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

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.

PTC Identifier:
PTC124-GD-016 DMD
ClinicalTrials.gov Identifier:
NCT01247207
EudraCT Identifier:
N/A
Recruiting

Trial Purpose

Age
N/A - N/A
Sex
Male
Healthy Volunteers
No

Key Participation Requirements

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).

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.

Trial Summary

Total Participants

This is the number of individuals who are taking part in the trial.

Total Participants
270

Trial Dates

These are the start and end dates for the trial. Note that future dates are estimates that may be updated before or during the trial.

Trial Dates
Nov 2010 - Apr 2025

Phase

These are the different steps that a treatment goes through during clinical development before it is approved for use.

Phase
Phase 3

Placebo Controlled

In this type of study, some participants receive a “placebo,” which looks like the study treatment, but does not actually contain the active treatment.

Placebo Controlled
No

Product

This is any type of drug, device, or other treatment that is being studied in the trial.

Product
Ataluren

Accepts Healthy Volunteers

Healthy volunteers are individuals who participate in the trial and do not have that specific disease or condition.

Accepts Healthy Volunteers
No
Discuss with the trial center for details

Trial Design

Trial Type

This is the general nature of the study.

Trial Type
Interventional

Interventional

Participants in these studies are subject to an intervention by researchers. These trials are used to understand the effects of a particular intervention.

Discuss with the trial center for details

Blinding

Study participants know which treatments they might get if they join the trial, however during a trial they may not be informed which treatment they are actually taking. The act of not disclosing this information to the participant is known as “blinding.”

Blinding
Open Label

Open Label

This means that the participants in the trial are aware of what treatment they are using, and the researchers are as well.

Discuss with the trial center for details

Intervention Type

This is a medicine, device, or other type of treatment that is being studied or is already approved for use. Interventions can also include behavior changes, like diet and exercise.

Intervention Type
Drug

Allocation

This determines how study participants are assigned their treatment.

Allocation
N/A

Trial Purpose

This is the rationale behind why the clinical trial is being performed.

Trial Purpose
Treatment

Trial Arms

These are the groups of participants in a clinical study. Each different “arm” is assigned a different treatment to use. Different types of trial arms in studies can include:

Trial Arms
Experimental

Experimental arm

Experimental arm: The group assigned to use the study treatment

Primary Outcome

Primary Outcome

This is the most important measurement of the study and is used to help researchers evaluate the effect of a treatment.

Number of Participants With Adverse Events

Timeframe: Baseline up to end of study (up to approximately 8 years)

Number of Participants With Laboratory Parameters Abnormalities

Timeframe: Baseline up to end of study (up to approximately 8 years)

Number of Participants With Abnormal Physical Findings

Timeframe: Baseline up to end of study (up to approximately 8 years)

Secondary Outcome

Secondary Outcome

This measurement is a secondary measurement and is still important in helping researchers evaluate the effect of a treatment.

Contact Information

Location
Status
Location
University of Utah
Salt Lake City, UT, United States, 84112
Status
Enrolling by invitation
Location
Children's Hospital of Philadelphia
Philadelphia, PA, United States, 19104
Status
Enrolling by invitation
Location
University of Minnesota
Minneapolis, MN, United States, 55455
Status
Enrolling by invitation
Location
University of Iowa Children's Hospital
Iowa City, IA, United States, 52242
Status
Enrolling by invitation
Location
University of Kansas Medical Center
Kansas City, KS, United States, 66160
Status
Enrolling by invitation
Location
Washington University Medical School
St. Louis, MO, United States, 63110
Status
Terminated