Clinical trial NCT03218995: Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
Official title: An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: DMD
Duchenne
Eteplirsen
dystrophy
dystrophin
exon 51
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2017-07-17
Enrollment: 15
Sex: MALE
Minimum age (years): 6
Maximum age (years): 48
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.


Eligibility criteria: Inclusion Criteria:

* Male between 6 months to 48 months of age (inclusive)
* Diagnosis of DMD with a deletion mutation amenable to exon 51 skipping
* Parent(s) or legal guardian(s) who is willing to provide written informed consent

Exclusion Criteria:

* Received treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing
* Received previous or current treatment with any experimental treatment
* Clinically significant illness other than DMD
* Clinically significant laboratory abnormality
* Any other condition that could interfere with the participation in the study.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug
timeFrame: Baseline up to Week 100
measure: Number of Participants With at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality
timeFrame: Baseline up to Week 100
measure: Number of Participants With at Least 1 Markedly Abnormal Vital Sign
timeFrame: Baseline up to Week 100
measure: Abnormal Changes From Baseline or Worsening of Physical Examination Findings
timeFrame: Baseline up to Week 100
measure: Number of Participants With at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO)
timeFrame: Weeks 8, 12, 24, 36, 48, 60, 72, 84, 96
Secondary outcomes:
measure: Maximum Plasma Concentration (Cmax) of Eteplirsen
timeFrame: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)
measure: Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen
timeFrame: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)
measure: Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma
timeFrame: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)
measure: Amount of Drug Eliminated in Urine
timeFrame: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level)
Other outcomes:
Locations:
city: Gent
country: Belgium
facility: Universitair ziekenhuis Gent
city: Paris
country: France
facility: Armand-Trousseau Hospital
city: Roma
country: Italy
facility: Site Fondazione Policlinico Universitario Agostino Gemelli
city: London
country: United Kingdom
facility: UCL Great Ormond Street Institute of Child Health
Study officials:
affiliation: Sarepta Therapeutics, Inc.
name: Medical Director
role: STUDY_CHAIR
References: