Phase 3
N=94
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne Muscular Dystrophy · Becker Muscular Dystrophy · Dystrophinopathy
Bottom Line
View on ClinicalTrials.gov: NCT01557400 ↗Enrolled (actual)
94
Serious AEs
33.0%
Results posted
Nov 2020
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 91; 31; 26 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Ataluren (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- Male
- Sponsor
- PTC Therapeutics
- Primary completion
- Jan 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) |
91; 31; 26 | — |
| SECONDARY Change From Baseline in 6MWD as Measured by the 6MWT |
-41.71; -76.80; -97.57; -109.37; -134.16 | — |
| SECONDARY Change From Baseline in Physical Function as Measured by the NSAA |
-2.9; -7.4; -8.8; -13.4 | — |
| SECONDARY Change From Baseline in Time to Stand From Supine Position |
3.33; 6.38; 6.11; 3.84; 11.16 | — |
| SECONDARY Change From Baseline in Time to Walk/Run 10 Meters |
1.67; 3.48; 3.07; 3.19; 3.62 | — |
| SECONDARY Change From Baseline in Pulmonary Function as Measured by Spirometry |
-0.00; -0.06; -0.18; -0.18; -0.24; -7.98 | — |
| SECONDARY Change From Baseline in Participant and Parent/Caregiver-Reported ADL, as Measured by the EK Scale |
2.0; 2.6; 3.3; 5.3; 7.0 | — |
Summary
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of DBMD in approximately 10-15% of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study comprises a Phase 3, open-label study of ataluren in participants with nmDBMD who previously received ataluren at an Investigator site in a prior PTC-sponsored clinical study. A separate open-label study (PTC124-GD-016-DMD; NCT01247207) is being conducted for nmDBMD participants who previously received ataluren at an Investigator site in the United States (US).
Eligibility Criteria
Inclusion Criteria
- Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethics Committee (IRB/IEC) regarding whether 1 or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
- History of exposure to ataluren in a prior PTC study in nmDBMD. Note: Participants are considered eligible only if they received ataluren during their participation in 1 or more prior PTC-sponsored studies of ataluren in nmDBMD. Note: Participants who have participated in a prior or ongoing PTC study with ataluren in nmDBMD at a trial site in the US or Canada, but reside outside of the US and Canada, may be eligible for this study (with the approval of the PTC Therapeutics Medical Monitor).
- Male sex.
- 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 6-week follow-up period.
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions. Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered.
Exclusion Criteria
- Exposure to another investigational drug within 1 month prior to start of study treatment.
- Eligibility for another ataluren clinical trial that is actively enrolling study participants.
- Known hypersensitivity to any of the ingredients or excipients of ataluren (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P [colloidal silica], magnesium stearate).
- Ongoing use of the following medications:
- Coumarin-based anticoagulants (for example, warfarin), phenytoin, tolbutamide, or paclitaxel.
- Systemic aminoglycoside therapy
- Ongoing uncontrolled medical/surgical condition, electrocardiogram (ECG) findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant or make it unlikely that follow-up would be completed.
Data sourced from ClinicalTrials.gov (NCT01557400). 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.