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Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)

Primary Purpose

Spinal Muscular Atrophy

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
NMD670
Placebo
Sponsored by
NMD Pharma A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinal Muscular Atrophy focused on measuring Transmission Enhancer, Neuromuscular Junction Transmission, ClC-1

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Participants who are 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. Participant with genetic confirmation of diagnosis (i.e., homozygous deletion 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 19-30 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. Exclusion Criteria: 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.

Sites / Locations

  • UF Fixel Institute for Neurological DiseasesRecruiting
  • University of Kansas Medical CenterRecruiting
  • Roy Blunt NextGen Precision Health InstituteRecruiting
  • Washington University School of MedicineRecruiting
  • The Ohio State University Wexner Medical CenterRecruiting
  • UZ Leuven - Neurochirurgie Campus GasthuisbergRecruiting
  • CHR de la Citadelle - NeurologieRecruiting
  • Aarhus Universitetshospital, Neurologisk AfdelingRecruiting
  • Rigshospitalet - Neurologisk AfdelingRecruiting
  • Charite - Campus Virchow-Klinikum (CVK)Recruiting
  • Universitätsklinikum Essen - Klinik Für NeurologieRecruiting
  • Istituto Neurologico C. Besta, Fondazione IRCCSRecruiting
  • Ospedale Niguarda, ASST Grande Ospedale Metropolitano NiguardaRecruiting
  • Universitair Medisch Centrum Utrecht, locatie Academisch Zie - NeurologyRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Cohort 1

Cohort 2

Arm Description

Experimental drug followed by placebo

Placebo followed by experimental drug

Outcomes

Primary Outcome Measures

Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
Distance walked (meters)

Secondary Outcome Measures

Change from baseline in muscle strength versus placebo
Handgrip, knee flexor, elbow flexor, elbow extension and should abduction (Newton)
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
Change from baseline in Revised Hammersmith Scale (RHS) versus placebo
Total score. Scale goes from 0-69 and higher score indicates improvement of symptoms
Change from baseline in time to dropout in the endurance shuttle 9-hole peg test (ESNHPT) versus placebo
time to dropout (seconds)
Change from baseline in proportion of patients that drops out in the endurance shuttle 9-hole peg test (ESNHPT) versus placebo
Proportion of patient dropout (%)
Change from baseline in jitter versus placebo
Jitter (micro seconds) assessed with single fiber EMG
Change from baseline in blocking versus placebo
Blocking (%) assessed with single fiber EMG
Incidence of treatment emergent adverse events
Summarised per treatment
Incidence of serious treatment emergent adverse events
Summarised per treatment
Incidence of clinically significant abnormalities on physical examinations
Summarised per treatment
Incidence of clinically significant abnormalities on safety laboratory parameters
Summarised per treatment
Incidence of clinically significant vital signs abnormalities
Summarised per treatment
Incidence of clinically significant ECG abnormalities
Summarised per treatment
Incidence of Suicidal Ideation or Suicidal Behavior
Summarised per treatment

Full Information

First Posted
March 15, 2023
Last Updated
September 29, 2023
Sponsor
NMD Pharma A/S
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1. Study Identification

Unique Protocol Identification Number
NCT05794139
Brief Title
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 21, 2023 (Actual)
Primary Completion Date
September 2024 (Anticipated)
Study Completion Date
September 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NMD Pharma A/S

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinal Muscular Atrophy
Keywords
Transmission Enhancer, Neuromuscular Junction Transmission, ClC-1

