Summary

Eligibility
for people ages 18-75 (full criteria)
Location
at UCLA
Dates
study started
completion around

Description

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

Official Title

A Phase 2, Randomised, 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

Keywords

Spinal Muscular Atrophy, Transmission Enhancer, Neuromuscular Junction Transmission, ClC-1, Muscular Atrophy, Atrophy, NMD670

Eligibility

You can join if…

Open to people ages 18-75

  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.

You CAN'T join if...

  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.

Locations

  • UCLA David Geffen School Of Medicine - Neurology accepting new patients
    Los Angeles California 90095 United States
  • Stanford University Medical Center accepting new patients
    Palo Alto California 94304 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
NMD Pharma A/S
ID
NCT05794139
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 54 study participants
Last Updated