Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis (SYNAPSE-MG)

A Phase 2b, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 3 Dose Levels of NMD670 Over 21 Days in Adult Patients With AChR/MuSK-Ab+ Myasthenia Gravis

This Phase 2 proof-of-concept, dose range finding study aims to evaluate the safety and efficacy of 3 dose levels of NMD670 vs placebo in adult patients with MG with antibodies against AChR or MuSK, administered twice a day (BID) for 21 days.

Study Overview

• Status: Not yet recruiting
• Conditions: Myasthenia Gravis; Myasthenia Gravis, MuSK
• Intervention / Treatment: Drug: NMD670; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 84
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: NMD Pharma A/S; Phone Number: contact@nmdpharma.com; Email: contact@nmdpharma.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participant must be a male or female being 18 to 75 years (both included), at the time of signing the informed consent
• Diagnosis of MG, MGFA class II, III or IV
• Documented positive AChR or MuSK antibody test.
• Participant must be able to swallow tablets
• Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
• 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 and has given signed informed consent

Exclusion Criteria:

• Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study
• Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
• Participants that received treatment with an investigational medical product 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 MG therapy
• Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Tablets taken twice a day for 21 days
Experimental: NMD670 high dose	Drug: NMD670; Tablets taken twice a day for 21 days
Experimental: NMD670 mid dose	Drug: NMD670; Tablets taken twice a day for 21 days
Experimental: NMD670 low dose	Drug: NMD670; Tablets taken twice a day for 21 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline to day 21 in QMG total score for NMD670 vs placebo	Scale goes from 0-36 and higher score indicates worse symptomatology	Baseline to day 21

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of serious treatment emergent adverse events	Summarised per treatment	Over 21 days of dosing
Incidence of clinically significant abnormalities on physical examinations	Summarised per treatment	Over 21 days of dosing
Incidence of clinically significant abnormalities on safety laboratory parameters	Summarised per treatment	Over 21 days of dosing
Incidence of clinically significant vital signs abnormalities	Summarised per treatment	Over 21 days of dosing
Incidence of clinically significant ECG abnormalities	Summarised per treatment	Over 21 days of dosing
Incidence of Suicidal Ideation or Suicidal Behavior	Summarised per treatment	Over 21 days of dosing
Change from baseline to day 21 in MG-ADL total score for NMD670 vs placebo	Scale goes from 0-24 and higher score indicates worse symptomatology	Baseline to day 21
Change from baseline to day 21 in MGC total score for NMD670 vs placebo	Scale goes from 0-50 and higher score indicate worse symptomatology	Baseline to day 21
Change from baseline to day 21 in MG-QOL15r for NMD670 vs placebo	Scale goes from 0-30 and higher score indicate worse quality of life	Baseline to day 21
Change from baseline to day 21 in Neuro-QoL Fatigue Short Form	Scale goes from 8-40 and higher score indicate worse symptomalogy	Baseline to day 21
Incidence of treatment emergent adverse event	Summarised per treatment	Over 21 days of dosing
Incidence of clinically significant abnormalities on opthalmological examinations	Summarised per treatment	From screening (day -28 to day -1) until follow up (day 28)

Collaborators and Investigators

• Sponsor: NMD Pharma A/S

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2024
• Primary Completion (Estimated): November 1, 2025
• Study Completion (Estimated): November 1, 2025

Study Registration Dates

• First Submitted: May 10, 2024
• First Submitted That Met QC Criteria: May 10, 2024
• First Posted (Actual): May 16, 2024

Study Record Updates

• Last Update Posted (Actual): May 16, 2024
• Last Update Submitted That Met QC Criteria: May 10, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis
• Other Study ID Numbers: NMD670-02-0002; 2023-507539-40 (Other Identifier: EUCTR)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No