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

Официальное название

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

Обзор

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.

Вмешательства

• Препарат NMD670
  Tablets taken twice a day for 21 days
• Препарат Placebo
  Tablets taken twice a day for 21 days

Первичные конечные точки

• Change from baseline to day 21 in QMG total score for NMD670 vs placebo [Срок оценки: Baseline to day 21]

Вторичные конечные точки (12)

• Change from baseline to day 21 in MG-ADL total score for NMD670 vs placebo [Срок оценки: Baseline to day 21]
• Change from baseline to day 21 in MGC total score for NMD670 vs placebo [Срок оценки: Baseline to day 21]
• Change from baseline to day 21 in MG-QOL15r for NMD670 vs placebo [Срок оценки: Baseline to day 21]
• Change from baseline to day 21 in Neuro-QoL Fatigue Short Form [Срок оценки: Baseline to day 21]
• Incidence of treatment emergent adverse event [Срок оценки: Over 21 days of dosing]
• Incidence of serious treatment emergent adverse events [Срок оценки: Over 21 days of dosing]
• Incidence of clinically significant abnormalities on physical examinations [Срок оценки: Over 21 days of dosing]
• Incidence of clinically significant abnormalities on safety laboratory parameters [Срок оценки: Over 21 days of dosing]
• Incidence of clinically significant vital signs abnormalities [Срок оценки: Over 21 days of dosing]
• Incidence of clinically significant ECG abnormalities [Срок оценки: Over 21 days of dosing]
• Incidence of Suicidal Ideation or Suicidal Behavior [Срок оценки: Over 21 days of dosing]
• Incidence of clinically significant abnormalities on opthalmological examinations [Срок оценки: From screening (day -28 to day -1) until follow up (day 28)]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

США · 11 центров

• Profound Research LLC — Carlsbad
• University of California Irvine Medical Center — Irvine
• University of Colorado Neuromuscular Division — Aurora
• SFM Clinical Research, LLC — Boca Raton
• Neuromuscular Research Division | University of South Florida — Tampa
• Augusta University, Neuroscience Center — Augusta
• NextGen Precision Health — Columbia
• The University of North Carolina at Chapel Hill — Chapel Hill
• … и ещё 3 центра

Италия · 8 центров

• ASST Papa Giovanni XXIII (Azienda Ospedaliera Papa Giovanni XXIII) — Bergamo
• IRCCS Istituo delle Scienze neurologiche di Bologna, UOC Clinica neurologica Ospedale — Bologna
• IRCCS Ospedale Policlinico San Martino — Genova
• IRCCS Fondazione Istituto Neurologico Carlo Besta, Dipartimento di Ricerca e Sviluppo clin — Milan
• DIMER, IRCCS, Ospedale San Raffaele — Milan
• Centro Sclerosi Multipla Napoli - AOU Vanvitelli — Naples
• AOU Pisana — Pisa
• Azienda Ospedaliera Sant'Andrea - Universita di Roma La Sapienza — Roma

Польша · 6 центров

• Neurologia Śląska Centrum Medyczne — Katowice
• Szpital Specjalistyczny im. Ludwika Rydygiera w Krakowie sp.z o.o — Krakow
• Centrum Medyczne Hope Clinic — Lublin
• Galen Clinic — Lublin
• NeuroCor Banaszkiewicz, Tomaszewski Lekarze Spółka Partnerska — Myślenice
• Centrum Medyczne Neuro Protect — Warsaw

Франция · 5 центров

• Centre de référence des maladies neuromusculaires AOC-CHU de Bordeaux - Hôpital Pellegrin — Bordeaux
• Centre de Référence des Maladies Neuromusculaires et de la SLA — Marseille
• Centre Hospitalier Universitaire de Nantes - Hôtel Dieu Centre de Référence des Maladies N — Nantes
• CHU de Nice — Nice
• Unité de Recherche Clinique NeuroSciences — Nice

Грузия · 3 центра

• JSC Curatio — Tbilisi
• LTD David Tatishvili Health Center — Tbilisi
• Ltd New Hospitals — Tbilisi

Дания · 2 центра

• Aarhus University Hospital — Aarhus
• Rigshospitalet University of Copenhagen — Copenhagen

Сербия · 2 центра

• University Clinical Center of Serbia, Neurology Clinic — Belgrade
• University Clinical Center Nis, Neurology Clinic — Niš

Бельгия · 1 центр

• UZ Leuven — Leuven

Нидерланды · 1 центр

• Leiden Universitair Medisch Centrum (LUMC) — Leiden

Испания · 1 центр

• Hospìtal Universitari Vall d'Hebrón — Barcelona

Публикации

• 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

Идентификаторы

NCT: NCT06414954 · NMD670-02-0002 · 2023-507539-40

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