.0 | Systematic Assessment |
|
Chiesi Farmaceutici S.p.A. (Sponsor) can publish or present results of this study at scientific meetings and submit the data to national and international Regulatory Authorities. The Sponsor reserves the right to use the data for industry purposes. Investigators will inform the Sponsor before using the results of the study for publication or presentation, and will provide the Sponsor with a copy of the proposed presentation. Data from individual study sites must not be published separately.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Transparency | Chiesi Farmaceutici S.p.A. | +39 0521 2791 | clinicaltrials_info@chiesi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 18, 2018 | Apr 9, 2021 | SAP_001.pdf |
| ID | Term |
|---|---|
| D012127 | Respiratory Distress Syndrome, Newborn |
| ID | Term |
|---|---|
| D012128 | Respiratory Distress Syndrome |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D012120 | Respiration Disorders |
| D007235 | Infant, Premature, Diseases |
| D007232 | Infant, Newborn, Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C000592408 | CHF5633 |
| C068291 | poractant alfa |
Not provided
Not provided
Not provided
| 27 to 29 Weeks |
|
| 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 |
|
| Day 1, 1 h |
|
|
| Day 1, 3 h |
|
|
| Day 1, 6 h |
|
|
| Day 1, 12 h |
|
|
| Day 1, 18 h |
|
|
| Day 1, 24 h |
|
|
| Day 2 |
|
|
| Day 3 |
|
|
| Day 5 |
|
|
| Day 7 |
|
|
| Day 1, 1 h post dose | Mixed model for repeated measurements | 0.346 | Mean Difference (Final Values) | -0.19 | 2-Sided | 95 | -0.59 | 0.21 | Superiority |
| Day 1, 3 h post dose | Mixed model for repeated measurements | 0.549 | Mean Difference (Final Values) | -0.10 | 2-Sided | 95 | -0.43 | 0.23 | Superiority |
| Day 1, 6 h post dose | Mixed model for repeated measurements | 0.539 | Mean Difference (Final Values) | -0.10 | 2-Sided | 95 | -0.43 | 0.23 | Superiority |
| Day 1, 12 h post dose | Mixed model for repeated measurements | 0.491 | Mean Difference (Final Values) | 0.11 | 2-Sided | 95 | -0.21 | 0.43 | Superiority |
| Day 1, 18 h post dose | Mixed model for repeated measurements | 0.623 | Mean Difference (Final Values) | 0.07 | 2-Sided | 95 | -0.22 | 0.37 | Superiority |
| Day 1, 24 h post dose | Mixed model for repeated measurements | 0.608 | Mean Difference (Final Values) | 0.09 | 2-Sided | 95 | -0.25 | 0.43 | Superiority |
| Day 2, post dose SpO2/FiO2 was compared between treatments at the remaining post-treatment time points (i.e., Days 2, 3, 5, 7): analyzed using mixed model including treatment, investigational site and gestational age group as fixed effects and pre-dose ratio as covariate. | Mixed Models Analysis | 0.869 | Mean Difference (Final Values) | -0.03 | 2-Sided | 95 | -0.35 | 0.30 | Superiority |
| Day 3, post dose | Mixed Models Analysis | 0.833 | Mean Difference (Final Values) | -0.04 | 2-Sided | 95 | -0.38 | 0.31 | Superiority |
| Day 5, post dose | Mixed Models Analysis | 0.772 | Mean Difference (Final Values) | -0.05 | 2-Sided | 95 | -0.39 | 0.29 | Superiority |
| Day 7, post dose | Mixed Models Analysis | 0.963 | Mean Difference (Final Values) | 0.01 | 2-Sided | 95 | -0.33 | 0.35 | Superiority |
|
|
| Units | Counts |
|---|
| Participants |
|
|
|
|
|
|
|
|
|
|
|
Single dose within 24 hours from birth
Poractant alfa: Rescue treatment (if needed)
|
|