Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Spinal Muscular Atrophy Clinical Trial

— SYNAPSE-SMA  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05794139
• Other study ID #: NMD670-02-0001
• Secondary ID: 2022-002301-24
• Status: Recruiting
• Phase: Phase 2
• Start date: September 21, 2023
• Est. completion date: December 2024

Study information

• Verified date: April 2024
• Source: NMD Pharma A/S
• Contact: NMD Pharma A/S
• Phone: contact@nmdpharma.com
• Email: contact[@]nmdpharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 54
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

1. Participants with a clinical diagnosis of Type 3 SMA.

2. 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.

3. Participant with genetic confirmation of diagnosis (e.g., homozygous deletion or compound heterozygous deletion and mutation of survival of motor neuron 1 gene [SMN1])

4. Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].

5. Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).

6. Participant is male or female.

7. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

8. 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.

Exclusion Criteria:

1. Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.

2. Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).

3. Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study

4. 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.

5. Participants with history of poor compliance with relevant SMA therapy.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Intervention

Drug:
• NMD670
Tablets
• Placebo
Tablets

Locations

Country	Name	City	State
Belgium	UZ Leuven - Neurochirurgie Campus Gasthuisberg	Leuven
Belgium	CHR de la Citadelle - Neurologie	Liège
Canada	Heritage Medical Research Clinic	Calgary
Canada	Genge Partners Inc.	Montréal
Denmark	Aarhus Universitetshospital, Neurologisk Afdeling	Aarhus
Denmark	Rigshospitalet - Neurologisk Afdeling	København
Germany	Charite - Campus Virchow-Klinikum (CVK)	Berlin
Germany	Universitätsklinikum Essen - Klinik Für Neurologie	Essen
Italy	Istituto Giannina Gaslini, IRCCS	Genova
Italy	Istituto Neurologico C. Besta, Fondazione IRCCS	Milano
Italy	Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda	Milano
Italy	Università degli studi di Pisa	Pisa
Italy	PU A. Gemelli, Università Cattolica del Sacro Cuore	Roma
Netherlands	Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology	Utrecht
Spain	Hospital Universitari Vall D Hebron	Barcelona
Spain	H. Donostia	Donostia
Spain	Hospital Materno Infantil La Paz	Madrid
Spain	Hospital Universitario y Politécnico La Fe	Valencia
United States	Rare Disease Center	Atlanta	Georgia
United States	Roy Blunt NextGen Precision Health Institute	Columbia	Missouri
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio
United States	UF Fixel Institute for Neurological Diseases	Gainesville	Florida
United States	Rare Disease Research - Raleigh-Durham	Hillsborough	North Carolina
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	UCLA David Geffen School Of Medicine - Neurology	Los Angeles	California
United States	Stanford University Medical Center	Palo Alto	California
United States	Washington University School of Medicine	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
NMD Pharma A/S

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  Denmark,  Germany,  Italy,  Netherlands,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in 6 minute walk test (6MWT) total distance versus placebo	Distance walked (meters)	Baseline to day 21
Secondary	Change from baseline in muscle strength versus placebo	Handgrip, knee flexor, elbow flexor, elbow extension and should abduction (Newton)	Baseline to day 21
Secondary	Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo	percentage change in distance walked in 6th minute compared to 1st minute	Baseline to day 21
Secondary	Change from baseline in Revised Hammersmith Scale (RHS) versus placebo	Total score. Scale goes from 0-69 and higher score indicates improvement of symptoms	Baseline to day 21
Secondary	Change from baseline in jitter versus placebo	Jitter (micro seconds) assessed with single fiber EMG	Baseline to day 21
Secondary	Change from baseline in blocking versus placebo	Blocking (%) assessed with single fiber EMG	Baseline to day 21
Secondary	Incidence of treatment emergent adverse events	Summarised per treatment	Over 21 days of dosing
Secondary	Incidence of serious treatment emergent adverse events	Summarised per treatment	Over 21 days of dosing
Secondary	Incidence of clinically significant abnormalities on physical examinations	Summarised per treatment	Over 21 days of dosing
Secondary	Incidence of clinically significant abnormalities on safety laboratory parameters	Summarised per treatment	Over 21 days of dosing
Secondary	Incidence of clinically significant vital signs abnormalities	Summarised per treatment	Over 21 days of dosing
Secondary	Incidence of clinically significant ECG abnormalities	Summarised per treatment	Over 21 days of dosing
Secondary	Incidence of Suicidal Ideation or Suicidal Behavior	Summarised per treatment	Over 21 days of dosing
Secondary	Incidence of clinically significant abnormalities on opthalmological examinations	Summarised per treatment	Over 21 days of dosing