Nonsense mutation dystrophinopathy MedDRA version: 16.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.1 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ataluren
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg m
Sponsors
PTC Therapeutics, Inc
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. 2. History of exposure to ataluren in a prior PTC study in nmDBMD. Note: Subjects 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). 3. Male sex. 4. Confirmed screening laboratory values within the central laboratory ranges specified in the protocol. 5. In patients 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. 6. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions. Are the trial subjects under 18? yes Number of subjects for this age range: 94 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 2 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Exposure to another investigational drug within 1 month prior to start of study treatment. 2. Eligibility for another ataluren clinical trial that is actively enrolling study participants. 3. Known hypersensitivity to any of the ingredients or excipients of ataluren. 4. Ongoing use of the following medications: a. Coumarin based anticoagulants (eg, warfarin), phenytoin, tolbutamide, or paclitaxel. b. Systemic aminoglycoside therapy. 5. Ongoing uncontrolled medical/surgical condition, ECG findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect the safety of the patient or make it unlikely that follow-up would be completed.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the long-term safety and tolerability of 10, 10, 20 mg/kg ataluren in patients with nmDBMD who had prior exposure to ataluren in a PTC-sponsored clinical trial.;Secondary Objective: - Ambulatory patients (able to run/walk 10 meters in =30 seconds): To determine the effect of ataluren on ambulation and other aspects of physical function - Nonambulatory patients (unable to run/walk 10 meters in =30 seconds): To assess the effect of ataluren on activities of daily living, upper limb function, and pulmonary function - All patients: To assess patient and/or parent/caregiver reports of changes in disease status: *Retrospectively during and after participation in previous studies (Studies 007 and 007e) *Prospectively during the current study;Primary end point(s): Safety profile characterized by type, frequency, severity, timing, and relationship to ataluren of any adverse events or laboratory abnormalities.;Timepoint(s) of evaluation of this end point: Every 12 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • In ambulatory patients, change from baseline in 6MWD as measured by the 6-minute walk test (6MWT) •In ambulatory patients, change from baseline in physical function as measured by the North Star Ambulatory Assessment (NSAA) • In ambulatory patients, change from baseline in timed function tests (time to stand from supine and time to run/walk 10 meters) • In nonambulatory patients, change from baseline in pulmonary function as measured by spirometry • In nonambulatory patients, change from baseline in patient and parent/caregiver-reported activities of daily living, as measured by the Egen Klassifikation (EK) scale •In all patients, changes in patient and/or parent/caregiver reports of disease status as measured by a standardized survey administered by site personnel;Timepoint(s) of evaluation of this end point: 6MWT: Screening Visit, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144 NSAA: Screening, Week 48, Week 96, Week 144 Timed function test: Screening, Week 48, Week 96, Week 144 Spirometry: Screening, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144 EK Scale: Screening, Week 48, Week 96, Week 144 Disease status survey: Screening, Week 1, all treatment visits | — |
Countries
Australia, Belgium, Canada, Germany, Israel, Italy, Spain, Sweden, United Kingdom
Contacts
Public ContactClinical Trial Operations
Voisin Consulting
Outcome results
None listed