Clinical Trials Logo
  1. Home
  2. Duchenne Muscular Dystrophy
  3. NCT06280209

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

Official title:
A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy

Clinical Trial Summary

The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.

Clinical Trial Description

This is Phase 1/2, open-label, multi-center study consisting of 2 parts to evaluate the safety and tolerability of BMN 351 at escalating doses in participants with Duchenne Muscular Dystrophy (DMD) with genetic mutations amenable to exon 51 skipping. Participants will be assigned to one of three groups called cohorts (Cohort 1, 2 or 3). Cohort 1 participants are further divided into Cohort 1A and Cohort 1B. In Cohort 1A, 3 participants will receive increasing doses once every 2 weeks with a visit to assess safety measures collected the week after dosing prior to escalating doses of BMN 351. In part 2, the participants in cohort 1A will transition to once weekly dosing. The participants in Cohort 1B, 2, and 3 will initiate low, medium, and high doses of BMN 351 and continue once weekly dosing at that same dose. The study will enroll approximately 18 participants. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06280209
Study type Interventional
Source BioMarin Pharmaceutical
Contact
Status Recruiting
Phase Phase 1/Phase 2
Start date January 3, 2024
Completion date December 2025
View Details
See also
  Status Clinical Trial Phase
Completed NCT05575648 - Dual Task in Duchenne Muscular Dystrophy N/A
Terminated NCT03907072 - Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy Phase 2/Phase 3
Not yet recruiting NCT06450639 - An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy Phase 2
Completed NCT04335942 - Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor N/A
Active, not recruiting NCT04906460 - Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) Phase 1/Phase 2
Active, not recruiting NCT02500381 - Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) Phase 3
Enrolling by invitation NCT05967351 - A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study Phase 3
Recruiting NCT03067831 - Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy Phase 1/Phase 2
Recruiting NCT01834040 - Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy Phase 1/Phase 2
Completed NCT02246478 - A Study of TAS-205 for Duchenne Muscular Dystrophy Phase 1
Active, not recruiting NCT01772043 - Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping N/A
Terminated NCT01168908 - Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Phase 2
Completed NCT00758225 - Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) Phase 2
Completed NCT03680365 - Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
Recruiting NCT03513367 - The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
Recruiting NCT05712447 - Duchenne Muscular Dystrophy Video Assessment Registry
Recruiting NCT01484678 - Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Completed NCT03319030 - Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
Terminated NCT01753804 - A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. N/A
Completed NCT02530905 - Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients Phase 1