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

Launched by NMD PHARMA A/S · Mar 29, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called NMD670 for adults with Type 3 Spinal Muscular Atrophy (SMA), a condition that affects muscle strength and movement. The main goal is to see how well NMD670 works, how safe it is, and how it behaves in the body. The trial is currently looking for participants who can walk at least 50 meters without help and have a confirmed diagnosis of Type 3 SMA, including specific genetic markers. Eligible participants should be between 18 and 65 years old and have a body mass index (BMI) between 19 and 35.

If you join the trial, you will receive the NMD670 treatment and be monitored by healthcare professionals to ensure your safety and track any changes in your condition. It's important to note that certain health issues, previous surgeries, or treatments may exclude you from participating. This trial aims to provide valuable information that could help improve treatment options for those with SMA in the future.

Gender

ALL

Eligibility criteria

• 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.