Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Completed · Phase 2 · Has a placebo group

Conditions studied: Spinal Muscular Atrophy

In brief

The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3

Key facts

Study ID

NCT05794139

Run by

NMD Pharma A/S

People needed

52

Starts

2023-09-21

Expected to finish

2026-05-18

Last updated by the study team

2026-06-03

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants with a clinical diagnosis of Type 3 SMA.
• 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.
• Participant with genetic confirmation of diagnosis (e.g., homozygous deletion or compound heterozygous deletion and mutation of survival of motor neuron 1 gene [SMN1])
• Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
• Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
• Participant is male or female.
• Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• 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.

You may not qualify if…

• Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
• Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
• Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
• 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.
• Participants with history of poor compliance with relevant SMA therapy.

Where it is running

• UCLA David Geffen School Of Medicine - Neurology — Los Angeles, California, United States
• Stanford University Medical Center — Palo Alto, California, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• The Johns Hopkins Medicine, Spinal Muscular Atrophy Center — Baltimore, Maryland, United States
• Roy Blunt NextGen Precision Health Institute — Columbia, Missouri, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• Rare Disease Research - Raleigh-Durham — Hillsborough, North Carolina, United States
• The Ohio State University Wexner Medical Center — Columbus, Ohio, United States
• Neurology Rare Disease Center — Denton, Texas, United States
• UZ Leuven - Neurochirurgie Campus Gasthuisberg — Leuven, Belgium
• CHR de la Citadelle - Neurologie — Liège, Belgium
• Heritage Medical Research Clinic — Calgary, Canada
• Genge Partners Inc. — Montreal, Canada
• Aarhus Universitetshospital, Neurologisk Afdeling — Aarhus, Denmark
• Rigshospitalet - Neurologisk Afdeling — Copenhagen, Denmark
• Charite - Campus Virchow-Klinikum (CVK) — Berlin, Germany
• Universitätsklinikum Essen - Klinik Für Neurologie — Essen, Germany
• Istituto Giannina Gaslini, IRCCS — Genova, Italy
• Istituto Neurologico C. Besta, Fondazione IRCCS — Milan, Italy
• Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda — Milan, Italy
• AOU Città della Salute e della Scienza di Torino — Torino, Italy
• Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology — Utrecht, Netherlands
• Hospital Universitari Vall D Hebron — Barcelona, Spain
• Hospital Materno Infantil La Paz — Madrid, Spain
• Hospital Universitario y Politécnico La Fe — Valencia, Spain

Full record on ClinicalTrials.gov

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