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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:
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EUCTR |
Last refreshed on:
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10 December 2019 |
Main ID: |
EUCTR2013-005489-20-SE |
Date of registration:
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11/07/2014 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy
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Scientific title:
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A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy |
Date of first enrolment:
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22/08/2014 |
Target sample size:
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220 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-005489-20 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
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Countries of recruitment
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Australia
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Belgium
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Brazil
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Bulgaria
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Canada
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Chile
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Czech Republic
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France
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Germany
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Israel
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Italy
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Korea, Republic of
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Spain
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Sweden
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Switzerland
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Turkey
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trial Operations
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Address:
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3 rue des Longs Prés
92100
Boulogne-Billancourt
France |
Telephone:
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Email:
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clinicaltrialinformation@voisinconsulting.com |
Affiliation:
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Voisin Consulting |
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Name:
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Clinical Trial Operations
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Address:
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3 rue des Longs Prés
92100
Boulogne-Billancourt
France |
Telephone:
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Email:
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clinicaltrialinformation@voisinconsulting.com |
Affiliation:
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Voisin Consulting |
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Key inclusion & exclusion criteria
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Inclusion criteria: 1. Completion of study treatment in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-020-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. 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, ataluren administration plan, study procedures, laboratory tests, and study restrictions. Are the trial subjects under 18? yes Number of subjects for this age range: 220 F.1.2 Adults (18-64 years) no 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: 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 patient, 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
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Health Condition(s) or Problem(s) studied
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Nonsense mutation dystrophinopathy
MedDRA version: 18.1
Level: PT
Classification code 10059117
Term: Becker's muscular dystrophy
System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1
Level: PT
Classification code 10013801
Term: Duchenne muscular dystrophy
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
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Intervention(s)
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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-
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Primary Outcome(s)
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Primary end point(s): Safety profile characterized by type, frequency, severity, timing, and relationship to ataluren of any adverse events or laboratory abnormalities.
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Timepoint(s) of evaluation of this end point: Screening visit, every 12 weeks up to 6 weeks post-treatment.
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Secondary Objective: Physical: • To determine the effect of ataluren on ambulation in subjects who are ambulatory • To evaluate the effect of ataluren on proximal muscle function in subjects who are able to perform timed function tests • To evaluate the effect of ataluren on physical function in subjects who are able to perform the North Star Ambulatory Assessment • To evaluate the effect of ataluren on upper limb function Pulmonary function: • To evaluate the effect of ataluren on pulmonary function Patient-reported outcomes: • To determine the effect of ataluren on health-related quality of life (HRQL) as reported by subjects and parents/caregivers • To assess the effect of ataluren on activities of daily living Exposure: • To assess long-term ataluren plasma exposure
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Main Objective: To evaluate the long-term safety of ataluren in boys with nonsense mutation dystrophinopathy, as determined by adverse events and laboratory abnormalities.
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Secondary Outcome(s)
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Secondary end point(s): Physical: 1. Change in ambulation as measured by the 6MWT 2. Change in proximal muscle function as assessed by timed function tests (time to rise from supine position, time to run 10 meters, and time to climb/descend 4 stairs) 3. Change in physical function as assessed by the North Star Ambulatory Assessment 4. Change in motor performance of the upper limb as measured by the Performance Upper Limb (PUL) Pulmonary function: 5. Change in pulmonary function as measured by spirometry (eg. FVC, FEV1) Patient-reported outcomes: 6. Change in patient- and parent/caregiver-reported HRQL as measured by the PODCI Transfer/Basic Mobility and Sports/Physical Functioning scores 7. Change in patient and parent/caregiver reported activities of daily living Exposure: 8. Pre-dose ataluren plasma concentrations prior to morning ataluren administration at each clinic visit as assessed by a validated bioanalytical method Safety: 9. Change from baseline in other safety parameters (eg, vital signs)
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Timepoint(s) of evaluation of this end point: 1, 2, 3, 4, 6, 7, 8: Screening visit and every 12 weeks up to week 144 5: Screening visit, at weeks 24, 48, 72, 96, 120, 144 9: Screening visit, every 12 weeks up to 6 weeks post-treatment
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Secondary ID(s)
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PTC124-GD-020e-DMD
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68431
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NCT02090959
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Source(s) of Monetary Support
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PTC Therapeutics, Inc.
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Ethics review
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Status: Approved
Approval date:
Contact:
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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