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RECRUITINGPhase 2INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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

Who May Be Eligible (Plain English)

Who May Qualify: 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 willing to sign a consent form which includes compliance with the requirements and restrictions listed in the willing to sign a consent form form (ICF) and in the protocol. Who Should NOT Join This Trial: 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

NMD670

Tablets

DRUG

Placebo

Tablets

Locations (20)

UCLA David Geffen School Of Medicine - Neurology
Los Angeles, California, United States
Stanford University Medical Center
Palo Alto, California, United States
University of Colorado Neurology Clinic
Aurora, Colorado, United States
UF Fixel Institute for Neurological Diseases
Gainesville, Florida, United States
Rare Disease Center
Atlanta, Georgia, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
The Johns Hopkins Medicine, Spinal Muscular Atrophy Center
Baltimore, Maryland, United States
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri, United States
Washington University School of Medicine
St Louis, Missouri, United States
Rare Disease Research - Raleigh-Durham
Hillsborough, North Carolina, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States
Neurology Rare Disease Center
Denton, Texas, United States
UZ Leuven - Neurochirurgie Campus Gasthuisberg
Leuven, Belgium
CHR de la Citadelle - Neurologie
Liège, Belgium
Heritage Medical Research Clinic
Calgary, Canada
Genge Partners Inc.
Montreal, Canada
Aarhus Universitetshospital, Neurologisk Afdeling
Aarhus, Denmark
Rigshospitalet - Neurologisk Afdeling
Copenhagen, Denmark
Charite - Campus Virchow-Klinikum (CVK)
Berlin, Germany
Universitätsklinikum Essen - Klinik Für Neurologie
Essen, Germany