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Becker Muscular Dystrophy - ClinicalTrials.gov

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Last Updated: 03 August 2022

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A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy

Approximately 48 adults and 18 adolescents with Becker muscular dystrophy are expected to enroll in this study. In a 1:1 ratio, approximately 48 adult participants will randomly assign to Cohort 1 or Cohort 2 in a 1:1 ratio, and then each cohort will be randomly assigned to either EDG-5506 or placebo in a 3:1 ratio. After Cohort 2, participants will be randomly assigned in a 3:1 ratio to either EDG-5506 or placebo and will enroll after Cohort 2. After Cohort 5, the Cohort 5 will randomize an additional 9 participants in a 2:1 ratio to either EDG-5506 or placebo.

Source link: https://clinicaltrials.gov/ct2/show/NCT05291091


A PHASE 1B MULTICENTER, OPEN-LABEL, SINGLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND TOLERABILITY OF PF-06939926 IN AMBULATORY AND NON-AMBULATORY SUBJECTS WITH DUCHENNE MUSCULAR DYSTROPHY

This is the first-in-human/first-in-patient, multi-center, open-label, non-randomized, growing dose, safety, and tolerability study of a single intravenous injection of PF-06939926 in ambulatory and non-ambulatory subjects with Duchenne muscular dystrophy. Enrollment will be staggered within and between two estimated dose-levels, as well as a formal examination by an external data monitoring committee prior to dose change in order to minimize unanticipated risks to subject safety.

Source link: https://clinicaltrials.gov/ct2/show/NCT03362502

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions