An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab (ONYX)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-511654-42-00

Acronym

SRK-015-004

Enrollment

125

Registered

2024-03-25

Start date

2023-06-28

Completion date

Unknown

Last updated

2025-08-04

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

Conditions

Spinal Muscular Atrophy (SMA)

Brief summary

Incidence of treatment-emergent adverse event (TEAEs) and serious adverse event (SAEs) by severity

Detailed description

HFMSE total score at prespecified time points (excludes TOPAZ Cohort 1 patients), RULM total score at prespecified time points (excludes TOPAZ Cohort 1 patients), Number of WHO motor development milestones attained at prespecified time points (excludes TOPAZ Cohort 1 patients), RHS total score and results for 6-Minute Walk Test, 30-Second Sit-to-Stand, 10-Meter Walk/Run (from the RHS), and timed rise from floor (from the RHS) at prespecified time points (TOPAZ Cohort 1 patients only)

Interventions

DRUGAPITEGROMAB

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Incidence of treatment-emergent adverse event (TEAEs) and serious adverse event (SAEs) by severity	—

Secondary

Measure	Time frame
HFMSE total score at prespecified time points (excludes TOPAZ Cohort 1 patients), RULM total score at prespecified time points (excludes TOPAZ Cohort 1 patients), Number of WHO motor development milestones attained at prespecified time points (excludes TOPAZ Cohort 1 patients), RHS total score and results for 6-Minute Walk Test, 30-Second Sit-to-Stand, 10-Meter Walk/Run (from the RHS), and timed rise from floor (from the RHS) at prespecified time points (TOPAZ Cohort 1 patients only)	—

Measure

Time frame

—

Countries

Belgium, France, Germany, Italy, Netherlands, Poland, Spain

Outcome results

None listed

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