Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
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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. |
ClinicalTrials.gov Identifier: NCT03218995 |
Recruitment Status
:
Recruiting
First Posted
: July 17, 2017
Last Update Posted
: November 1, 2017
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: Eteplirsen | Phase 2 |
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 |

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:
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- Incidence of adverse events [ Time Frame: Up to 96 Weeks ]
- 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 worsening of vital signs [ Time Frame: Change from Baseline ]
- Abnormal changes from baseline or worsening of physical examination findings [ Time Frame: Change from Baseline ]
- Abnormal changes from baseline or clinically significant worsening of electrocardiogram (ECG) and echocardiogram (ECHO) [ Time Frame: Change from Baseline ]
- Maximum plasma concentration [ Time Frame: 24 Weeks ]
- Time of Cmax (Tmax) [ Time Frame: 24 Weeks ]
- Area under the concentration-time curve (AUC) [ Time Frame: 24 Weeks ]
- Apparent volume of distribution at steady state (Vss) [ Time Frame: 24 Weeks ]
- Clearance (CL) [ Time Frame: 24 Weeks ]
- Elimination half-life (t½) [ Time Frame: 24 Weeks ]
- Amount of drug eliminated in urine (Ae%) [ Time Frame: 24 Weeks ]

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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

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 ClinicalTrials.gov identifier (NCT number): NCT03218995
Contact: Medical Information | 617.274.4000 | medinfo@sarepta.com |
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 |
Study Chair: | Catherine Stehman-Breen | Sarepta Therapeutics |
Responsible Party: | Sarepta Therapeutics |
ClinicalTrials.gov Identifier: | NCT03218995 History of Changes |
Other Study ID Numbers: |
4658-102 |
First Posted: | July 17, 2017 Key Record Dates |
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 |