Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
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ClinicalTrials.gov Identifier: NCT03179631 |
Recruitment Status
:
Recruiting
First Posted
: June 7, 2017
Last Update Posted
: March 30, 2018
|
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Muscular Dystrophy, Duchenne Muscular Dystrophies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Disease Neuromuscular Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn | Drug: Ataluren Drug: PLACEBO | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 250 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | This study describes the randomized, double-blind, placebo-controlled, 72-week study and its 72-week open-label extension |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Masking Description: | A randomized, double-blind, placebo-controlled,72-week study and its 72-week open-label extension |
Primary Purpose: | Treatment |
Official Title: | A Phase 3,Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension |
Actual Study Start Date : | July 6, 2017 |
Estimated Primary Completion Date : | March 2020 |
Estimated Study Completion Date : | December 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: Ataluren
10, 10, 20 mg/kg
|
Drug: Ataluren
10, 10, 20 mg/kg
Other Name: PTC124
|
Placebo Comparator: Placebo
10, 10, 20 mg/kg
|
Drug: PLACEBO
10, 10, 20 mg/kg
Other Name: Matching Placebo
|
- 6- Minute Walk Test [ Time Frame: 72 weeks ]
- Timed Function Tests [ Time Frame: 72 weeks ]
- North Star Ambulatory Assessment [ Time Frame: 72 weeks ]
- Performance of Upper Limb (in patients >=7 years old at baseline) [ Time Frame: 72 weeks ]
- Myometry (in patients <7 years old at baseline) [ Time Frame: 72 weeks ]
- Magnetic Resonance Imaging (MRI) (at pre-qualified sites) [ Time Frame: 72 weeks ]
- EQ-5D [ Time Frame: 72 weels ]
- Ataluren safety profile characterized by adverse events and abnormalities of laboratory tests, vital signs, physical examinations, or electrocardiograms [ Time Frame: 72 weeks ]

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Ages Eligible for Study: | 5 Years and older (Child, Adult, Senior) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria -
- Male sex.
- Age ≥5 years.
- Phenotypic evidence of DMD
- Nonsense point mutation in the dystrophin gene
- Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
- 6MWD ≥150 meters
- Ability to perform timed function tests within 30 seconds
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion Criteria:
- Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.
- Prior or ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
- Prior or ongoing therapy with ataluren.
- Known hypersensitivity to any of the ingredients or excipients of the study drug
- Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial.
- History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period.
- Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy.
- Uncontrolled clinical symptoms and signs of congestive heart failure
- Elevated serum creatinine or cystatin C at screening.

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): NCT03179631
Contact: Mary Frances Harmon | 908-912-9256 | mharmon@ptcbio.com | |
Contact: Amy Perniciaro | 908-912-9264 | aperniciaro@ptcbio.com |

Study Director: | Francesco Bibbiani, MD | PTC Therapeutics, Inc. |
Additional Information:
Responsible Party: | PTC Therapeutics |
ClinicalTrials.gov Identifier: | NCT03179631 History of Changes |
Other Study ID Numbers: |
PTC124-GD-041-DMD |
First Posted: | June 7, 2017 Key Record Dates |
Last Update Posted: | March 30, 2018 |
Last Verified: | March 2018 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes | |
Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by PTC Therapeutics:
Duchenne Muscular Dystrophy Dystrophinopathy Nonsense Mutation Premature Stop Codon |
Becker Muscular Dystrophy DMD/BMD PTC124 Ataluren |
Additional relevant MeSH terms:
Muscular Dystrophies Muscular Dystrophy, Duchenne Nervous System Diseases Genetic Diseases, Inborn Neuromuscular Diseases |
Musculoskeletal Diseases Muscular Diseases Genetic Diseases, X-Linked Muscular Disorders, Atrophic |