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Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping

NCT ID: NCT03218995

Last Updated: 2021-12-09

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-08-16

Study Completion Date

2021-03-10

Brief Summary

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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.

Detailed Description

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Conditions

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Duchenne Muscular Dystrophy

Keywords

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DMD Duchenne Eteplirsen dystrophy dystrophin exon 51

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Eteplirsen

Eteplirsen will be administered once every 7 days by intravenous (IV) infusion starting on Day 1 for up to 96 weeks. The starting dose will be 2 milligrams/kilogram (mg/kg) eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants will continue to receive eteplirsen at 30 mg/kg for the duration of the study.

Group Type EXPERIMENTAL

Eteplirsen

Intervention Type DRUG

Infusion for intravenous use.

Interventions

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Eteplirsen

Infusion for intravenous use.

Intervention Type DRUG

Other Intervention Names

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AVI-4658 EXONDYS 51®

Eligibility Criteria

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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.
Minimum Eligible Age

6 Months

Maximum Eligible Age

48 Months

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Sarepta Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_CHAIR

Sarepta Therapeutics, Inc.

Locations

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Universitair ziekenhuis Gent

Ghent, , Belgium

Site Status

Armand-Trousseau Hospital

Paris, , France

Site Status

Site Fondazione Policlinico Universitario Agostino Gemelli

Roma, , Italy

Site Status

UCL Great Ormond Street Institute of Child Health

London, , United Kingdom

Site Status

Countries

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Germany Belgium France Italy United Kingdom

References

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Mercuri E, Seferian AM, Servais L, Deconinck N, Stevenson H, Ni X, Zhang W, East L, Yonren S, Muntoni F; 4658-102 Study Group. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping. Neuromuscul Disord. 2023 Jun;33(6):476-483. doi: 10.1016/j.nmd.2023.03.008. Epub 2023 Mar 24.

Reference Type DERIVED
PMID: 37207382 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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4658-102

Identifier Type: -

Identifier Source: org_study_id