Back to resultsA 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)
Primary Purpose
Nonsene Mutation Duchenne Muscular Dystrophy
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Ataluren
Sponsored by
About this trial
This is an interventional treatment trial for Nonsene Mutation Duchenne Muscular Dystrophy
Eligibility 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:
- 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).
Sites / Locations
- Rare Disease Research, LLC
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ataluren
Arm Description
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.
Outcomes
Primary Outcome Measures
Number of Participants With Treatment-Emergent Adverse Events
Secondary Outcome Measures
Pharmacokinetics: Area Under the Concentration Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren
Pharmacokinetics: Area Under the Concentration Curve Between Dosing Interval (AUC0-τ) of Ataluren
Pharmacokinetics: Maximum Concentration (Cmax) of Ataluren
Pharmacokinetics: Time to Maximal Plasma Concentration (Tmax) of Ataluren
Pharmacokinetics: Trough Concentration (Ctrough) of Ataluren
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04336826
Brief Title
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)
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
December 29, 2021 (Actual)
Primary Completion Date
August 7, 2023 (Actual)
Study Completion Date
August 7, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
PTC Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Nonsene Mutation Duchenne Muscular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Ataluren
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Ataluren
Other Intervention Name(s)
PTC124
Intervention Description
Ataluren will be administered as per the dose and schedule specified in the arm.
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-Emergent Adverse Events
Time Frame
Baseline up to Week 24
Secondary Outcome Measure Information:
Title
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
Title
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
Title
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
Title
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
Title
Pharmacokinetics: Trough Concentration (Ctrough) of Ataluren
Time Frame
Pre-morning dose at Week 24
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility 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:
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).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Vinay Penematsa
Organizational Affiliation
PTC Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
12. IPD Sharing Statement
Learn more about this trial
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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