A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
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|ClinicalTrials.gov Identifier: NCT03985878|
Recruitment Status : Enrolling by invitation
First Posted : June 14, 2019
Last Update Posted : February 10, 2022
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Eteplirsen||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102|
|Actual Study Start Date :||June 26, 2019|
|Estimated Primary Completion Date :||November 30, 2026|
|Estimated Study Completion Date :||February 28, 2027|
Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks.
Eteplirsen IV infusion once weekly.
- Number of Participants with Adverse Events (AEs) [ Time Frame: Up to 288 weeks ]
- Number of Deaths Due to Adverse Events [ Time Frame: Up to 288 weeks ]
- Number of Participants with 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 findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia.
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
|Gent, Belgium, 9000|
|Hopital Trousseau, Bâtiment lemariey|
|Paris, France, 75021|
|Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile|
|Rome, Italy, 00168|
|UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre|
|London, England, United Kingdom, WC1N 1EH|
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|