Longitudinal study of clinical outcome measures in adult patients with Spinal Muscular Atrophy (SMA) treated with Risdiplam.

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2022-501760-17-00

Acronym

version 4.0

Enrollment

Registered

2022-11-28

Start date

2023-06-13

Completion date

2024-10-02

Last updated

2023-04-24

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

The primary endpoint of this study is: The change in clinical and functional outcome measures in adult patients with SMA type 2 or 3 or patients with up to 4 SMN2-gene copies treated with Risdiplam from baseline to 24 months.

Detailed description

The secondary endpoints of this study are: Quantitative changes of biomarkers in blood (neurofilament light chain and neurofilament heavy chain) in adult patients with SMA type 2 or 3 or patients with up to 4 SMN2-gene copies treated with Risdiplam from baseline to 24 months, AND To assess the safety and tolerability of Risdiplam treatment.

Interventions

DRUGEvrysdi 0.75 mg/mL powder for oral solution

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
The primary endpoint of this study is: The change in clinical and functional outcome measures in adult patients with SMA type 2 or 3 or patients with up to 4 SMN2-gene copies treated with Risdiplam from baseline to 24 months.	—

Secondary

Measure	Time frame
The secondary endpoints of this study are: Quantitative changes of biomarkers in blood (neurofilament light chain and neurofilament heavy chain) in adult patients with SMA type 2 or 3 or patients with up to 4 SMN2-gene copies treated with Risdiplam from baseline to 24 months, AND To assess the safety and tolerability of Risdiplam treatment.	—

Countries

Belgium

Outcome results

None listed

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