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Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02819557
Recruitment Status : Completed
First Posted : June 30, 2016
Last Update Posted : January 28, 2019
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Brief Summary:
This is a Phase 2, multiple-dose, open-label study evaluating the safety, and pharmacokinetics (PK) of ataluren in patients aged ≥2 to <5 years old with Duchenne muscular dystrophy caused by a nonsense mutation in the dystrophin gene. The study includes a 4-week screening period, a 4-week study period, and a 48-week extension period for patients who complete the 4-week study period (52 weeks total treatment).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Ataluren Phase 2

Detailed Description:
This protocol describes a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in patients aged ≥2 to <5 years old with nmDMD. In nmDMD, early start of treatment is important and necessary and, therefore, it is relevant to understand the correct and tolerable dose in this age group, particularly since ataluren is dosed by weight. The study will include a 4-week screening period, a 4-week study period, and a 48-week extension period for patients who complete the 4-week study period (52 weeks total treatment). The objective of the extension period is to assess the long-term safety of chronic administration of ataluren in this patient population.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy
Actual Study Start Date : June 16, 2016
Actual Primary Completion Date : February 9, 2018
Actual Study Completion Date : February 9, 2018


Arm Intervention/treatment
Experimental: Ataluren
Oral administration of ataluren at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for 52 weeks.
Drug: Ataluren
Ataluren
Other Names:
  • PTC124
  • Translarna




Primary Outcome Measures :
  1. Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment [ Time Frame: 56 weeks ]
    Safety of ataluren as measured by type, frequency, severity, timing, and relationship to study drug of treatment emergent adverse events,laboratory abnormalities and electrocardiograms


Secondary Outcome Measures :
  1. Maximum Plasma Concentration [Cmax] [ Time Frame: 4 weeks ]
    Pharmacokinetic parameters (tmax, Cmax) based on frequent blood sampling for PK (drug concentrations) on Days 1 and 28 of ataluren treatment.

  2. Area Under the Curve [AUC] and Cmin [ Time Frame: 4 weeks ]
    Pharmacokinetic parameters C(trough) @ 6hrs, and AUC (0-t), t=last time point) based on frequent blood sampling for PK (drug concentrations) on Days 1 and 28 of ataluren treatment.

  3. Descriptive assessments of efficacy [ Time Frame: 56 weeks ]
    TFTs (time to walk/run 10 meters, time to climb 4 stairs, time to descend 4 stairs, and time to stand up from a supine position) ∙ NSAA ∙ Bayley-III (in patients ≤30 months old)

  4. Effects on growth parameters [ Time Frame: 56 weeks ]
    Changes in body weight, height, and BMI



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 5 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males ≥2 to <5 years of age
  • Body weight ≥12 kg
  • Diagnosis of DMD
  • Nonsense mutation in at least 1 allele of the dystrophin gene

Exclusion Criteria:

  • Participation in any other drug or device clinical investigation
  • Ongoing use of prohibited concomitant medications

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): NCT02819557


Locations
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United States, Florida
Child Neuro NWF
Gulf Breeze, Florida, United States, 32561
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Texas
Children's Medical Center Dallas
Dallas, Texas, United States, 75390-8843
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84112
Sponsors and Collaborators
PTC Therapeutics
Investigators
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Study Director: Francesco Bibbiani, MD PTC Therapeutics

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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02819557     History of Changes
Other Study ID Numbers: PTC124-GD-030-DMD
First Posted: June 30, 2016    Key Record Dates
Last Update Posted: January 28, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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