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
Status: Recruiting
Location: See all (7) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 4
Maximum Age: 10
Healthy Volunteers: f
View:
• Age 4 to 10
• Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping
• Able to walk
• Not requiring assistance from a ventilator to breathe
• Currently on consistent doses of steroid treatment for the last 12 weeks
Locations
Other Locations
Italy
Fondazione Serena Onlus - Centro Clinico NeMO Milano
RECRUITING
Milan
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
RECRUITING
Rome
Netherlands
Leids Universitair Medisch Centrum
RECRUITING
Leiden
Spain
Hospital Sant Joan de Deu
RECRUITING
Barcelona
Hospital Viamed Santa Angela De la Cruz
RECRUITING
Seville
Turkey
Yeditepe University Kosuyolu Hospital
RECRUITING
Istanbul
United Kingdom
Great Ormond Street Hospital NHS Foundation Trust
RECRUITING
London
Time Frame
Start Date: 2024-01-03
Estimated Completion Date: 2026-09-30
Participants
Target number of participants: 18
Treatments
Experimental: Cohort 1A
Cohort 1A will consist of both a single ascending dose (SAD) part and a multiple ascending dose (MAD). BMN 351 will be administered once every 2 weeks during the SAD portion of the study for up to 8 weeks and once weekly during the MAD portion for up to 89 weeks.
Experimental: Cohort 1B
BMN 351 low dose will be administered once weekly for up to 97 weeks
Experimental: Cohort 2
BMN 351 medium dose will be administered once weekly for up to 73 weeks
Experimental: Cohort 3
BMN 351 high dose will be administered once weekly for up to 48 weeks
Related Therapeutic Areas
Sponsors
Leads: BioMarin Pharmaceutical