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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01247207
Recruitment Status : Enrolling by invitation
First Posted : November 24, 2010
Last Update Posted : July 11, 2019
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

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.

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy
Actual Study Start Date : November 30, 2010
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : December 31, 2020


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




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.



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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 in nmDBMD.
  • In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 6-week follow up period.
  • Willingness and ability to comply with scheduled visits, drug administration 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.
  • Known hypersensitivity to any of the ingredients or excipients of ataluren (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P [colloidal silica], magnesium stearate).
  • Ongoing use of the systemic aminoglycoside 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.

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


  Hide Study Locations
Locations
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United States, California
UCLA
Los Angeles, California, United States, 90095
University of California-Davis
Sacramento, California, United States, 95817
United States, Colorado
The Children's Hospital
Aurora, Colorado, United States, 80045
United States, Florida
Child Neurology Center of NW Florida
Gulf Breeze, Florida, United States, 32561
United States, Illinois
Rush Univ Medical Center
Chicago, Illinois, United States, 60612
United States, Iowa
University of Iowa Children's Hospital
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Massachusetts
Children's Hospital of Boston/Harvard Medical School
Boston, Massachusetts, United States, 02115
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Washington University Medical School
Saint Louis, Missouri, United States, 63110
United States, New York
Columbia University Medical School
New York, New York, United States, 10032
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Ohio
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Oregon
Shriners Hospital for Children-Portland
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Univ of Pitt Medical Center
Pittsburgh, Pennsylvania, United States, 16148
United States, Texas
Southwestern University
Dallas, Texas, United States, 75207
Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84112
United States, Virginia
Univ of Virginia
Charlottesville, Virginia, United States, 22903
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada
Canada, British Columbia
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Canada, Ontario
Children's Hospital
London, Ontario, Canada
Sponsors and Collaborators
PTC Therapeutics
Investigators
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Study Director: Francesco Bibbiani, MD PTC Therapeutics, Inc.

Additional Information:
Publications of Results:
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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT01247207     History of Changes
Other Study ID Numbers: PTC124-GD-016 DMD
First Posted: November 24, 2010    Key Record Dates
Last Update Posted: July 11, 2019
Last Verified: July 2019
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