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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03218995
Recruitment Status : Recruiting
First Posted : July 17, 2017
Last Update Posted : July 25, 2019
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

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

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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 : March 15, 2021
Estimated Study Completion Date : March 15, 2021

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®

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 ]

Information from the National Library of Medicine

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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03218995

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Contact: Medical Information +1-888-727-3782

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Universitair ziekenhuis Gent Recruiting
Gent, Belgium, 9000
Contact: Elke De Vos    +32 93321954   
Principal Investigator: Nicolas Deconinck         
Armand-Trousseau Hospital Recruiting
Paris, France, 75012
Contact: Dominique Duchene    +33 171738258   
Principal Investigator: Laurent Servais         
Site Fondazione Policlinico Universitario Agostino Gemelli Recruiting
Roma, Italy
Contact: Maria Vizzino    +39 0630156330   
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   
Principal Investigator: Francesco Muntoni         
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
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Study Chair: Medical Director Sarepta Therapeutics, Inc.

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Responsible Party: Sarepta Therapeutics, Inc. Identifier: NCT03218995     History of Changes
Other Study ID Numbers: 4658-102
First Posted: July 17, 2017    Key Record Dates
Last Update Posted: July 25, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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, Inc.:
exon 51
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked