NCT05794139

Brief 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

Trial Health

62
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
54

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2023

Geographic Reach
8 countries

25 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 15, 2023

Completed
19 days until next milestone

First Posted

Study publicly available on registry

April 3, 2023

Completed
6 months until next milestone

Study Start

First participant enrolled

September 21, 2023

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2026

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2026

Completed
Last Updated

April 13, 2026

Status Verified

October 1, 2025

Enrollment Period

2.6 years

First QC Date

March 15, 2023

Last Update Submit

April 10, 2026

Conditions

Spinal Muscular Atrophy

Keywords

Transmission EnhancerNeuromuscular Junction TransmissionClC-1

Outcome Measures

Primary Outcomes (1)

  • Change from baseline in 6 minute walk test (6MWT) total distance versus placebo

    Distance walked (meters)

    Baseline to day 21

Secondary Outcomes (13)

  • Change from baseline in muscle strength versus placebo

    Baseline to day 21

  • Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo

    Baseline to day 21

  • Change from baseline in Revised Hammersmith Scale (RHS) versus placebo

    Baseline to day 21

  • Change from baseline in jitter versus placebo

    Baseline to day 21

  • Change from baseline in blocking versus placebo

    Baseline to day 21

  • +8 more secondary outcomes

Study Arms (2)

Cohort 1

EXPERIMENTAL

Experimental drug followed by placebo

Drug: NMD670Drug: Placebo

Cohort 2

EXPERIMENTAL

Placebo followed by experimental drug

Drug: NMD670Drug: Placebo

Interventions

NMD670DRUG

Tablets

Cohort 1Cohort 2
PlaceboDRUG

Tablets

Cohort 1Cohort 2

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
  • Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
  • Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
  • 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.
  • Participants with history of poor compliance with relevant SMA therapy.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (25)

UCLA David Geffen School Of Medicine - Neurology

Los Angeles, California, 90095, United States

Location

Stanford University Medical Center

Palo Alto, California, 94304, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

The Johns Hopkins Medicine, Spinal Muscular Atrophy Center

Baltimore, Maryland, 21287, United States

Location

Roy Blunt NextGen Precision Health Institute

Columbia, Missouri, 65212, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Rare Disease Research - Raleigh-Durham

Hillsborough, North Carolina, 27278, United States

Location

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

Neurology Rare Disease Center

Denton, Texas, 76208, United States

Location

UZ Leuven - Neurochirurgie Campus Gasthuisberg

Leuven, Belgium

Location

CHR de la Citadelle - Neurologie

Liège, Belgium

Location

Heritage Medical Research Clinic

Calgary, Canada

Location

Genge Partners Inc.

Montreal, Canada

Location

Aarhus Universitetshospital, Neurologisk Afdeling

Aarhus, Denmark

Location

Rigshospitalet - Neurologisk Afdeling

Copenhagen, Denmark

Location

Charite - Campus Virchow-Klinikum (CVK)

Berlin, Germany

Location

Universitätsklinikum Essen - Klinik Für Neurologie

Essen, Germany

Location

Istituto Giannina Gaslini, IRCCS

Genova, Italy

Location

Istituto Neurologico C. Besta, Fondazione IRCCS

Milan, Italy

Location

Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda

Milan, Italy

Location

AOU Città della Salute e della Scienza di Torino

Torino, Italy

Location

Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology

Utrecht, Netherlands

Location

Hospital Universitari Vall D Hebron

Barcelona, Spain

Location

Hospital Materno Infantil La Paz

Madrid, Spain

Location

Hospital Universitario y Politécnico La Fe

Valencia, Spain

Location

Related Publications (1)

  • Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025.

    PMID: 41224659DERIVED

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Model Details: 2-way crossover
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 15, 2023

First Posted

April 3, 2023

Study Start

September 21, 2023

Primary Completion

May 1, 2026

Study Completion

May 1, 2026

Last Updated

April 13, 2026

Record last verified: 2025-10

Data Sharing

IPD Sharing
Will not share

Locations

DE(2)IT(4)DK(2)ES(3)BE(2)CA(2)US(9)NL(1)