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

March 1, 2024 updated by: PTC Therapeutics

An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

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.

Study Overview

Status

Enrolling by invitation

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

270

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Quebec, Canada
        • CHU de Québec - Université Laval
    • Alberta
      • Calgary, Alberta, Canada
        • Alberta Children'S Hospital
    • British Columbia
      • Vancouver, British Columbia, Canada
        • British Columbia Children's Hospital
    • Ontario
      • London, Ontario, Canada
        • London Health Sciences Centre
      • Ottawa, Ontario, Canada
        • Children's Hospital of East Ontario
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Childrens Hospital
    • California
      • Los Angeles, California, United States, 90095
        • UCLA
      • Los Angeles, California, United States, 90027
        • Children's Hospital of Los Angeles
      • Palo Alto, California, United States, 94304
        • Stanford University
      • Sacramento, California, United States, 95817
        • University of California-Davis
      • San Francisco, California, United States, 94158
        • University of CA, San Francisco-Benioff Children's Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado - Center for Cancer and Blood Disorders
    • Florida
      • Gulf Breeze, Florida, United States, 32561
        • Child Neurology Center of NW Florida
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Rare Disease Research, LLC
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush Univ Medical Center
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Children's Hospital
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Children's Hospital of Boston/Harvard Medical School
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University Medical School
    • New York
      • New York, New York, United States, 10032
        • Columbia University Pediatric Neuromuscular Center
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Childrens Hospital Medical Center
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Shriners Hospital for Children-Portland
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 16148
        • Univ of Pitt Medical Center
    • Texas
      • Dallas, Texas, United States, 75207
        • Childrens Medical Center Dallas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
      • San Antonio, Texas, United States, 78229
        • University of Texas Health Science Center at San Antonio
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • University of Utah
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Children's Hospital of The King's Daughters
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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).
Ataluren will be administered as per the dose and schedule specified in the arm.
Other Names:
  • PTC124

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse Events
Time Frame: Baseline up to end of study (up to approximately 8 years)
Baseline up to end of study (up to approximately 8 years)
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.
Baseline up to end of study (up to approximately 8 years)
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.
Baseline up to end of study (up to approximately 8 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Study Director: Vinay Penematsa, MD, PTC Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 30, 2010

Primary Completion (Estimated)

April 30, 2024

Study Completion (Estimated)

April 30, 2024

Study Registration Dates

First Submitted

November 22, 2010

First Submitted That Met QC Criteria

November 23, 2010

First Posted (Estimated)

November 24, 2010

Study Record Updates

Last Update Posted (Actual)

March 5, 2024

Last Update Submitted That Met QC Criteria

March 1, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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