Trial document





This study has been imported from ClinicalTrials.gov without additional data checks.
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  DRKS00007237

Trial Description

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Title

A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

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Trial Acronym

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URL of the Trial

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Brief Summary in Lay Language

Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which
develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is
important for maintaining normal muscle structure and function. Loss of dystrophin causes
muscle fragility that leads to weakness and loss of walking ability. A specific type of
mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy
in approximately 10-15% of boys with the disease. Ataluren is an orally delivered,
investigational drug that has the potential to overcome the effects of the nonsense
mutation. The main goal of this Phase 3 extension study is to obtain long term safety of
ataluren in boys with nonsense mutation dystrophinopathy as determined by adverse events and
laboratory abnormalities. The study will also assess changes in physical function,
pulmonary function and other important clinical and laboratory measures.

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Brief Summary in Scientific Language

This Phase 3, open label safety and efficacy study will be performed at participating
sites worldwide. The study will enroll ~ 220 boys with nonsense mutation dystrophinopathy
who participated in a previous Phase 3 study of ataluren (Protocol # PTC124-GD-020-DMD).
Patients will receive 10, 10, 20 mg/kg of ataluren TID at morning, midday, and evening for
approximately 96 weeks. Study assessments will be performed at clinic visits every 12
weeks.

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Organizational Data

  •   DRKS00007237
  •   2015/05/07
  •   2014/03/17
  •   no
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Secondary IDs

  •   NCT02090959  (ClinicalTrials.gov)
  •   PTC124-GD-020e-DMD  (PTC Therapeutics)
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Health Condition or Problem studied

  •   Muscular Dystrophy, Duchenne
  •   Muscular Dystrophies
  •   Muscular Disorders, Atrophic
  •   Muscular Diseases
  •   Musculoskeletal Diseases
  •   Neuromuscular Diseases
  •   Nervous System Diseases
  •   Genetic Diseases, X-Linked
  •   Genetic Diseases, Inborn
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Interventions/Observational Groups

  •   Drug: Ataluren
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Characteristics

  •   Interventional
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  •   Single arm study
  •   Open (masking not used)
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  •   Uncontrolled/Single arm
  •   Treatment
  •   Single (group)
  •   III
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Primary Outcome

- Long term safety of ataluren in boys with nonsense mutation dystrophinopathy, as determined by adverse events and laboratory abnormalities; time frame: Baseline and 96 weeks

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Secondary Outcome

- Physical Function; time frame: Baseline and 96 weeks; North Star Ambulatory Assessment,Timed Function Testing, Upper Limb Function, 6 Minute Walk Test
- Patient and/or parent-reported activities of daily living and disease symptoms; time frame: Baseline and 96 weeks
- Quality of Life; time frame: Baseline and 96 weeks
- Pulmonary function; time frame: Baseline and 96 weeks
- Ataluren blood levels; time frame: Baseline and 96 weeks

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Countries of Recruitment

  •   United States
  •   Australia
  •   Belgium
  •   Brazil
  •   Canada
  •   Chile
  •   Czech Republic
  •   France
  •   Germany
  •   Israel
  •   Italy
  •   Korea, Republic of
  •   Poland
  •   Spain
  •   Sweden
  •   Switzerland
  •   Turkey
  •   United Kingdom
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Locations of Recruitment

  •  
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Recruitment

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  •   2014/03/31
  •   220
  •   Multicenter trial
  •   International
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Inclusion Criteria

  •   Male
  •   7   Years
  •   18   Years
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Additional Inclusion Criteria

- Completion of study treatment in the previous Phase 3, double-blind study protocol
(Protocol PTC124-GD-020-DMD).

- Evidence of signed and dated informed consent/assent document(s) indicating that the
patient (and/or his parent/legal guardian) has been informed of all pertinent aspects
of the trial.

- Willingness to abstain from sexual intercourse or employ an approved method of
contraception during the period of study drug administration and 6-week follow-up
period.

- Willingness and ability to comply with scheduled visits, drug administration plan,
study procedures, laboratory tests, and study restrictions.

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Exclusion Criteria

- Known hypersensitivity to any of the ingredients or excipients of the study drug

- Ongoing participation in any clinical trial (except for studies specifically approved
by PTC Therapeutics).

- 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.

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Addresses

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    • PTC Therapeutics
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    • PTC Therapeutics
    • Robert Spiegel, M.D. 
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    • PTC Therapeutics
    • Robert Spiegel, M.D. 
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Sources of Monetary or Material Support

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    • Bitte wenden Sie sich an den Sponsor / Please refer to primary sponsor
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Status

  •   Recruiting ongoing
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Trial Publications, Results and other Documents

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The parameters in ClinicalTrials.gov and DRKS are not identical. Therefore the data import from ClinicalTrials.gov required adjustments. For full details please see the DRKS FAQs .
  •   1
  •   2014/11/05
* This entry means the parameter is not applicable or has not been set.