Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00847379
• Date of registration: 16/02/2009
• Prospective Registration: No
• Primary sponsor: PTC Therapeutics
• Public title: Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
• Scientific title: A Phase 2B Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy
• Date of first enrolment: January 31, 2009
• Target sample size: 173
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00847379
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Canada, France, Germany, Israel, Italy, Spain, Sweden, United Kingdom, United States

Contacts

• Name: Leone Atkinson, M.D., Ph.D.
• Affiliation: PTC Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Completion of blinded study drug treatment in the previous Phase 2b study
(PTC124-GD-007-DMD).

- Ability to provide written informed consent (parental/guardian consent if
applicable)/assent (if less than [<]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 PTC124
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.

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], 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, ECG findings, or laboratory abnormality that, in the investigator's opinion,
could adversely affect the safety of the subject, makes it unlikely that the course of
treatment or follow up would be completed, or could impair the assessment of study
results.



Age minimum: 5 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Becker Muscular Dystrophy

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Ataluren

Primary Outcome(s)

Number of Participants With Treatment-Emergent Adverse Events (AEs) [Time Frame: Baseline (Week 48 of Study 007) up to Week 102]

Number of Participants With Clinically Significant Abnormal Laboratory Parameters [Time Frame: Baseline (Week 48 of Study 007) up to Week 102]

Secondary Outcome(s)

Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Trough Ataluren Plasma Concentration [Time Frame: Pre-morning dose (0 hour) at Baseline (Week 48 of 007 study), Weeks 54, 60, 72, 84, and 96]

Change From Baseline in Time to Climb 4 Stairs at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Time to Descend 4 Stairs at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Mean Activity Period/Day/Visit at Week 60, as Assessed by Step Activity Monitoring (SAM) [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Serum Concentration of Creatine Kinase (CK) at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Time to Walk/Run 10 Meters at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Participant-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 60, as Assessed by SAM [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 60, as Assessed by SAM [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Parent/Caregiver-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [=]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Participant and Parent/Caregiver Reported Activities of Daily Living of Participants Who Were Unable to Complete the 6MWT (Nonambulatory Participants), as Measured by the Egen Klassifikation (EK) Scale at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Change From Baseline in Time to Stand From Supine Position at Week 60 [Time Frame: Baseline (Week 48 of Study 007), Week 60]

Study Drug Compliance [Time Frame: Baseline (Week 48 of Study 007) to Week 96]

Secondary ID(s)

PTC124-GD-007e-DMD

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Genzyme, a Sanofi Company

Results

• Results available: Yes
• Date Posted: 15/07/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00847379