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

Phase 2

Recruiting

• Conditions: Myasthenia Gravis; Myasthenia Gravis, MuSK
• Interventions: Drug: NMD670; Drug: Placebo

• Registration Number: NCT06414954
• Lead Sponsor: NMD Pharma A/S

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-05-10
• Study First Submitted QC: 2024-05-10
• Study Start: 2024-05-16
• Study First Posted: 2024-05-16
• Status Verified: 2025-02
• Last Update Submitted: 2025-04-12
• Last Update Posted: 2025-04-15
• Primary Completion: 2025-11-01
• Study Completion: 2025-11-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 84

Inclusion Criteria

• Participant must be a male or female being 18 or more, 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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NMD670 high dose	NMD670	-
NMD670 mid dose	NMD670	-
Placebo	Placebo	-
NMD670 low dose	NMD670	-

Primary Outcome Measures

Name	Time	Method
Change from baseline to day 21 in QMG total score for NMD670 vs placebo	Baseline to day 21	Scale goes from 0-39 and higher score indicates worse symptomatology

Secondary Outcome Measures

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

Trial Locations

Locations (37)

Profound Research LLC; 🇺🇸 Carlsbad, California, United States

University of California Irvine Medical Center; 🇺🇸 Irvine, California, United States

University of Colorado Neuromuscular Division; 🇺🇸 Aurora, Colorado, United States

SFM Clinical Research, LLC; 🇺🇸 Boca Raton, Florida, United States

Neuromuscular Research Division | University of South Florida; 🇺🇸 Tampa, Florida, United States

Augusta University, Neuroscience Center; 🇺🇸 Augusta, Georgia, United States

NextGen Precision Health; 🇺🇸 Columbia, Missouri, United States

The University of North Carolina at Chapel Hill; 🇺🇸 Chapel Hill, North Carolina, United States

University of Oregon; 🇺🇸 Portland, Oregon, United States

Semmes Murphey Clinic; 🇺🇸 Memphis, Tennessee, United States

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