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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT01247207
• Other study ID #: PTC124-GD-016 DMD
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: November 30, 2010
• Est. completion date: April 30, 2025

Study information

• Verified date: May 2024
• Source: PTC Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 270
• Est. completion date: April 30, 2025
• Est. primary completion date: April 30, 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Ataluren
Ataluren will be administered as per the dose and schedule specified in the arm.

Locations

Country	Name	City	State
Canada	Alberta Children's Hospital	Calgary	Alberta
Canada	London Health Sciences Centre	London	Ontario
Canada	Children's Hospital of East Ontario	Ottawa	Ontario
Canada	CHU de Québec - Université Laval	Quebec
Canada	British Columbia Children's Hospital	Vancouver	British Columbia
United States	University of Michigan	Ann Arbor	Michigan
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Children's Hospital Colorado - Center for Cancer and Blood Disorders	Aurora	Colorado
United States	Children's Hospital of Boston/Harvard Medical School	Boston	Massachusetts
United States	Rush Univ Medical Center	Chicago	Illinois
United States	Cincinnati Childrens Hospital Medical Center	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Childrens Medical Center Dallas	Dallas	Texas
United States	Duke University Medical Center	Durham	North Carolina
United States	Child Neurology Center of NW Florida	Gulf Breeze	Florida
United States	Texas Children's Hospital	Houston	Texas
United States	University of Iowa Children's Hospital	Iowa City	Iowa
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	UCLA	Los Angeles	California
United States	University of Minnesota	Minneapolis	Minnesota
United States	Columbia University Pediatric Neuromuscular Center	New York	New York
United States	Children's Hospital of the King's Daughters	Norfolk	Virginia
United States	Stanford University	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Phoenix Childrens Hospital	Phoenix	Arizona
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
United States	Shriners Hospital for Children-Portland	Portland	Oregon
United States	University of California-Davis	Sacramento	California
United States	Washington University Medical School	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah
United States	University of Texas Health Science Center at San Antonio	San Antonio	Texas
United States	University of CA, San Francisco-Benioff Children's Hospital	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
PTC Therapeutics

Countries where clinical trial is conducted

United States,  Canada, 

References & Publications (2)

Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL. Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple- — View Citation

Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events		Baseline up to end of study (up to approximately 8 years)
Primary	Number of Participants With Laboratory Parameters Abnormalities	Laboratory parameters include hematological, serum biochemistry, adrenal laboratories, and urine data.	Baseline up to end of study (up to approximately 8 years)
Primary	Number of Participants With Abnormal Physical Findings	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)

External Links

•   Protocol V7.0
•   PTC Therapeutics, Inc website