A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
![]() |
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04336826 |
Recruitment Status :
Recruiting
First Posted : April 7, 2020
Last Update Posted : June 29, 2022
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Nonsene Mutation Duchenne Muscular Dystrophy | Drug: Ataluren | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 10 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy |
Actual Study Start Date : | December 29, 2021 |
Estimated Primary Completion Date : | March 31, 2023 |
Estimated Study Completion Date : | March 31, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: Ataluren
Participants will receive ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 24 weeks.
|
Drug: Ataluren
Ataluren will be administered as per the dose and schedule specified in the arm.
Other Name: PTC124 |
- Number of Participants With Treatment-Emergent Adverse Events [ Time Frame: Baseline up to Week 24 ]
- Pharmacokinetics: Area Under the Concentration Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren [ Time Frame: Predose, 1, 2, 3, and 4 hours post morning and mid-day doses; and pre-dose, 1, 2, 3, 4 and 12 (before the next day morning dose) hours post evening dose at Week 24 ]
- Pharmacokinetics: Area Under the Concentration Curve Between Dosing Interval (AUC0-τ) of Ataluren [ Time Frame: Predose, 1, 2, 3, and 4 hours post morning and mid-day doses; and pre-dose, 1, 2, 3, 4 and 12 (before the next day morning dose) hours post evening dose at Week 24 ]
- Pharmacokinetics: Maximum Concentration (Cmax) of Ataluren [ Time Frame: Predose, 1, 2, 3, and 4 hours post morning and mid-day doses; and pre-dose, 1, 2, 3, 4 and 12 (before the next day morning dose) hours post evening dose at Week 24 ]
- Pharmacokinetics: Time to Maximal Plasma Concentration (Tmax) of Ataluren [ Time Frame: Predose, 1, 2, 3, and 4 hours post morning and mid-day doses; and pre-dose, 1, 2, 3, 4 and 12 (before the next day morning dose) hours post evening dose at Week 24 ]
- Pharmacokinetics: Trough Concentration (Ctrough) of Ataluren [ Time Frame: Pre-morning dose at Week 24 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 6 Months to 2 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Body weight ≥7.5 kilograms (kg)
- Diagnosis of duchenne muscular dystrophy (DMD) based on an elevated serum creatine kinase and genotypic evidence of dystrophinopathy.
- Documentation of the presence of a nonsense mutation of the dystrophin gene as determined by gene sequencing prior to enrollment.
Exclusion Criteria:
- Participation in any drug or device investigation or whose sibling is currently participating in a blinded portion of another ataluren study or received an investigational drug within three months prior to the Screening Visit or who anticipate participating in any other drug or device clinical investigation or receiving any other investigational drug within the duration of this study.
- Expectation of a major surgical procedure during the study period.
- Known hypersensitivity to any of the ingredients or excipients of the study drug (polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, colloidal silica, or magnesium stearate).
-
Ongoing use of the following drugs:
- Systemic aminoglycoside therapy and/or intravenous (IV) vancomycin.
- Coumarin-based anticoagulants (for example, warfarin), phenytoin, tolbutamide, or paclitaxel.
- Inducers of UGT1A9 (for example, rifampicin), or substrates of OAT1 or OAT3 (for example, ciprofloxacin, adefovir, oseltamivir, aciclovir, captopril, furosemide, bumetanide, valsartan, pravastatin, rosuvastatin, atorvastatin, pitavastatin).

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): NCT04336826
Contact: Patient Advocacy Corporate Relations | 1-866-562-4620 | medinfo@ptcbio.com |
United States, Georgia | |
Rare Disease Research, LLC | Recruiting |
Atlanta, Georgia, United States, 30329 | |
Principal Investigator: Han C. Phan, MD |
Study Director: | Vinay Penematsa | PTC Therapeutics, Inc. |
Responsible Party: | PTC Therapeutics |
ClinicalTrials.gov Identifier: | NCT04336826 |
Other Study ID Numbers: |
PTC124-GD-048-DMD 2020-000980-21 ( EudraCT Number ) |
First Posted: | April 7, 2020 Key Record Dates |
Last Update Posted: | June 29, 2022 |
Last Verified: | June 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |