A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2008-007648-32-BE
• Date of registration: 25/02/2009
• Prospective Registration: Yes
• Primary sponsor: PTC Therapeutics, Inc.
• Public title: A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy
• Scientific title: A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy
• Date of first enrolment: 14/04/2009
• Target sample size: 174
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-007648-32
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Belgium, France, Germany, Italy, Spain, Sweden, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
Patients must meet all of the following conditions to be eligible for enrollment into the study:
1. Completion of study treatment in the previous Phase 2b study protocol (Protocol PTC124 GD 007 DMD)
2. 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. 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 one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the subject should be followed.
3. In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 6 week follow up period.
4. Willingness and ability to comply with scheduled visits, PTC124 administration plan, study procedures, laboratory tests, and study restrictions.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patients meeting any of the following conditions will not be eligible for enrollment into the study:
1. 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).
2. Ongoing participation in any other therapeutic clinical trial.
3. Prior or ongoing medical condition (eg, 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:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10059117 Term: Becker's muscular dystrophy

Intervention(s)

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
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-

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
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-

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
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-

Primary Outcome(s)

Main Objective: To evaluate the long term safety of PTC124 in boys with nonsense mutation mediated DMD/BMD, as determined by adverse events and laboratory abnormalities.

Primary end point(s): Safety profile characterized by type, frequency, severity, timing, and relationship to PTC124 of any adverse events or laboratory abnormalities

Secondary Objective: Physical
• To determine the effect of PTC124 on ambulation in the clinic and in the community setting in subjects who are ambulatory
• To evaluate the effect of PTC124 on functional aspects of proximal muscle strength in subjects who are able to perform timed function tests
Cardiac function
• To evaluate the effect of PTC124 on cardiac function
Cognitive
• To evaluate the effect of PTC124 on cognitive ability
Patient reported outcomes
• To determine the effect of PTC124 on HRQL and treatment satisfaction as reported by subjects and parents/caregivers
• To assess the effect of PTC124 on activities of daily living in subjects who are unable to complete the 6MWT
Pharmacodynamics
• To evaluate the effect of PTC124 on muscle fragility as determined by serum CK levels
Exposure
• To determine compliance with PTC124 therapy
• To assess long-term PTC124 plasma exposure

Secondary ID(s)

PTC124 GD 007e DMD