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Clinical Trials/CTRI/2018/01/011526
CTRI/2018/01/011526CompletedPhase 3

A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLEDEFFICACY AND SAFETY STUDY OF ATALUREN INPATIENTS WITH NONSENSE MUTATION DUCHENNE MUSCULARDYSTROPHY AND OPEN-LABEL EXTENSIO

PTC Therapeutics Inc0 sites68 target enrollmentStarted: TBDLast updated:
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Trial Snapshot

Phase
Phase 3
Status
Completed
Enrollment
68
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional

Eligibility Criteria

Inclusion Criteria

  • •Age 5 years and older
  • •Phenotypic evidence of Duchenne Muscular Dystrophy
  • •Nonsense point mutation in the dystrophin gene
  • •Use of systemic corticosteroids (prednisone/prednisolone or deflazacort) for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
  • •6MWD greater than or equal to 150 meters
  • •Ability to perform timed function tests within 30 seconds
  • •Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

Exclusion Criteria

  • •- Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start
  • •of study treatment.
  • •- Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
  • •- Prior or ongoing therapy with ataluren.
  • •- Known hypersensitivity to any of the ingredients or excipients of the study drug
  • •- Exposure to another investigational drug within 6 months prior to start of study
  • •treatment, or ongoing participation in any interventional clinical trial.
  • •- History of major surgical procedure within 12 weeks prior to start of study treatment,
  • •or expectation of major surgical procedure during the 72-week placebo-controlled
  • •treatment period.
  • •- Requirement for daytime ventilator assistance or any use of invasive mechanical
  • •ventilation via tracheostomy.
  • •- Uncontrolled clinical symptoms and signs of congestive heart failure
  • •- Elevated serum creatinine or cystatin C at screening.

Investigators

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