Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping

• Register: ClinicalTrials.gov
• Last refreshed on: 19 April 2021
• Main ID: NCT03218995
• Date of registration: 09/07/2017
• Prospective Registration: Yes
• Primary sponsor: Sarepta Therapeutics, Inc.
• Public title: Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
• Scientific title: An Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
• Date of first enrolment: August 16, 2017
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03218995
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Belgium, France, Germany, Italy, United Kingdom

Contacts

• Name: Medical Director
• Affiliation: Sarepta Therapeutics, Inc.

Key inclusion & exclusion 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 patient's participation



Age minimum: 6 Months
Age maximum: 48 Months
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Eteplirsen

Primary Outcome(s)

Abnormal changes from baseline or worsening of physical examination findings [Time Frame: Change from Baseline]

Abnormal changes from baseline or worsening of vital signs [Time Frame: Change from Baseline]

Abnormal changes from baseline or clinically significant worsening of clinical safety laboratory abnormalities (hematology, chemistry, coagulation, and urinalysis) [Time Frame: Change from Baseline]

Abnormal changes from baseline or clinically significant worsening of electrocardiogram (ECG) and echocardiogram (ECHO) [Time Frame: Change from Baseline]

Incidence of adverse events [Time Frame: Up to 96 Weeks]

Secondary Outcome(s)

Clearance (CL) [Time Frame: 24 Weeks]

Apparent volume of distribution at steady state (Vss) [Time Frame: 24 Weeks]

Maximum plasma concentration [Time Frame: 24 Weeks]

Amount of drug eliminated in urine (Ae%) [Time Frame: 24 Weeks]

Elimination half-life (t½) [Time Frame: 24 Weeks]

Area under the concentration-time curve (AUC) [Time Frame: 24 Weeks]

Time of Cmax (Tmax) [Time Frame: 24 Weeks]

Secondary ID(s)

4658-102

Source(s) of Monetary Support

Please refer to primary and secondary sponsors