An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02090959
• Date of registration: 17/03/2014
• Prospective Registration: Yes
• Primary sponsor: PTC Therapeutics
• Public title: An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
• Scientific title: A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
• Date of first enrolment: March 20, 2014
• Target sample size: 219
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02090959
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Brazil, Bulgaria, Canada, Chile, Czech Republic, Czechia, France, Germany, Israel, Italy, Korea, Republic of, Poland, Spain, Sweden, Switzerland, Turkey, United Kingdom, United States

Contacts

• Name: Francesco Bibbiani, M.D.
• Affiliation: PTC Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Completion of study treatment in the previous Phase 3, double-blind study
(PTC124-GD-020-DMD).

- 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 Ethic 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.

- In participants who are sexually active, willingness to abstain from sexual
intercourse or employ a barrier or medical method of contraception during the period
of study drug administration and 6-week follow-up period.

- Willingness and ability to comply with scheduled visits, ataluren administration plan,
study procedures, laboratory tests, and study restrictions.

Exclusion Criteria:

- Known hypersensitivity to any of the ingredients or excipients of the study drug
(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], and magnesium stearate).

- 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, electrocardiogram (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.



Age minimum: 7 Years
Age maximum: 15 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Genetic Diseases, X-Linked

Muscular Dystrophies

Muscular Dystrophy, Duchenne

Genetic Diseases, Inborn

Neuromuscular Diseases

Muscular Disorders, Atrophic

Musculoskeletal Diseases

Muscular Diseases

Nervous System Diseases

Intervention(s)

Drug: Ataluren

Primary Outcome(s)

Number of Participants With Abnormalities in Clinical Laboratory Parameters [Time Frame: Baseline (Day 1) up to 6 weeks post-treatment (Week 150)]

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [Time Frame: Baseline (Day 1) up to 6 weeks post-treatment (Week 150)]

Secondary Outcome(s)

Change From Baseline in PCF as Measured by Spirometry at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in Percent Predicted FVC as Measured by Spirometry at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in PUL Total Score at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in Pulse Rate at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in 6MWD at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in Body Temperature at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in PODCI Transfers/Basic Mobility Score at Week 144 [Time Frame: Baseline, Week 144]

Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 144, as Assessed by a Standardized Survey Administered by Site Personnel [Time Frame: Baseline, Week 144]

Change From Baseline in Time to Descend 4 Stairs at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in Time to Stand From Supine Position at Week 144 [Time Frame: Baseline, Week 144]

Ataluren Plasma Concentration [Time Frame: Pre-dose at Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144]

Change From Baseline in Percent Predicted FEV1 as Measured by Spirometry at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in Time to Climb 4 Stairs at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in Time to Walk/Run 10 Meters at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in PEF as Measured by Spirometry at Week 144 [Time Frame: Baseline, Week 144]

Change From Baseline in Systolic and Diastolic Blood Pressure at Week 144 [Time Frame: Baseline, Week 144]

Secondary ID(s)

PTC124-GD-020e-DMD

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 11/08/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02090959