RecruitingPhase 2NCT05794139

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

Sponsor

NMD Pharma A/S

Enrollment

54 participants

Start Date

Sep 21, 2023

Study Type

INTERVENTIONAL

Conditions

Spinal Muscular Atrophy

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

Min Age: 18 YearsMax Age: 75 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a new oral drug called NMD670 in adults with Type 3 Spinal Muscular Atrophy (SMA), a genetic condition affecting the muscles. SMA is caused by mutations in the SMN1 gene, which reduces the production of a protein essential for motor nerve cells to survive. Type 3 SMA is less severe than Types 1 or 2 — people can walk — but still causes muscle weakness that can worsen over time and significantly impact daily life. NMD670 works by modifying how the related SMN2 gene produces protein, potentially increasing the amount of functional SMN protein available. Eligible participants are adults aged 18 to 75 with a confirmed genetic and clinical diagnosis of Type 3 SMA who can walk at least 50 metres unaided during a 6-minute walk test, have 3 to 5 copies of the SMN2 gene, and have a BMI between 19 and 35. People who have had prior spinal surgery causing mobility limitations, those with other major medical conditions, or those who have recently taken another investigational drug are not eligible. Participants will take NMD670 orally and be evaluated for safety, tolerability, and changes in motor function and walking ability. This research is significant because it explores a new therapeutic approach for SMA beyond the current treatments, with potential to benefit those living with progressive muscle weakness and loss of function.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGNMD670

Tablets

DRUGPlacebo

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

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NCT05794139

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