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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-005449-35 | EudraCT Number |
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Based on the results of study PTC124-GD-021-CF (NCT02139306), clinical development of ataluren in cystic fibrosis was discontinued and this study was closed.
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The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ataluren | Experimental | Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ataluren | Drug | Ataluren will be administered per dose and schedule specified in the arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. | Baseline (Day 1) up to end of study (Week 196) |
| Number of Participants With Clinically Significant Laboratory Abnormalities | Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement. | Baseline (Day 1) up to end of study (Week 196) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Percent of predicted FEV1 = (observed value)/(predicted value) * 100%. Change from baseline in percent predicted FEV1 at the end of treatment was reported. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at the End of Treatment (Week 192), as Assessed by Spirometry | FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position. Percent of predicted FVC = (observed value)/(predicted value) * 100%. Change from baseline in percent predicted FVC at the end of treatment was reported. | Baseline, Week 192 |
Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Joseph McIntosh, MD | PTC Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama-Birmingham | Birmingham | Alabama | 35233 | United States | ||
| Miller Children's Hospital Long Beach |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36866921 | Derived | Aslam AA, Sinha IP, Southern KW. Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis. Cochrane Database Syst Rev. 2023 Mar 3;3(3):CD012040. doi: 10.1002/14651858.CD012040.pub3. |
| Label | URL |
|---|---|
| PTC Therapeutics, Inc. website | View source |
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On 2 March 2017, it was announced that the Phase 3 double-blind study PTC124-GD-021-CF (NCT02139306) did not achieve its primary or secondary endpoints. Based on these results, clinical development of ataluren in cystic fibrosis was discontinued and this ongoing open-label extension study was closed.
Participants with nonsense mutation cystic fibrosis (nmCF) who had completed the double-blind study PTC124-GD-009-CF (NCT00803205) were enrolled and treated in this open-label extension study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Ataluren | Participants received ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
As-treated population included all participants who received at least 1 dose of ataluren.
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| ID | Title | Description |
|---|---|---|
| BG000 | Ataluren | Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. | As-treated population included all participants who received at least 1 dose of ataluren. | Posted | Count of Participants | Participants | Baseline (Day 1) up to end of study (Week 196) |
Baseline up to end of study (Week 196)
As-treated population included all participants who received at least 1 dose of ataluren.
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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 | Ataluren | Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Appendicitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastroenteritis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
Based on the results of study PTC124-GD-021-CF (NCT02139306) (did not achieve its primary or secondary endpoints), clinical development of ataluren in cystic fibrosis was discontinued and this ongoing open-label extension study was closed.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Patient Advocacy | PTC Therapeutics, Inc. | 1-866-562-4620 | medinfo@ptcbio.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 14, 2017 | Mar 11, 2020 | SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Feb 8, 2016 | Mar 11, 2020 | Prot_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| C515878 | ataluren |
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| Baseline, Week 192 |
| Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria | The modified Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms without the requirement for treatment with antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature greater than (>) 38 degrees celsius (°C); anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. | Baseline up to Week 192 |
| Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria | The expanded Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring any form of antibiotic treatment (inhaled, oral, or intravenous): change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. | Baseline up to Week 192 |
| Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria | The Classic Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring treatment with parenteral antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. | Baseline up to Week 192 |
| Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196) | ECG parameters included RR duration, PR duration, QRS duration, QT duration, QTCB (Bazett's correction formula) duration, QTCF (Fridericia's correction formula) duration. | Baseline, Week 196 |
| Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG | Heart rate was measured using 12-lead ECG. | Baseline, Week 196 |
| Change From Baseline in Vital Signs at Final Visit (Week 196) | Vital Signs included systolic blood pressure (SBP) and diastolic blood pressure (DBP). | Baseline, Week 196 |
| Change From Baseline in Percent Predicted Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) at the End of Treatment (Week 192), as Assessed by Spirometry | FEF25-75 is the forced expiratory flow between 25 and 75% of vital capacity. | Baseline, Week 192 |
| Long Beach |
| California |
| 90806 |
| United States |
| Denver Children's Hospital | Aurora | Colorado | 80045 | United States |
| Children's Hospital Chicago | Chicago | Illinois | 60614 | United States |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | United States |
| Beth Israel Medical Center | New York | New York | 10003 | United States |
| Rainbow Babies & Children's Hospital | Cleveland | Ohio | 44106 | United States |
| Hôpital Universitaire des Enfants Reine Fabiola | Brussels | Belgium |
| University Hospital Brussels | Brussels | Belgium |
| University Hospital Leuven | Leuven | Belgium |
| Hôpital Necker - Enfants Malades | Paris | France |
| Hôpital des Enfants | Toulouse | 31059 | France |
