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Evaluating NMD670 for treating ambulatory adults with Type 3 Spinal Muscular Atrophy

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

PHASE2 · NMD Pharma A/S · NCT05794139

This study is testing a new drug called NMD670 to see if it can help adults with Type 3 Spinal Muscular Atrophy who can walk on their own feel better and improve their condition.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment54 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorNMD Pharma A/S (industry)
Locations29 sites (Los Angeles, California and 28 other locations)
Trial IDNCT05794139 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety, efficacy, tolerability, and pharmacokinetics of NMD670 in ambulatory adults diagnosed with Type 3 Spinal Muscular Atrophy (SMA). Participants must be able to walk at least 50 meters without assistance and have a confirmed genetic diagnosis of SMA. The study will compare the effects of NMD670 against a placebo to determine its potential benefits for this patient population.

Who should consider this trial

Good fit: Ideal candidates are ambulatory adults with a clinical diagnosis of Type 3 SMA and specific genetic confirmations.

Not a fit: Patients who are non-ambulatory or do not meet the genetic criteria for Type 3 SMA may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve mobility and quality of life for patients with Type 3 Spinal Muscular Atrophy.

How similar studies have performed: Other studies targeting neuromuscular conditions have shown promise, but this specific approach with NMD670 is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Participants with a clinical diagnosis of Type 3 SMA.
2. Participants who are ambulatory, defined as being able to walk at least 50 metres without walking aids at screening during the 6-minute walk test.
3. Participant with genetic confirmation of diagnosis (e.g., homozygous deletion or compound heterozygous deletion and mutation of survival of motor neuron 1 gene \[SMN1\])
4. Participant with 3 to 5 copies of survival of motor neuron 2 gene \[SMN2\].
5. Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
6. Participant is male or female.
7. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
8. Participant is capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.

Exclusion Criteria:

1. Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
2. Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
3. Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
4. Participants received treatment with an investigational medical product (IMP) within 30 days (or 5 half-lives of the medication, whichever is longer) prior to Day 1.
5. Participants with history of poor compliance with relevant SMA therapy.

Where this trial is running

Los Angeles, California and 28 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Spinal Muscular Atrophy, Transmission Enhancer, Neuromuscular Junction Transmission, ClC-1

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.