Phase 3
N=191
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT01140451 ↗Enrolled (actual)
191
Serious AEs
55.0%
Results posted
Oct 2020
Primary outcome: Primary: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) — 100; 97.9; 14.7; 18.8 percent of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Ataluren (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- PTC Therapeutics
- Primary completion
- Dec 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) |
100; 97.9; 14.7; 18.8; 62.1; 51.0 | — |
| PRIMARY Number of Participants With Any Treatment-Emergent Laboratory Abnormality (TELA) |
13; 18; 39; 47; 44; 49 | — |
| SECONDARY Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in 1 Second (FEV1) at Baseline |
60.61; 56.49 | — |
| SECONDARY Percentage Change From Baseline in Percent-Predicted of FEV1 at Weeks 48 and 96 |
-0.73; 1.09; -3.09; -2.12 | — |
| SECONDARY Percent-Predicted of Forced Vital Capacity (FVC) at Baseline |
76.37; 73.26 | — |
| SECONDARY Percentage Change From Baseline in Percent-Predicted of FVC at Weeks 48 and 96 |
0.05; 0.69; -1.48; -1.17 | — |
| SECONDARY Percent-Predicted of Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) at Baseline |
38.16; 33.11 | — |
| SECONDARY Percentage Change From Baseline in Percent-Predicted of FEF25-75 at Weeks 48 and 96 |
-0.55; 7.89; -5.55; -3.29 | — |
| SECONDARY Number of Participants With Pulmonary Exacerbations as Defined by Modified Fuch's Criteria |
50; 56; 57; 68 | — |
| SECONDARY Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks |
1.150; 1.614 | — |
| SECONDARY Duration of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria |
3.675; 4.734; 3.567; 3.912 | — |
| SECONDARY Number of Participants With Severe Pulmonary Exacerbations as Defined by Modified Fuch's Criteria |
1; 1; 0; 1 | — |
| SECONDARY Change From Baseline for the Respiratory Domain Score of the Cystic Fibrosis (CF) Questionnaire-Revised (CFQ-R) at Weeks 48 and 96 |
83.333; 86.111; -13.095; -0; -5.556; 0 | — |
| SECONDARY Rate of Study Drug Compliance |
86.057; 80.118; 81.50; 78.59 | — |
| SECONDARY Predose Concentration of Ataluren |
1.668; 0; 5.744; 7.552; 6.298; 7.694 | — |
| SECONDARY Number of Participants Who Required Interventions for Pulmonary Symptoms |
44; 49; 10; 18; 80; 80 | — |
| SECONDARY Number of Participants With Disruptions in Activities of Daily Living Because of Pulmonary Symptoms |
23; 25; 27; 25 | — |
| SECONDARY Duration of Disruptions in Activities of Daily Living Because of Pulmonary Symptoms |
25.0; 21.5; 25.0; 22.0 | — |
| SECONDARY Change From Baseline in Body Weight at Weeks 48 and 96 |
53.354; 57.444; 1.232; 0.540; 2.149; 0.830 | — |
| SECONDARY Change From Baseline in Body Mass Index (BMI) at Weeks 48 and 96 |
20.023; 21.044; 0.179; 0.102; 0.144; 0.105 | — |
| SECONDARY Total Lung Computed Tomography (CT) Score at Weeks 48 and 96 |
— | — |
| SECONDARY Change From Baseline in the Nasal Transepithelial Potential Difference (TEPD) at Week 48 |
— | — |
| SECONDARY Change From Baseline in the Concentration of Sweat Chloride at Week 48 |
100.26; 97.88; 5.34; 3.60 | — |
Summary
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of patients with the disease. Ataluren is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 extension trial that will evaluate the long-term safety of ataluren in adult and pediatric participants with nonsense mutation CF (nmCF), as determined by adverse events and laboratory abnormalities. The study will also assess changes in pulmonary function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology. Funding source for this study is the FDA OOPD.
Eligibility Criteria
Inclusion Criteria
- Completion of blinded study drug treatment in the previous Phase 3 study (PTC124-GD-009-CF).
- Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if <18 years of age).
- In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 4-week follow up period.
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion Criteria
- Known hypersensitivity to any of the ingredients or excipients of the study drug (list provided at study sites).
- Current pregnancy or lactating, or pregnancy or lactating during the previous Phase 3 study.
- Ongoing participation in any other therapeutic clinical trial.
- Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results.
Data sourced from ClinicalTrials.gov (NCT01140451). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.