Phase 3
N=360
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Muscular Dystrophy, Duchenne · Muscular Dystrophies · Muscular Disorders, Atrophic · Muscular Diseases · Musculoskeletal Disease
Bottom Line
View on ClinicalTrials.gov: NCT03179631 ↗Enrolled (actual)
360
Serious AEs
8.1%
Results posted
Mar 2026
Primary outcome: Primary: DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population — -81.83; -90.09 meters — p== 0.3626
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Ataluren (Drug); PLACEBO (Drug)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- Male
- Sponsor
- PTC Therapeutics
- Primary completion
- Mar 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population |
-81.83; -90.09 | = 0.3626 |
| PRIMARY DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population |
-53.01; -67.43 | = 0.0248 sig |
| PRIMARY DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population |
-1.14; -1.25 | = 0.3626 |
| PRIMARY DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population |
-0.74; -0.94 | = 0.0248 sig |
| SECONDARY DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - mITT Population |
3.06; 3.79 | = 0.1877 |
| SECONDARY DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - ITT Population |
3.04; 3.82 | = 0.0422 sig |
| SECONDARY DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - mITT Population |
5.25; 6.98 | = 0.0179 sig |
| SECONDARY DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - ITT Population |
4.98; 6.04 | = 0.0293 sig |
| SECONDARY DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - mITT Population |
4.58; 4.78 | = 0.7997 |
| SECONDARY DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - ITT Population |
4.96; 5.25 | = 0.5749 |
| SECONDARY DB Period: Composite of Average Change From Baseline in TFTs at Week 72 - mITT Population |
4.15; 5.19 | = 0.0892 |
| SECONDARY DB Period: Composite of Average Change From Baseline in TFTs at Week 72 - ITT Population |
4.24; 4.93 | = 0.0904 |
| SECONDARY DB Period: Composite of Average Rate of Change From Baseline in TFTs at Week 72 - mITT Population |
0.058; 0.072 | = 0.0892 |
| SECONDARY DB Period: Composite of Average Rate of Change From Baseline in TFTs at Week 72 - ITT Population |
0.059; 0.069 | = 0.0904 |
| SECONDARY DB Period: Time to Persistent 10% Worsening in 6MWD at Week 72 - mITT Population |
36.7; 35.6 | = 0.0659 |
| SECONDARY DB Period: Time to Persistent 10% Worsening in 6MWD - ITT Population |
74.3; 48.0 | = 0.0078 sig |
| SECONDARY DB Period: Change From Baseline in North Start Ambulatory Assessment (NSAA) Total Score at Week 72 - mITT Population |
-5.2; -6.1 | = 0.1258 |
| SECONDARY DB Period: Change From Baseline in NSAA Total Score at Week 72 - ITT Population |
-3.7; -4.5 | = 0.0235 sig |
| SECONDARY DB Period: Time to Loss of Ambulation - mITT Population |
84.1; NA | = 0.4803 |
| SECONDARY DB Period: Time to Loss of Ambulation Over 72 Weeks - ITT Population |
NA; NA | = 0.1768 |
| SECONDARY DB Period: Time to Loss of Stair-Climbing - mITT Population |
84.1; NA | = 0.5452 |
| SECONDARY DB Period: Time to Loss of Stair-Climbing - ITT Population |
87.1; NA | = 0.3055 |
| SECONDARY DB Period: Time to Loss of Stair-Descending - mITT Population |
84.1; NA | = 0.3906 |
| SECONDARY DB Period: Time to Loss of Stair-Descending - ITT Population |
87.1; NA | = 0.8165 |
| SECONDARY DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - mITT Population |
6; 10; 5; 10; 22; 23 | — |
| SECONDARY DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - ITT Population |
14; 21; 14; 21; 36; 35 | — |
| SECONDARY DB Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
157; 149 | — |
| SECONDARY Overall Treatment Period: Change From Baseline in 6MWD at Week 144 |
-130.49; -140.00 | — |
| SECONDARY Overall Treatment Period: Composite of Average Change From Baseline in TFTs at Week 144 |
9.00; 9.29 | — |
| SECONDARY Overall Treatment Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 144 |
7.99; 8.16 | — |
| SECONDARY Overall Treatment Period: Change From Baseline in Time to Climb 4 Stairs at Week 144 |
9.89; 10.18 | — |
| SECONDARY Overall Treatment Period: Change From Baseline in Time to Descend 4 Stairs at Week 144 |
9.56; 10.15 | — |
| SECONDARY Overall Treatment Period: Change From Baseline in NSAA Total Score at Week 144 |
-7.0; -7.2 | — |
| SECONDARY Overall Treatment Period: Time to Loss of Ambulation Over 144 Weeks |
NA; NA | — |
| SECONDARY Overall Treatment Period: Time to Loss of Stair-Climbing Over 144 Weeks |
NA; NA | — |
| SECONDARY Overall Treatment Period: Time to Loss of Stair- Descending Over 144 Weeks |
NA; NA | — |
| SECONDARY Overall Treatment Period: Number of Participants With Function Loss of NSAA Items at Week 144 |
49; 49; 49; 48; 64; 73 | — |
| SECONDARY Overall Treatment Period: Number of Participants With TEAEs |
170; 110 | — |
| SECONDARY OL Period: Plasma Pharmacokinetic (PK) Concentration of Ataluren |
4.45; 4.11; 12.16; 12.21 | — |
Summary
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
Eligibility Criteria
Inclusion Criteria
- Male sex
- Age ≥5 years
- Phenotypic evidence of Duchenne Muscular Dystrophy
- Nonsense point mutation in the dystrophin gene
- Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
- 6MWD ≥150 meters
- Ability to perform timed function tests within 30 seconds
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion Criteria
- Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.
- Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
- Prior or ongoing therapy with ataluren.
- Known hypersensitivity to any of the ingredients or excipients of the study drug
- Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial.
- History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period.
- Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy.
- Uncontrolled clinical symptoms and signs of congestive heart failure
- Elevated serum creatinine or cystatin C at screening.
Data sourced from ClinicalTrials.gov (NCT03179631). 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.