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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 12 March 2018
Main ID:  EUCTR2011-004853-18-BE
Date of registration: 03/01/2012
Prospective Registration: Yes
Primary sponsor: PTC Therapeutics, Inc
Public title: Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy
Scientific title: An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy
Date of first enrolment: 27/04/2012
Target sample size: 96
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-004853-18
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no
Randomised: no
Open: no
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Australia Belgium Canada Germany Israel Italy Spain Sweden
United Kingdom
Contacts
Name: Clinical Trial Operations   
Address:  3 rue des Longs Prés 92100 Boulogne-Billancourt France
Telephone:
Email: clinicaltrialinformation@voisinconsulting.com
Affiliation:  Voisin Consulting
Name: Clinical Trial Operations   
Address:  3 rue des Longs Prés 92100 Boulogne-Billancourt France
Telephone:
Email: clinicaltrialinformation@voisinconsulting.com
Affiliation:  Voisin Consulting
Key inclusion & exclusion 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:
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.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes
Health Condition(s) or Problem(s) studied
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
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
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
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
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
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-

Primary Outcome(s)
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.
Primary end point(s): Safety profile characterized by type, frequency, severity, timing, and relationship to ataluren of any adverse events or laboratory abnormalities.
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
Timepoint(s) of evaluation of this end point: Every 12 weeks
Secondary Outcome(s)
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
Secondary ID(s)
PTC124-GD-019-DMD
68,431
NCT01557400
Source(s) of Monetary Support
PTC Therapeutics, Inc.
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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