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Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

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ClinicalTrials.gov Identifier: NCT01247207
Recruitment Status : Enrolling by invitation
First Posted : November 24, 2010
Last Update Posted : February 2, 2023
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Study Description
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Brief Summary:

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial).

The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.


Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Ataluren Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 270 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy
Actual Study Start Date : November 30, 2010
Estimated Primary Completion Date : December 15, 2023
Estimated Study Completion Date : December 15, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Ataluren
Participants will receive 3 doses of ataluren oral suspension per day (10 mg/kg in the morning, 10 mg/kg at mid-day and 20 mg/kg in the evening).
 Drug: Ataluren
Ataluren will be administered as per the dose and schedule specified in the arm.
Other Name: PTC124


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: Baseline up to end of study (up to approximately 8 years) ]
  2. Number of Participants With Laboratory Parameters Abnormalities [ Time Frame: Baseline up to end of study (up to approximately 8 years) ]
    Laboratory parameters include hematological, serum biochemistry, adrenal laboratories, and urine data.

  3. Number of Participants With Abnormal Physical Findings [ Time Frame: Baseline up to end of study (up to approximately 8 years) ]
    Physical findings include weight, physical examination data, systolic and diastolic blood pressure, radial pulse rate, and body temperature.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC) regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
  • History of exposure to ataluren in a prior PTC study or treatment plan and effected nmDBMD siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial).
  • Fertile men, who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 50 days after the last dose of study drug.
  • Willingness and ability to comply with scheduled visits, drug administration and return plan, study procedures, laboratory tests, and study restrictions Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered.

Exclusion Criteria:

  • Exposure to another investigational drug within 1 month prior to start of study treatment.
  • Eligibility for another ataluren clinical trial that is actively enrolling study participants.
  • Positive for Hepatitis B core antibody or Hepatitis C antibody at screening for ataluren naïve participants (siblings) or participants who have a temporary treatment gap of 1 year before entering study.
  • Known hypersensitivity to any of the ingredients or excipients of ataluren (refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, colloidal silica, magnesium stearate).
  • Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
  • Ongoing uncontrolled medical/surgical condition, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant or make it unlikely that follow-up would be completed.
Contacts and Locations
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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): NCT01247207


Locations
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Sponsors and Collaborators
PTC Therapeutics
Investigators
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Study Director: Vinay Penematsa, MD PTC Therapeutics, Inc.
More Information
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Additional Information:

Publications of Results:

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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT01247207    
Other Study ID Numbers: PTC124-GD-016 DMD
First Posted: November 24, 2010    Key Record Dates
Last Update Posted: February 2, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by PTC Therapeutics:
Duchenne muscular dystrophy
Becker muscular dystrophy
Nonsense mutation
Premature stop codon
DMD
 BMD
nmDBMD
DBMD
Ataluren
PTC124
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