Find Spinal Muscular Atrophy Type 3 Clinical Trials Near You
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
Status: Active_not_recruiting
Location: See all (25) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:
• 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.
Locations
United States
California
UCLA David Geffen School Of Medicine - Neurology
Los Angeles
Stanford University Medical Center
Palo Alto
Kansas
University of Kansas Medical Center
Kansas City
Maryland
The Johns Hopkins Medicine, Spinal Muscular Atrophy Center
Baltimore
Missouri
Roy Blunt NextGen Precision Health Institute
Columbia
Washington University School of Medicine
St Louis
North Carolina
Rare Disease Research - Raleigh-Durham
Hillsborough
Ohio
The Ohio State University Wexner Medical Center
Columbus
Texas
Neurology Rare Disease Center
Denton
Other Locations
Belgium
UZ Leuven - Neurochirurgie Campus Gasthuisberg
Leuven
CHR de la Citadelle - Neurologie
Liège
Canada
Heritage Medical Research Clinic
Calgary
Genge Partners Inc.
Montreal
Denmark
Aarhus Universitetshospital, Neurologisk Afdeling
Aarhus
Rigshospitalet - Neurologisk Afdeling
Copenhagen
Germany
Charite - Campus Virchow-Klinikum (CVK)
Berlin
Universitätsklinikum Essen - Klinik Für Neurologie
Essen
Italy
Istituto Giannina Gaslini, IRCCS
Genova
Istituto Neurologico C. Besta, Fondazione IRCCS
Milan
Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda
Milan
AOU Città della Salute e della Scienza di Torino
Torino
Netherlands
Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology