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A TWO-PART, OPEN-LABEL SYSTEMIC GENE DELIVERY STUDY TO EVALUATE THE SAFETY AND EXPRESSION OF RO7494222 (SRP‑9001) IN SUBJECTS UNDER THE AGE OF FOUR WITH DUCHENNE MUSCULAR DYSTROPHY

Status
Suspended
Phases
Phase 2
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-509901-57-00
Acronym
BN43881
Enrollment
15
Registered
2024-08-06
Start date
2023-10-26
Completion date
Unknown
Last updated
2025-03-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Brief summary

Incidence of treatment-emergent adverse events, Incidence of serious adverse events, Incidence of adverse events of special interest, Clinically significant changes in vital signs and physical examination findings, Clinically significant changes in safety laboratory assessments, ECGs, and ECHOs

Detailed description

Change in quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 as measured by Western blot

Interventions

Sponsors

F. Hoffmann-La Roche AG
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
Male
Age
0 Years to 17 Years

Design outcomes

Primary

MeasureTime frame
Incidence of treatment-emergent adverse events, Incidence of serious adverse events, Incidence of adverse events of special interest, Clinically significant changes in vital signs and physical examination findings, Clinically significant changes in safety laboratory assessments, ECGs, and ECHOs

Secondary

MeasureTime frame
Change in quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 as measured by Western blot

Countries

Belgium, France, Germany, Italy, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026