Clinical Trials/NCT05794139

NCT05794139

Active, not recruiting

Phase 2

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

NMD Pharma A/S61 sites in 8 countries54 target enrollmentSeptember 21, 2023

ConditionsSpinal Muscular Atrophy

InterventionsNMD670 Placebo

DrugsNMD670 Placebo

Overview

Phase: Phase 2
Intervention: NMD670
Conditions: Spinal Muscular Atrophy
Sponsor: NMD Pharma A/S
Enrollment: 54
Locations: 61
Primary Endpoint: Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
Status: Active, not recruiting
Last Updated: 19 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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

Registry

clinicaltrials.gov

Start Date

September 21, 2023

End Date

May 1, 2026

Last Updated

19 days ago

Study Type

Interventional

Study Design

Crossover

Sex

All

Investigators

Sponsor

NMD Pharma A/S

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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.

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

Arms & Interventions

Cohort 1

Experimental drug followed by placebo

Intervention: NMD670

Cohort 1

Experimental drug followed by placebo

Intervention: Placebo

Cohort 2

Placebo followed by experimental drug

Intervention: NMD670

Cohort 2

Placebo followed by experimental drug

Intervention: Placebo

Outcomes

Primary Outcomes

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

Time Frame: Baseline to day 21

Distance walked (meters)

Secondary Outcomes

Incidence of treatment emergent adverse events(Over 21 days of dosing)
Change from baseline in Revised Hammersmith Scale (RHS) versus placebo(Baseline to day 21)
Incidence of clinically significant abnormalities on safety laboratory parameters(Over 21 days of dosing)
Incidence of clinically significant vital signs abnormalities(Over 21 days of dosing)
Incidence of clinically significant ECG abnormalities(Over 21 days of dosing)
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 jitter versus placebo(Baseline to day 21)
Change from baseline in blocking versus placebo(Baseline to day 21)
Incidence of serious treatment emergent adverse events(Over 21 days of dosing)
Incidence of Suicidal Ideation or Suicidal Behavior(Over 21 days of dosing)
Incidence of clinically significant abnormalities on opthalmological examinations(Over 21 days of dosing)
Incidence of clinically significant abnormalities on physical examinations(Over 21 days of dosing)