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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)

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ClinicalTrials.gov Identifier: NCT04336826
Recruitment Status : Not yet recruiting
First Posted : April 7, 2020
Last Update Posted : June 22, 2021
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Brief Summary:
This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).

Condition or disease Intervention/treatment Phase
Nonsene Mutation Duchenne Muscular Dystrophy Drug: Ataluren Phase 2

Detailed Description:
Participants who complete the 24-week treatment period in this study will be offered participation to a follow-up extension period for at least 52 weeks from the date of first administration of ataluren in this parent study.

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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
Estimated Study Start Date : July 31, 2021
Estimated Primary Completion Date : December 20, 2022
Estimated Study Completion Date : December 20, 2022


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




Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events [ Time Frame: Baseline up to Week 24 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Pharmacokinetics: Area Under the Concentration Curve From Time 0 to 6 Hours (AUC0-6) 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 ]
  3. 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 ]
  4. 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 ]
  5. Pharmacokinetics: Trough Concentration (Ctrough) of Ataluren [ Time Frame: Pre-morning dose at Week 24 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 2 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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:

    1. Systemic aminoglycoside therapy and/or intravenous (IV) vancomycin.
    2. Coumarin-based anticoagulants (for example, warfarin), phenytoin, tolbutamide, or paclitaxel.
    3. 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).

Information from the National Library of Medicine

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


Contacts
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Contact: Patient Advocacy Corporate Relations 1-866-562-4620 medinfo@ptcbio.com

Sponsors and Collaborators
PTC Therapeutics
Investigators
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Study Director: Vinay Penematsa PTC Therapeutics, Inc.
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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 22, 2021
Last Verified: June 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked