A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients with Spinal Muscular Atrophy who Experienced a Plateau or Decline in Function After Gene Therapy

Status

Recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-505161-81-00

Acronym

BN44621

Enrollment

Registered

2024-02-27

Start date

2024-12-16

Completion date

Unknown

Last updated

2025-10-10

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

Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment in this population of SMA patients

Detailed description

1. Incidence and severity of adverse events, with severity determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE v5.0), 2. Incidence and severity of serious adverse events, 3. Incidence of treatment discontinuation due to adverse events

Interventions

DRUGEvrysdi 0.75 mg/mL powder for oral solution

DRUGRO7034067

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment in this population of SMA patients	—

Secondary

Measure	Time frame
1. Incidence and severity of adverse events, with severity determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE v5.0), 2. Incidence and severity of serious adverse events, 3. Incidence of treatment discontinuation due to adverse events	—

Countries

Germany, Poland

Outcome results

None listed

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