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A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT03985878
Recruitment Status : Not yet recruiting
First Posted : June 14, 2019
Last Update Posted : June 14, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Brief Summary:
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male DMD patients who have successfully completed the 96-week eteplirsen study: Study 4658-102 (NCT03218995)

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
Estimated Study Start Date : June 19, 2019
Estimated Primary Completion Date : November 2026
Estimated Study Completion Date : February 2027


Arm Intervention/treatment
Experimental: Eteplirsen
Patients will receive eteplirsen via intravenous (IV) infusions, once weekly, for up to 284 weeks.
Drug: Eteplirsen
Eteplirsen IV infusion once weekly.




Primary Outcome Measures :
  1. Incidence of Adverse Events (AEs) [ Time Frame: Up to 288 weeks ]
  2. Incidence of Death Due to Adverse Events [ Time Frame: Up to 288 weeks ]
  3. Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 288 weeks ]
    AESIs will be defined as any AE that is of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor is appropriate. AESIs will include infusion-related reactions, hypersensitivity, and renal events.



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 5 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient successfully completes 96 weeks of treatment in Study 4658-102.

Exclusion Criteria:

  • Patient has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the patient, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.

Other inclusion/exclusion criteria apply.


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 ClinicalTrials.gov identifier (NCT number): NCT03985878


Contacts
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Contact: Medical Information +1 888-727-3782 clinicaltrials@sarepta.com

Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.

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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03985878     History of Changes
Other Study ID Numbers: 4658-102-OLE
2019‐000337‐39 ( EudraCT Number )
First Posted: June 14, 2019    Key Record Dates
Last Update Posted: June 14, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Sarepta Therapeutics, Inc.:
DMD
Duchenne
Eteplirsen
Dystrophy
Dystrophin
Exon Skipping
Exon 51
Ambulatory
Pediatric

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