A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients with Spinal Muscular Atrophy After Gene Therapy

Status

Recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-504508-26-00

Acronym

BN44620

Enrollment

Registered

2024-02-08

Start date

2024-11-14

Completion date

Unknown

Last updated

2025-10-17

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

1.Change from baseline in the raw score of Bayley Scales of Infant and Toddler DevelopmentTM, Third Edition (BSID-III) Gross Motor Score at 72 weeks of risdiplam treatment in this population of SMA patients

Detailed description

Incidence and severity of adverse events, with severity determined according to the NCI CTCAE v5.0, Incidence and severity of serious adverse events, Incidence of treatment discontinuation due to adverse events

Interventions

DRUGRO7034067

DRUGEvrysdi 0.75 mg/mL powder for oral solution

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Secondary

Measure	Time frame
Incidence and severity of adverse events, with severity determined according to the NCI CTCAE v5.0, Incidence and severity of serious adverse events, Incidence of treatment discontinuation due to adverse events	—

Primary

Measure	Time frame
1.Change from baseline in the raw score of Bayley Scales of Infant and Toddler DevelopmentTM, Third Edition (BSID-III) Gross Motor Score at 72 weeks of risdiplam treatment in this population of SMA patients	—

Countries

Germany, Poland

Outcome results

None listed

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