Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis

Myasthenia Gravis Clinical Trial

— SYNAPSE-MG  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06414954
• Other study ID #: NMD670-02-0002
• Secondary ID: 2023-507539-40
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: May 2024
• Est. completion date: November 2025

Study information

• Verified date: May 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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 84
• Est. completion date: November 2025
• Est. primary completion date: November 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Muscle Weakness
• Myasthenia Gravis
• Myasthenia Gravis, MuSK

Intervention

Drug:
• NMD670
Tablets taken twice a day for 21 days
• Placebo
Tablets taken twice a day for 21 days

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
NMD Pharma A/S

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Incidence of treatment emergent adverse event	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	From screening (day -28 to day -1) until follow up (day 28)