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

  • Status
    Accepting Candidates
  • Age
    18 Years - 75 Years
  • Sexes
  • Healthy Volunteers


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


Full study title A Phase 2, Randomized, 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
Protocol number OCR43811 ID NCT05794139
Phase Phase 2


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.

Lead researcher

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  1. Step

    Contact the research team

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    Primary contact

  2. Step

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step


    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.