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

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ClinicalTrials.gov Identifier: NCT02090959
Recruitment Status : Active, not recruiting
First Posted : March 19, 2014
Last Update Posted : January 18, 2018
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Muscular Dystrophies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Drug: Ataluren Phase 3

Detailed Description:
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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 219 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
Study Start Date : March 2014
Estimated Primary Completion Date : June 2018
Estimated Study Completion Date : June 2018


Arm Intervention/treatment
Experimental: Ataluren
10, 10, 20 mg/kg for 96 weeks.
Drug: Ataluren
Other Name: PTC124



Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. Physical Function [ Time Frame: Baseline and 96 weeks ]
    North Star Ambulatory Assessment,Timed Function Testing, Upper Limb Function, 6 Minute Walk Test

  2. Patient and/or parent-reported activities of daily living and disease symptoms [ Time Frame: Baseline and 96 weeks ]
  3. Quality of Life [ Time Frame: Baseline and 96 weeks ]
  4. Pulmonary function [ Time Frame: Baseline and 96 weeks ]
  5. Ataluren blood levels [ Time Frame: Baseline and 96 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02090959


  Show 54 Study Locations
Sponsors and Collaborators
PTC Therapeutics
Investigators
Study Director: Robert Spiegel, M.D. PTC Therapeutics

Additional Information:
Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02090959     History of Changes
Other Study ID Numbers: PTC124-GD-020e-DMD
First Posted: March 19, 2014    Key Record Dates
Last Update Posted: January 18, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by PTC Therapeutics:
Duchenne muscular dystrophy
Dystrophinopathy
Nonsense mutation
Premature stop codon
Becker muscular dystrophy
DMD/BMD
PTC124
Ataluren

Additional relevant MeSH terms:
Muscular Dystrophies
Nervous System Diseases
Genetic Diseases, Inborn
Neuromuscular Diseases
Musculoskeletal Diseases
Muscular Diseases
Muscular Dystrophy, Duchenne
Genetic Diseases, X-Linked
Muscular Disorders, Atrophic