A Phase 1b/2 Study of IM-101 in Adult Participants With Generalized Myasthenia Gravis and Ocular Myasthenia Gravis

Официальное название

A Phase 1b/2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Investigate A) the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Ascending Doses of IM-101 in Adult Participants With Generalized Myasthenia Gravis, and B) the Efficacy and Safety of Treatment of IM-101 in Adult Participants With Generalized Myasthenia Gravis and Ocular Myasthenia Gravis

Обзор

The goal of this clinical trial is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential efficacy of IM-101 in adult participants with AChR antibody-positive gMG. Subsequently, the safety and efficacy of the selected IM-101 dose-regimen will be tested in participants with AChR antibody-negative gMG and participants with AChR antibody-positive or AChR antibody-negative oMG.

Вмешательства

• Препарат IM-101 Part A
  Participants will receive IM-101 intravenously (IV), at a loading dose on Day 1 and Day 15 followed by maintenance dose and Day 29.
• Препарат Placebo Part A
  Participants will receive Placebo intravenously (IV), at a loading dose on Day 1 and Day 15 followed by maintenance dose on Day 29.
• Препарат IM-101 Part B
  Participants will receive IM-101 intravenously (IV), at a loading dose on Day 1 and Day 15 followed by maintenance dose on Day 29, D57 and D85.
• Препарат Placebo Part B
  Participants will receive Placebo intravenously (IV), at a loading dose on Day 1 and Day 15 followed by maintenance dose on Day 29, D57 and D85.

Первичные конечные точки

• [Part A] Incidence of TEAEs, SAEs, AEs that led to premature discontinuation, and AESIs across 3 ascending dose regimens (each with 3 administrations) in participants with AChR antibody-positive gMG [Срок оценки: From first dose of study drug (Day 1) up to 70 days after the last dose of study drug, up to approximately 99 days.]
• [Part B] Incidence of TEAEs, SAEs, AEs that led to premature discontinuation, and AESIs of IM-101, compared with placebo, in participants with AChR antibody-positive gMG, AChR antibody-negative gMG, and oMG [Срок оценки: From first dose of study drug (Day 1) up to 84 days after the last dose of study drug, up to approximately 169 days.]
• [Part B] Change from baseline to Week 16 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score for gMG cohorts [Срок оценки: Baseline, Week 16]
• [Part B]Change from baseline to Week 16 in Myasthenia Gravis Impairment Index (MGII) ocular score for oMG cohorts [Срок оценки: Baseline, Week 16]

Критерии участия

Критерии включения

• Able and willing to provide signed informed consent
• Willingness to consent to screening for genetic muscular diseases
• Male or female aged ≥ 18 years and < 75 years
• Diagnosed with MG
• On a stable dose of background therapy for the treatment of MG
• Body weight ≥ 40 kg at screening
• Vaccinated against meningococcal infection (Neisseria meningitidis), streptococcus pneumoniae, and haemophilus influenzae type B

Критерии исключения

• Previous exposure to IM-101
• Anti-MuSK antibody Positive
• History of malignant thymoma, or history of cancer within the past 5 years of screening
• History of N. meningitidis infection
• Has been treated with any complement inhibitor, but failed due to intolerability or lack of efficacy

Full eligibility criteria is available in the study protocol.

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

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 8 центров

• Neurology of Central Florida Research Center, LLC — Altamonte Springs
• SFM Clinical Research, LLC — Boca Raton
• Aqualane Clinical Research — Naples
• Medsol Clinical Research Center — Port Charlotte
• University of South Florida — Tampa
• University of Kansas Medical Center Research Institute, Inc. — Kansas City
• Nerve & Muscle Center of Texas — Houston
• Houston Methodist Neurological Institute — Houston

Болгария · 5 центров

• Medical Center Hera - branch Montana — Montana
• "MHAT Avis - Medica" OOD — Pleven
• UMHAT 'Dr. Georgi Stranski', EAD — Pleven
• UMHAT 'Tsaritsa Yoanna - ISUL', EAD — Sofia
• Haelan Care 4 Medical Center EOOD — Varna

Польша · 5 центров

• Neurologia Slaska Centrum Medyczne — Katowice
• Twoja Przychodnia NCM — Nowa Sól
• Twoja Przychodnia PCM — Poznan
• NZOZ Neuro-Kard Ilkowski i Partnerzy Spółka Partnerska Lekarzy — Poznan
• Samodzielny Publiczny Szpital Kliniczny nr 1 im. Prof. Stanislawa Szyszko, SUM — Zabrze

Италия · 3 центра

• Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (Presidio Spedali Civ — Brescia
• Ospedale San Raffaele — Milan
• Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan

Испания · 3 центра

• Hospital Universitario Clinico San Carlos — Madrid
• Hospital Universitario La Paz — Madrid
• Clinica Universidad de Navarra — Pamplona

Сербия · 1 центр

• General Hospital MSB Medical System Belgrade — Belgrade

Идентификаторы

NCT: NCT07250750 · IM-101_MG_2.1 · 2025-522406-20-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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