Open Actively Recruiting
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
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
- Participants who are 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.
- Participant with genetic confirmation of diagnosis (i.e., homozygous deletion 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 19-30 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.
- 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.
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Genetic and Rare Diseases
- UCLA Westwood