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Model Description
2-way crossover
Masking
ParticipantInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
54 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
Experimental drug followed by placebo
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
Placebo followed by experimental drug
Intervention Type
Drug
Intervention Name(s)
NMD670
Intervention Description
Tablets
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Tablets
Primary Outcome Measure Information:
Title
Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
Description
Distance walked (meters)
Time Frame
Baseline to day 21
Secondary Outcome Measure Information:
Title
Change from baseline in muscle strength versus placebo
Description
Handgrip, knee flexor, elbow flexor, elbow extension and should abduction (Newton)
Time Frame
Baseline to day 21
Title
Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo
Description
percentage change in distance walked in 6th minute compared to 1st minute
Time Frame
Baseline to day 21
Title
Change from baseline in Revised Hammersmith Scale (RHS) versus placebo
Description
Total score. Scale goes from 0-69 and higher score indicates improvement of symptoms
Time Frame
Baseline to day 21
Title
Change from baseline in time to dropout in the endurance shuttle 9-hole peg test (ESNHPT) versus placebo
Description
time to dropout (seconds)
Time Frame
Baseline to day 21
Title
Change from baseline in proportion of patients that drops out in the endurance shuttle 9-hole peg test (ESNHPT) versus placebo
Description
Proportion of patient dropout (%)
Time Frame
Baseline to day 21
Title
Change from baseline in jitter versus placebo
Description
Jitter (micro seconds) assessed with single fiber EMG
Time Frame
Baseline to day 21
Title
Change from baseline in blocking versus placebo
Description
Blocking (%) assessed with single fiber EMG
Time Frame
Baseline to day 21
Title
Incidence of treatment emergent adverse events
Description
Summarised per treatment
Time Frame
Over 21 days of dosing
Title
Incidence of serious treatment emergent adverse events
Description
Summarised per treatment
Time Frame
Over 21 days of dosing
Title
Incidence of clinically significant abnormalities on physical examinations
Description
Summarised per treatment
Time Frame
Over 21 days of dosing
Title
Incidence of clinically significant abnormalities on safety laboratory parameters
Description
Summarised per treatment
Time Frame
Over 21 days of dosing
Title
Incidence of clinically significant vital signs abnormalities
Description
Summarised per treatment
Time Frame
Over 21 days of dosing
Title
Incidence of clinically significant ECG abnormalities
Description
Summarised per treatment
Time Frame
Over 21 days of dosing
Title
Incidence of Suicidal Ideation or Suicidal Behavior
Description
Summarised per treatment
Time Frame
Over 21 days of dosing

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Participants who are 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. Participant with genetic confirmation of diagnosis (i.e., homozygous deletion 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 19-30 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. Exclusion Criteria: 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
NMD Pharma A/S
Phone
contact@nmdpharma.com
Email
contact@nmdpharma.com
Facility Information:
Facility Name
UF Fixel Institute for Neurological Diseases
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Name
Roy Blunt NextGen Precision Health Institute
City
Columbia
State/Province
Missouri
ZIP/Postal Code
65212
Country
United States
Individual Site Status
Recruiting
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Name
The Ohio State University Wexner Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Individual Site Status
Recruiting
Facility Name
UZ Leuven - Neurochirurgie Campus Gasthuisberg
City
Leuven
Country
Belgium
Individual Site Status
Recruiting
Facility Name
CHR de la Citadelle - Neurologie
City
Liège
Country
Belgium
Individual Site Status
Recruiting
Facility Name
Aarhus Universitetshospital, Neurologisk Afdeling
City
Aarhus
Country
Denmark
Individual Site Status
Recruiting
Facility Name
Rigshospitalet - Neurologisk Afdeling
City
København
Country
Denmark
Individual Site Status
Recruiting
Facility Name
Charite - Campus Virchow-Klinikum (CVK)
City
Berlin
Country
Germany
Individual Site Status
Recruiting
Facility Name
Universitätsklinikum Essen - Klinik Für Neurologie
City
Essen
Country
Germany
Individual Site Status
Recruiting
Facility Name
Istituto Neurologico C. Besta, Fondazione IRCCS
City
Milano
Country
Italy
Individual Site Status
Recruiting
Facility Name
Ospedale Niguarda, ASST Grande Ospedale Metropolitano Niguarda
City
Milano
Country
Italy
Individual Site Status
Recruiting
Facility Name
Universitair Medisch Centrum Utrecht, locatie Academisch Zie - Neurology
City
Utrecht
Country
Netherlands
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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