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

Study Identifier:
NMD670-02-0001
ClinicalTrials.gov Identifier:
NCT05794139
EudraCT Identifier:
2022-002301-24
EU CT Number:
N/A
Sponsor:
NMD Pharma A/S
Active, not recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: NMD670
  • Drug: Placebo
Date
Sep 2023 - May 2026
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 18 - 75 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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.
Exclusion Criteria
  • 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.

Protocol 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

Trial Locations

Location
Status
Location
UCLA David Geffen School Of Medicine - Neurology
Los Angeles, California, United States, 90095
Status
N/A
Location
Stanford University Medical Center
Palo Alto, California, United States, 94304
Status
N/A
Location
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Status
N/A
Location
The Johns Hopkins Medicine, Spinal Muscular Atrophy Center
Baltimore, Maryland, United States, 21287
Status
N/A
Location
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri, United States, 65212
Status
N/A
Location
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Status
N/A
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