Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

NCT ID: NCT02819557

Last Updated: 2020-08-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-06-09
• Study Completion Date: 2018-02-09

Brief Summary

This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

Detailed Description

In nonsense mutation DMD (nmDMD), early start of treatment is important and necessary and, therefore, it is relevant to understand the correct and tolerable dose in this age group, particularly since ataluren is dosed by weight. This study included a 4-week screening period, a 52-week treatment period (the first 4 weeks of which included PK parameters), and a 4-week follow-up period for participants who completed the treatment period (60 weeks total duration). The objective of the extension period (treatment period after PK parameters have been completed) was to assess the long-term safety of chronic administration of ataluren in this participant population.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ataluren

Participants will be administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose will be provided based upon the weight of each participant, which will be assessed every 12 weeks.

Group Type: EXPERIMENTAL

Ataluren

Intervention Type: DRUG

White to off-white powder for oral suspension.

Interventions

Ataluren

White to off-white powder for oral suspension.

Intervention Type: DRUG

Other Intervention Names

PTC124; Translarna

Eligibility Criteria

Inclusion Criteria

• Males ≥2 to <5 years of age
• Body weight ≥12 kg
• Diagnosis of DMD
• Nonsense mutation in at least 1 allele of the dystrophin gene

Exclusion Criteria

• Participation in any other drug or device clinical investigation
• Ongoing use of prohibited concomitant medications

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 5 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

PTC Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Francesco Bibbiani, MD

Role: STUDY_DIRECTOR

PTC Therapeutics

Locations

Child Neuro NWF

Gulf Breeze, Florida, United States

Rush University Medical Center

Chicago, Illinois, United States

Children's Hospital Boston

Boston, Massachusetts, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Medical Center Dallas

Dallas, Texas, United States

University of Utah

Salt Lake City, Utah, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

PTC124-GD-030-DMD

Identifier Type: -

Identifier Source: org_study_id