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

Study Description

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Duchenne Muscular Dystrophy	Drug: Eteplirsen	Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	12 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	An Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Actual Study Start Date :	August 16, 2017
Estimated Primary Completion Date :	April 2020
Estimated Study Completion Date :	April 2020

Arms and Interventions

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Arm	Intervention/treatment
Experimental: Experimental: Treated Group	Drug: Eteplirsen; Eteplirsen will be administered once a week by IV infusion for up to 96 weeks. The starting dose is 2 mg/kg eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg over the course of the dose-titration period.; Other Names: AVI-4658; EXONDYS 51®

Outcome Measures

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Primary Outcome Measures :

1. Incidence of adverse events [ Time Frame: Up to 96 Weeks ]
2. Abnormal changes from baseline or clinically significant worsening of clinical safety laboratory abnormalities (hematology, chemistry, coagulation, and urinalysis) [ Time Frame: Change from Baseline ]
3. Abnormal changes from baseline or worsening of vital signs [ Time Frame: Change from Baseline ]
4. Abnormal changes from baseline or worsening of physical examination findings [ Time Frame: Change from Baseline ]
5. Abnormal changes from baseline or clinically significant worsening of electrocardiogram (ECG) and echocardiogram (ECHO) [ Time Frame: Change from Baseline ]

Secondary Outcome Measures :

1. Maximum plasma concentration [ Time Frame: 24 Weeks ]
2. Time of Cmax (Tmax) [ Time Frame: 24 Weeks ]
3. Area under the concentration-time curve (AUC) [ Time Frame: 24 Weeks ]
4. Apparent volume of distribution at steady state (Vss) [ Time Frame: 24 Weeks ]
5. Clearance (CL) [ Time Frame: 24 Weeks ]
6. Elimination half-life (t½) [ Time Frame: 24 Weeks ]
7. Amount of drug eliminated in urine (Ae%) [ Time Frame: 24 Weeks ]

Eligibility Criteria

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Ages Eligible for Study:	6 Months to 48 Months (Child)
Sexes Eligible for Study:	Male
Accepts Healthy Volunteers:	No

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

Contacts and Locations

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Contacts

Contact: Medical Information	617.274.4000	medinfo@sarepta.com

Locations

Belgium
Universitair ziekenhuis Gent	Recruiting
Gent, Belgium, 9000
Contact: Elke De Vos +32 93321954 Elke.DeVos@uzgent.be
Principal Investigator: Nicolas Deconinck
France
Armand-Trousseau Hospital	Recruiting
Paris, France, 75012
Contact: Dominique Duchene +33 0171738313 d.duchene@institut-myologie.org
Principal Investigator: François Renard
Germany
Universitaetsklinikum Freiburg	Recruiting
Freiburg, Germany, 79106
Contact: Sabine Wider +49 76127043440 sabine.wider@uniklinik-freiburg.de
Principal Investigator: Janbernd Kirschner
Italy
Site Fondazione Policlinico Universitario Agostino Gemelli	Recruiting
Roma, Italy
Contact: Maria Vizzino +39 0630156330 Npi.clinicaltrials@rm.unicatt.it
Principal Investigator: Eugenio M Mercuri
United Kingdom
UCL Great Ormond Street Institute of Child Health	Recruiting
London, United Kingdom, WC1N 1EH
Contact: Manju Agarwal +44 79052758 m.agarwal@ucl.ac.uk
Principal Investigator: Francesco Muntoni

Sponsors and Collaborators

Sarepta Therapeutics

Investigators

Study Chair:	Catherine Stehman-Breen	Sarepta Therapeutics

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Sarepta Therapeutics
ClinicalTrials.gov Identifier:	NCT03218995 History of Changes
Other Study ID Numbers:	4658-102
First Posted:	July 17, 2017
Last Update Posted:	November 1, 2017
Last Verified:	October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	No

Studies a U.S. FDA-regulated Drug Product:		Yes
Studies a U.S. FDA-regulated Device Product:		No
Product Manufactured in and Exported from the U.S.:		Yes

Keywords provided by Sarepta Therapeutics:

DMD; Duchenne; Eteplirsen	dystrophy; dystrophin; exon 51

Additional relevant MeSH terms:

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases	Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked