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

Korábbi kezelés

• Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
• 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.

Más betegség

• Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
• Participants with history of poor compliance with relevant SMA therapy.

Laborértékek

• Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study

• 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.