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

NCT06414954 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: NMD670-02-00022023-507539-40
• Study Type: interventional
• Target: 84
• Locations: 10 countries
• Sites: 40

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.

Conditions

Myasthenia Gravis; Myasthenia Gravis, MuSK

Outcome Measures

Primary Outcomes (1)

• Change from baseline to day 21 in QMG total score for NMD670 vs placebo

  Scale goes from 0-39 and higher score indicates worse symptomatology

  Baseline to day 21

Secondary Outcomes (12)

• Change from baseline to day 21 in MG-ADL total score for NMD670 vs placebo

  Baseline to day 21

• Change from baseline to day 21 in MGC total score for NMD670 vs placebo

  Baseline to day 21

• Change from baseline to day 21 in MG-QOL15r for NMD670 vs placebo

  Baseline to day 21

• Change from baseline to day 21 in Neuro-QoL Fatigue Short Form

  Baseline to day 21

• Incidence of treatment emergent adverse event

  Over 21 days of dosing

Study Arms (4)

NMD670 high dose

EXPERIMENTAL

Drug: NMD670

NMD670 mid dose

EXPERIMENTAL

Drug: NMD670

NMD670 low dose

EXPERIMENTAL

Drug: NMD670

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

NMD670 DRUG

Tablets taken twice a day for 21 days

NMD670 high dose; NMD670 low dose; NMD670 mid dose

Placebo DRUG

Tablets taken twice a day for 21 days

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• NMD Pharma A/S: lead

Study Sites (40)

Profound Research LLC

Carlsbad, California, 92011, United States

WITHDRAWN

University of California Irvine Medical Center

Irvine, California, 92868, United States

RECRUITING

University of Colorado Neuromuscular Division

Aurora, Colorado, 80045, United States

RECRUITING

SFM Clinical Research, LLC

Boca Raton, Florida, 33487, United States

RECRUITING

Neuromuscular Research Division | University of South Florida

Tampa, Florida, 33612, United States

RECRUITING

Augusta University, Neuroscience Center

Augusta, Georgia, 30912, United States

RECRUITING

NextGen Precision Health

Columbia, Missouri, 65212, United States

RECRUITING

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

The Ohio State University

Columbus, Ohio, 43221, United States

RECRUITING

University of Oregon

Portland, Oregon, 97239, United States

RECRUITING

Semmes Murphey Clinic

Memphis, Tennessee, 38120, United States

RECRUITING

UZ Leuven

Leuven, Belgium

WITHDRAWN

Aarhus University Hospital

Aarhus, Denmark

RECRUITING

Rigshospitalet University of Copenhagen

Copenhagen, Denmark

RECRUITING

Centre de référence des maladies neuromusculaires AOC-CHU de Bordeaux - Hôpital Pellegrin

Bordeaux, France

RECRUITING

Centre de Référence des Maladies Neuromusculaires et de la SLA

Marseille, France

RECRUITING

Centre Hospitalier Universitaire de Nantes - Hôtel Dieu Centre de Référence des Maladies Neuromusculaires Rares -

Nantes, France

RECRUITING

CHU de Nice

Nice, France

RECRUITING

Unité de Recherche Clinique NeuroSciences

Nice, France

RECRUITING

JSC Curatio

Tbilisi, Georgia

RECRUITING

LTD David Tatishvili Health Center

Tbilisi, Georgia

RECRUITING

Ltd New Hospitals

Tbilisi, Georgia

RECRUITING

ASST Papa Giovanni XXIII (Azienda Ospedaliera Papa Giovanni XXIII)

Bergamo, 21127, Italy

RECRUITING

IRCCS Istituo delle Scienze neurologiche di Bologna, UOC Clinica neurologica Ospedale

Bologna, 40139, Italy

RECRUITING

IRCCS Ospedale Policlinico San Martino

Genova, 16132, Italy

RECRUITING

IRCCS Fondazione Istituto Neurologico Carlo Besta, Dipartimento di Ricerca e Sviluppo clinico

Milan, 20133, Italy

RECRUITING

DIMER, IRCCS, Ospedale San Raffaele

Milan, Italy

RECRUITING

Centro Sclerosi Multipla Napoli - AOU Vanvitelli

Naples, 80138, Italy

RECRUITING

AOU Pisana

Pisa, 56126, Italy

RECRUITING

Azienda Ospedaliera Sant'Andrea - Universita di Roma La Sapienza

Roma, 00189, Italy

RECRUITING

Leiden Universitair Medisch Centrum (LUMC)

Leiden, 2333, Netherlands

RECRUITING

Neurologia Śląska Centrum Medyczne

Katowice, Poland

RECRUITING

Szpital Specjalistyczny im. Ludwika Rydygiera w Krakowie sp.z o.o

Krakow, Poland

RECRUITING

Centrum Medyczne Hope Clinic

Lublin, 20701, Poland

RECRUITING

Galen Clinic

Lublin, Poland

RECRUITING

NeuroCor Banaszkiewicz, Tomaszewski Lekarze Spółka Partnerska

Myślenice, Poland

RECRUITING

Centrum Medyczne Neuro Protect

Warsaw, Poland

RECRUITING

University Clinical Center of Serbia, Neurology Clinic

Belgrade, Serbia

RECRUITING

University Clinical Center Nis, Neurology Clinic

Niš, Serbia

RECRUITING

Hospìtal Universitari Vall d'Hebrón

Barcelona, Spain

RECRUITING

Related Publications (1)

• Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025.

  PMID: 41224659 DERIVED

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

NMD Pharma A/S

CONTACT

contact@nmdpharma.com; contact@nmdpharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2024
• First Posted: May 16, 2024
• Study Start: May 16, 2024
• Primary Completion: May 1, 2026
• Study Completion: May 1, 2026
• Last Updated: April 13, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1); GE (3); NL (1); US (11); DK (2); ES (1); SE (2); PL (6); FR (5); IT (8)