Active — Not RecruitingPhase 2

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

United States54 participantsStarted 2023-09-21

Plain-language summary

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

Who can participate

Age range18 Years – 75 Years

SexALL

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Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Change from baseline in 6 minute walk test (6MWT) total distance versus placebo

Timeframe: Baseline to day 21

Trial details

NCT IDNCT05794139

SponsorNMD Pharma A/S

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-05

View on ClinicalTrials.gov More Spinal Muscular Atrophy trials