Phase 2 N=14 Treatment

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

Duchenne Muscular Dystrophy

Bottom Line

View on ClinicalTrials.gov: NCT02819557 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Aug 2020

Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) — 14; 5; 8; 1 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Ataluren (Drug)
Age: Pediatric · 2+ yrs
Sex: Male
Sponsor: PTC Therapeutics
Primary completion: Feb 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs)	14; 5; 8; 1; 5; 0	—
PRIMARY Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter	—	—
PRIMARY Number of Participants With a Clinically Meaningful Abnormal Electrocardiogram (ECG) Test Results	—	—
PRIMARY Number of Participants With a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity	—	—
SECONDARY Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr)	15.95; 12.54	—
SECONDARY Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr)	3.84; 2.72	—
SECONDARY Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr)	101.64; 82.13	—
SECONDARY Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr)	9.12; 5.43	—
SECONDARY Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs	7.2; -3.1; -3.1; 6.6; -0.8; -1.1	—
SECONDARY Change From Baseline in Physical Function as Measured by the NSAA	16.0; 3.5; 5.5; 10.5; 1.5; 2.3	—
SECONDARY Change From Baseline in Height of Participants at Weeks 4, 16, 28, 40, 52, and 56	99.43; 0.83; 1.84; 3.11; 3.82; 5.95	—
SECONDARY Change From Baseline in Weight of Participants at Weeks 4, 16, 28, 40, 52, and 56	16.99; -0.04; 0.73; 1.16; 1.39; 1.90	—
SECONDARY Change From Baseline in Body Mass Index of Participants at Weeks 4, 16, 28, 40, 52, and 56	17.094; -0.346; 0.026; 0.030; 0.008; -0.186	—
SECONDARY Ataluren Palatability Characteristics as Determined by a Parent/Caregiver Questionnaire	0; 0; 2; 0; 0; 12	—

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.

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02819557). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.