A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)
|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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03375255|
Recruitment Status : Completed
First Posted : December 18, 2017
Last Update Posted : August 28, 2019
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Duchenne||Drug: SRP-5051||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment|
|Actual Study Start Date :||February 5, 2018|
|Actual Primary Completion Date :||August 19, 2019|
|Actual Study Completion Date :||August 19, 2019|
Patients will be sequentially assigned to receive 1 of the 5 escalating dose levels of SRP-5051 on Day 1.
Patients who complete the study and continue to meet safety eligibility criteria will have the opportunity to enroll in an open-label extension study to continue to receive SRP-5051.
Single dose of SRP-5051 administered as an intravenous (IV) infusion.
- Number of Participants with Adverse Events (AEs) [ Time Frame: From signing of informed consent to 12 weeks after the last infusion of SRP-5051 (Up to 14 weeks) ]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: Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours) ]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, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours) ]Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
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): NCT03375255
|United States, California|
|Neuromuscular Research Center|
|Sacramento, California, United States, 95817|
|United States, Florida|
|NW FL Clinical Research Group, LLC|
|Gulf Breeze, Florida, United States, 32561|
|United States, Georgia|
|Rare Disease Research, LLC|
|Atlanta, Georgia, United States, 30318|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|United States, Kansas|
|University of Kansas Medical Center|
|Kansas City, Kansas, United States, 66160|
|United States, Pennsylvania|
|Children's Hospital of Pittsburgh of UPMC|
|Pittsburgh, Pennsylvania, United States, 15224|
|United States, Texas|
|Children's Medical Center Dallas|
|Dallas, Texas, United States, 75207|
|London Health Sciences Centre|
|London, Ontario, Canada, N6A 5W9|
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|