RECRUITING

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

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Conditions

Spinal Muscular AtrophyTransmission EnhancerNeuromuscular Junction TransmissionClC-1

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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

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

Quick Facts

Study Start:2023-09-21
Study Completion:2025-07
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05794139

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.

Ages Eligible for Study:18 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Participants with a clinical diagnosis of Type 3 SMA.
  2. 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. 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. 4. Participant with 3 to 5 copies of survival of motor neuron 2 gene \[SMN2\].
  5. 5. Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
  6. 6. Participant is male or female.
  7. 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. 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. 1. Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
  2. 2. Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
  3. 3. Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
  4. 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. 5. Participants with history of poor compliance with relevant SMA therapy.

Contacts and Locations

Study Contact

NMD Pharma A/S
CONTACT
contact@nmdpharma.com
contact@nmdpharma.com

Study Locations (Sites)

UCLA David Geffen School Of Medicine - Neurology
Los Angeles, California, 90095
United States
Stanford University Medical Center
Palo Alto, California, 94304
United States
UF Fixel Institute for Neurological Diseases
Gainesville, Florida, 32608
United States
Rare Disease Center
Atlanta, Georgia, 30329
United States
University of Kansas Medical Center
Kansas City, Kansas, 66160
United States
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri, 65212
United States
Washington University School of Medicine
Saint Louis, Missouri, 63110
United States
Rare Disease Research - Raleigh-Durham
Hillsborough, North Carolina, 27278
United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
United States
Neurology Rare Disease Center
Denton, Texas, 76208
United States

Collaborators and Investigators

Sponsor: NMD Pharma A/S

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-09-21
Study Completion Date2025-07

Study Record Updates

Study Start Date2023-09-21
Study Completion Date2025-07

Terms related to this study

Keywords Provided by Researchers

  • Transmission Enhancer
  • Neuromuscular Junction Transmission
  • ClC-1

Additional Relevant MeSH Terms

  • Spinal Muscular Atrophy