A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 spinal muscular atrophy

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-516321-31-00

Acronym

NMD670-02-0001

Enrollment

Registered

2024-10-01

Start date

2023-07-07

Completion date

Unknown

Last updated

2025-10-24

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

Conditions

Type 3 spinal muscular atrophy

Brief summary

Change from baseline in 6MWT (total distance) after 21-day dosing

Detailed description

Change from baseline in individual muscle groups maximum strength (measured with a dynamometer) after 21-day dosing. Change from baseline after 21-day dosing in: -6MWT (fatigue index), -revised Hammersmith scale score. Change from baseline in jitter and blocking measured via sfEMG after 21- day dosing. AEs, physical examinations, clinical laboratory parameters, vital signs, ECG, C-SSRS.

Interventions

DRUGPlacebo 100 mg tablet

DRUGNMD670

DRUGPlacebo 300 mg tablet

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Change from baseline in 6MWT (total distance) after 21-day dosing	—

Secondary

Measure	Time frame
Change from baseline in individual muscle groups maximum strength (measured with a dynamometer) after 21-day dosing. Change from baseline after 21-day dosing in: -6MWT (fatigue index), -revised Hammersmith scale score. Change from baseline in jitter and blocking measured via sfEMG after 21- day dosing. AEs, physical examinations, clinical laboratory parameters, vital signs, ECG, C-SSRS.	—

Countries

Belgium, Denmark, Germany, Italy, Netherlands, Spain

Outcome results

None listed

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