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: Recruiting
Location: See all (29) 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
RECRUITING
Los Angeles
Stanford University Medical Center
RECRUITING
Palo Alto
Florida
UF Fixel Institute for Neurological Diseases
RECRUITING
Gainesville
Georgia
Rare Disease Center
RECRUITING
Atlanta
Kansas
University of Kansas Medical Center
RECRUITING
Kansas City
Missouri
Roy Blunt NextGen Precision Health Institute
RECRUITING
Columbia
Washington University School of Medicine
RECRUITING
St Louis
North Carolina
Rare Disease Research - Raleigh-Durham
RECRUITING
Hillsborough
Ohio
The Ohio State University Wexner Medical Center
RECRUITING
Columbus
Texas
Neurology Rare Disease Center
RECRUITING
Denton
Other Locations
Belgium
UZ Leuven - Neurochirurgie Campus Gasthuisberg
RECRUITING
Leuven
CHR de la Citadelle - Neurologie
RECRUITING
Liège
Canada
Heritage Medical Research Clinic
RECRUITING
Calgary
Genge Partners Inc.
RECRUITING
Montreal
Denmark
Aarhus Universitetshospital, Neurologisk Afdeling
RECRUITING
Aarhus
Rigshospitalet - Neurologisk Afdeling
RECRUITING
Copenhagen
Germany
Charite - Campus Virchow-Klinikum (CVK)
RECRUITING
Berlin
Universitätsklinikum Essen - Klinik Für Neurologie
RECRUITING
Essen
Italy
Istituto Giannina Gaslini, IRCCS
RECRUITING
Genova
Istituto Neurologico C. Besta, Fondazione IRCCS
RECRUITING
Milan
Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda
RECRUITING
Milan
Università degli studi di Pisa
RECRUITING
Pisa
PU A. Gemelli, Università Cattolica del Sacro Cuore
RECRUITING
Roma
AOU Città della Salute e della Scienza di Torino
RECRUITING
Torino
Netherlands
Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology
RECRUITING
Utrecht
Spain
Hospital Universitari Vall D Hebron
RECRUITING
Barcelona
H. Donostia
RECRUITING
Donostia / San Sebastian
Hospital Materno Infantil La Paz
RECRUITING
Madrid
Hospital Universitario y Politécnico La Fe
RECRUITING
Valencia
Contact Information
Primary
NMD Pharma A/S
contact@nmdpharma.com
contact@nmdpharma.com
Time Frame
Start Date: 2023-09-21
Estimated Completion Date: 2026-01
Participants
Target number of participants: 54
Treatments
Experimental: Cohort 1
Experimental drug followed by placebo
Experimental: Cohort 2
Placebo followed by experimental drug
Sponsors
Leads: NMD Pharma A/S

This content was sourced from clinicaltrials.gov