An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
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|ClinicalTrials.gov Identifier: NCT03675126|
Recruitment Status : Not yet recruiting
First Posted : September 18, 2018
Last Update Posted : November 26, 2018
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Duchenne||Drug: SRP-5051||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051|
|Estimated Study Start Date :||November 30, 2018|
|Estimated Primary Completion Date :||November 1, 2020|
|Estimated Study Completion Date :||November 1, 2020|
Participants to receive SRP-5051 via intravenous (IV) infusion. Dosage and frequency will be determined from the safety profile of other ongoing SRP-5051 (NCT03375255) study.
SRP-5051 administered as an IV infusion.
- Number of participants with adverse events (AEs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]An AE is any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of the study drug, whether or not considered related to the study drug.
- Maximum plasma concentration (Cmax) of SRP-5051 [ Time Frame: End of infusion ]Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
- Area under the plasma concentration versus time curve (AUC) of SRP-5051 [ Time Frame: Pre-dose, end of infusion, 6 hours post-dose ]Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
- Number of participants with clinically relevant abnormalities, as assessed by vital sign measurements, physical examination findings, clinical laboratory tests and electrocardiograms (ECGs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]*A clinically relevant abnormality is an abnormality confirmed by repeat testing that is changed sufficiently from screening/baseline so that, in the judgment of the Investigator, a change in management is warranted.
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): NCT03675126
|Contact: Medical information||+1 888 727 firstname.lastname@example.org|
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|