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

Description

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

Conditions

Spinal Muscular Atrophy

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Study Overview

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Study Details

Study overview

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

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

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

Condition
Spinal Muscular Atrophy
Intervention / Treatment

-

Contacts and Locations

Los Angeles

UCLA David Geffen School Of Medicine - Neurology, Los Angeles, California, United States, 90095

Palo Alto

Stanford University Medical Center, Palo Alto, California, United States, 94304

Gainesville

UF Fixel Institute for Neurological Diseases, Gainesville, Florida, United States, 32608

Atlanta

Rare Disease Center, Atlanta, Georgia, United States, 30329

Kansas City

University of Kansas Medical Center, Kansas City, Kansas, United States, 66160

Columbia

Roy Blunt NextGen Precision Health Institute, Columbia, Missouri, United States, 65212

Saint Louis

Washington University School of Medicine, Saint Louis, Missouri, United States, 63110

Hillsborough

Rare Disease Research - Raleigh-Durham, Hillsborough, North Carolina, United States, 27278

Columbus

The Ohio State University Wexner Medical Center, Columbus, Ohio, United States, 43210

Denton

Neurology Rare Disease Center, Denton, Texas, United States, 76208

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • 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.

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

NMD Pharma A/S,

Study Record Dates

2025-07