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Trial record 1 of 9 for:    NMD | Spinal Muscular Atrophy
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Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)

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ClinicalTrials.gov Identifier: NCT05794139
Recruitment Status : Recruiting
First Posted : April 3, 2023
Last Update Posted : April 26, 2024
Sponsor:
Information provided by (Responsible Party):
NMD Pharma A/S

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

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Drug: NMD670 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: 2-way crossover
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : September 21, 2023
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024


Arm Intervention/treatment
Experimental: Cohort 1
Experimental drug followed by placebo
Drug: NMD670
Tablets

Drug: Placebo
Tablets

Experimental: Cohort 2
Placebo followed by experimental drug
Drug: NMD670
Tablets

Drug: Placebo
Tablets




Primary Outcome Measures :
  1. Change from baseline in 6 minute walk test (6MWT) total distance versus placebo [ Time Frame: Baseline to day 21 ]
    Distance walked (meters)


Secondary Outcome Measures :
  1. Change from baseline in muscle strength versus placebo [ Time Frame: Baseline to day 21 ]
    Handgrip, knee flexor, elbow flexor, elbow extension and should abduction (Newton)

  2. Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo [ Time Frame: Baseline to day 21 ]
    percentage change in distance walked in 6th minute compared to 1st minute

  3. Change from baseline in Revised Hammersmith Scale (RHS) versus placebo [ Time Frame: Baseline to day 21 ]
    Total score. Scale goes from 0-69 and higher score indicates improvement of symptoms

  4. Change from baseline in jitter versus placebo [ Time Frame: Baseline to day 21 ]
    Jitter (micro seconds) assessed with single fiber EMG

  5. Change from baseline in blocking versus placebo [ Time Frame: Baseline to day 21 ]
    Blocking (%) assessed with single fiber EMG

  6. Incidence of treatment emergent adverse events [ Time Frame: Over 21 days of dosing ]
    Summarised per treatment

  7. Incidence of serious treatment emergent adverse events [ Time Frame: Over 21 days of dosing ]
    Summarised per treatment

  8. Incidence of clinically significant abnormalities on physical examinations [ Time Frame: Over 21 days of dosing ]
    Summarised per treatment

  9. Incidence of clinically significant abnormalities on safety laboratory parameters [ Time Frame: Over 21 days of dosing ]
    Summarised per treatment

  10. Incidence of clinically significant vital signs abnormalities [ Time Frame: Over 21 days of dosing ]
    Summarised per treatment

  11. Incidence of clinically significant ECG abnormalities [ Time Frame: Over 21 days of dosing ]
    Summarised per treatment

  12. Incidence of Suicidal Ideation or Suicidal Behavior [ Time Frame: Over 21 days of dosing ]
    Summarised per treatment

  13. Incidence of clinically significant abnormalities on opthalmological examinations [ Time Frame: Over 21 days of dosing ]
    Summarised per treatment



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05794139


Contacts
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Contact: NMD Pharma A/S contact@nmdpharma.com contact@nmdpharma.com

Locations
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Sponsors and Collaborators
NMD Pharma A/S
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Responsible Party: NMD Pharma A/S
ClinicalTrials.gov Identifier: NCT05794139    
Other Study ID Numbers: NMD670-02-0001
2022-002301-24 ( EudraCT Number )
First Posted: April 3, 2023    Key Record Dates
Last Update Posted: April 26, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by NMD Pharma A/S:
Transmission Enhancer
Neuromuscular Junction Transmission
ClC-1
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Spinal Cord Diseases
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases