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Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
Sponsor: NMD Pharma A/S
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
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
Key Details
Gender
All
Age Range
18 Years - 75 Years
Study Type
INTERVENTIONAL
Enrollment
54
Start Date
2023-09-21
Completion Date
2026-05
Last Updated
2026-02-13
Healthy Volunteers
No
Conditions
Interventions
NMD670
Tablets
Placebo
Tablets
Locations (31)
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
Istituto Giannina Gaslini, IRCCS
Genova, Italy
Istituto Neurologico C. Besta, Fondazione IRCCS
Milan, Italy
Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda
Milan, Italy
Università degli studi di Pisa
Pisa, Italy
PU A. Gemelli, Università Cattolica del Sacro Cuore
Roma, Italy
AOU Città della Salute e della Scienza di Torino
Torino, Italy
Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology
Utrecht, Netherlands
Hospital Universitari Vall D Hebron
Barcelona, Spain
H. Donostia
Donostia / San Sebastian, Spain
Hospital Materno Infantil La Paz
Madrid, Spain
Hospital Universitario y Politécnico La Fe
Valencia, Spain