| Hadassah University Hospital - Mount Scopus | Jerusalem | 91240 | Israel |
| Università La Sapienza | Roma | Italy |
| Azienda Ospedaliera di Verona | Verona | Italy |
| Hospital Universitario La Paz | Madrid | Spain |
| Karolinska University Hospital, Huddinge | Stockholm | Sweden |
| Other than specified |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | Ataluren | Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks. |
|
|
| Primary | Number of Participants With Clinically Significant Laboratory Abnormalities | Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement. | As-treated population included all participants who received at least 1 dose of ataluren. | Posted | Count of Participants | Participants | Baseline (Day 1) up to end of study (Week 196) |
|
|
|
| Secondary | Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Percent of predicted FEV1 = (observed value)/(predicted value) * 100%. Change from baseline in percent predicted FEV1 at the end of treatment was reported. | ITT population included all participants who had at least 1 post-baseline efficacy assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | percentage of predicted FEV1 | Baseline, Week 192 |
|
|
|
| Secondary | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria | The modified Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms without the requirement for treatment with antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature greater than (>) 38 degrees celsius (°C); anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. | ITT population included all participants who had at least 1 post-baseline efficacy assessment. | Posted | Number | percentage of participants | Baseline up to Week 192 |
|
|
|
| Secondary | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria | The expanded Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring any form of antibiotic treatment (inhaled, oral, or intravenous): change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. | ITT population included all participants who had at least 1 post-baseline efficacy assessment. | Posted | Number | percentage of participants | Baseline up to Week 192 |
|
|
|
| Secondary | Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria | The Classic Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring treatment with parenteral antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature >38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. | ITT population included all participants who had at least 1 post-baseline efficacy assessment. | Posted | Number | percentage of participants | Baseline up to Week 192 |
|
|
|
| Secondary | Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196) | ECG parameters included RR duration, PR duration, QRS duration, QT duration, QTCB (Bazett's correction formula) duration, QTCF (Fridericia's correction formula) duration. | As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | miiliseconds | Baseline, Week 196 |
|
|
|
| Secondary | Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG | Heart rate was measured using 12-lead ECG. | As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | beats/minute | Baseline, Week 196 |
|
|
|
| Secondary | Change From Baseline in Vital Signs at Final Visit (Week 196) | Vital Signs included systolic blood pressure (SBP) and diastolic blood pressure (DBP). | As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Number analyzed' signifies participants evaluable for specified categories. | Posted | Mean | Standard Deviation | millimeters of mercury (mmHg) | Baseline, Week 196 |
|
|
|
| Other Pre-specified | Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at the End of Treatment (Week 192), as Assessed by Spirometry | FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position. Percent of predicted FVC = (observed value)/(predicted value) * 100%. Change from baseline in percent predicted FVC at the end of treatment was reported. | ITT population included all participants who had at least 1 post-baseline efficacy assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | percentage of predicted FVC | Baseline, Week 192 |
|
|
|
| Other Pre-specified | Change From Baseline in Percent Predicted Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) at the End of Treatment (Week 192), as Assessed by Spirometry | FEF25-75 is the forced expiratory flow between 25 and 75% of vital capacity. | Due to change in planned analysis FEV25-75 was not calculated and summarized. | Posted | Baseline, Week 192 |
|
|
| 1 |
| 61 |
| 36 |
| 61 |
| 61 |
| 61 |
| Cellulitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Clostridium difficile infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Device related infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Distal ileal obstruction syndrome | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Blood creatine increased | Investigations | MedDRA 17.0 | Systematic Assessment |
|
| Forced expiratory volume decreased | Investigations | MedDRA 17.0 | Systematic Assessment |
|
| Pulmonary function test decreased | Investigations | MedDRA 17.0 | Systematic Assessment |
|
| Renal failure | Renal and urinary disorders | MedDRA 17.0 | Systematic Assessment |
|
| Renal failure acute | Renal and urinary disorders | MedDRA 17.0 | Systematic Assessment |
|
| Cystic fibrosis | Congenital, familial and genetic disorders | MedDRA 17.0 | Systematic Assessment |
|
| Gallbladder pain | Hepatobiliary disorders | MedDRA 17.0 | Systematic Assessment |
|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 17.0 | Systematic Assessment |
|
| Colon cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 17.0 | Systematic Assessment |
|
| Deep vein thrombosis | Vascular disorders | MedDRA 17.0 | Systematic Assessment |
|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Oral candidiasis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 17.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Haemorrhoids | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 17.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 17.0 | Systematic Assessment |
|
| Drug hypersensitivity | Immune system disorders | MedDRA 17.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 17.0 | Systematic Assessment |
|
The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
|
|
| Baseline: PR duration |
|
|
| Change at Week 196: PR duration |
|
|
| Baseline: QRS duration |
|
|
| Change at Week 196: QRS duration |
|
|
| Baseline: QT duration |
|
|
| Change at Week 196: QT duration |
|
|
| Baseline: QTCB duration |
|
|
| Change at Week 196: QTCB duration |
|
|
| Baseline: QTCF duration |
|
|
| Change at Week 196: QTCF duration |
|
|
|
|
| Baseline: DBP |
|
|
| Change at Week 196: DBP |
|
|
|