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New drug shows promise for improving walking in adults with spinal muscular atrophy

NCT ID NCT05794139

Symptom relief Sponsor: NMD Pharma A/S Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested an experimental drug called NMD670 in 52 adults with type 3 spinal muscular atrophy who can still walk. The goal was to see if the drug helps them walk farther in 6 minutes and improves muscle strength compared to a placebo. The trial is now complete, and results will show whether NMD670 can ease symptoms of this muscle-weakening condition.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

52 people

The number who actually took part.

Started

Sep 2023

Finished

May 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy spinal muscular atrophy, type III

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Spinal muscular atrophy

Contacts and locations

Locations

  • AOU Città della Salute e della Scienza di Torino

    Torino, Italy

  • Aarhus Universitetshospital, Neurologisk Afdeling

    Aarhus, Denmark

  • CHR de la Citadelle - Neurologie

    Liège, Belgium

  • Charite - Campus Virchow-Klinikum (CVK)

    Berlin, Germany

  • Genge Partners Inc.

    Montreal, Canada

  • Heritage Medical Research Clinic

    Calgary, Canada

  • Hospital Materno Infantil La Paz

    Madrid, Spain

  • Hospital Universitari Vall D Hebron

    Barcelona, Spain

  • Hospital Universitario y Politécnico La Fe

    Valencia, Spain

  • Istituto Giannina Gaslini, IRCCS

    Genova, Italy

  • Istituto Neurologico C. Besta, Fondazione IRCCS

    Milan, Italy

  • Neurology Rare Disease Center

    Denton, Texas, 76208, United States

  • Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda

    Milan, Italy

  • Rare Disease Research - Raleigh-Durham

    Hillsborough, North Carolina, 27278, United States

  • Rigshospitalet - Neurologisk Afdeling

    Copenhagen, Denmark

  • Roy Blunt NextGen Precision Health Institute

    Columbia, Missouri, 65212, United States

  • Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • The Johns Hopkins Medicine, Spinal Muscular Atrophy Center

    Baltimore, Maryland, 21287, United States

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210, United States

  • UCLA David Geffen School Of Medicine - Neurology

    Los Angeles, California, 90095, United States

  • UZ Leuven - Neurochirurgie Campus Gasthuisberg

    Leuven, Belgium

  • Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology

    Utrecht, Netherlands

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • Universitätsklinikum Essen - Klinik Für Neurologie

    Essen, Germany

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.