Efficacy and Safety of HN2302 in Refractory Myasthenia Gravis(MG)

Официальное название

A Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HN2302 in Patients With Refractory Myasthenia Gravis

Обзор

This is an open label, single arm study, to evaluate the safety , tolerability and preliminary efficacy of HN2302 for refractory myasthenia gravis.

Подробное описание

The study will consist of an up to 4-week Screening Period, Treatment Period and one year Follow-up Period.

Вмешательства

• Препарат HN2302 Injection
  Patients will be administrated with specified dose on specified days at a lower dose level and escalated to safe and effective dose levels.

Первичные конечные точки

• Incidence of Treatment-Emergent Adverse Events [Срок оценки: Up to 3 months]

Вторичные конечные точки (9)

• Changes from baseline in Myasthenia Gravis Activities of Daily Living（MG-ADL） score [Срок оценки: Up to 12 months]
• Changes from baseline in Myasthenia Gravis Composite （MGC） score [Срок оценки: Up to 12 months]
• Changes from baseline in Quantitative Myasthenia Gravis (QMG) score [Срок оценки: Up to 12 months]
• Changes from baseline in 15-item quality of life (MG-QOL15r) score [Срок оценки: Up to 12 months]
• Percentage of patients with symptom changes after treatment [Срок оценки: Up to 12 months]
• in vivo CAR T cell production [Срок оценки: Day-28 to14 days]
• B cell ratio and counts in peripheral blood [Срок оценки: Up to 12 months]
• Dynamic changes in cytokine levels after treatment [Срок оценки: Up to 12 months]
• Changes in acetylcholine receptor (AchR) antibody levels after treatment [Срок оценки: Up to 12 months]

Критерии участия

Критерии включения

• Age: 18-80 years, no gender restriction;
• Confirmed diagnosis of generalized myasthenia gravis (MG) with positive AchR or MuSK antibodies, meeting at least one of the following conditions：(1) Repetitive nerve stimulation suggesting neuromuscular transmission defect; (2) Positive response to neostigmine test; (3) Clinically judged improvement of --MG symptoms after oral cholinesterase inhibitor therapy;
• Clinical classification of MG according to MGFA types IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa, IVb);
• Baseline MG-ADL score ≥6, ocular-related score <50%;
• Poor response and/or lack of efficacy under standard therapies;
• Minimum life expectancy > 12 weeks;
• Adequate bone marrow, coagulation, cardiopulmonary, liver, and renal function.

Критерии исключения

• Subjects positive for hepatitis B surface antigen (HBsAg) and/or hepatitis B core antibody (HBcAb) with detectable or quantifiable HBV DNA, positive for hepatitis C antibody (HCV Ab) with detectable or quantifiable HCV RNA, positive for HIV antibody, positive CMV DNA, or CMV DNA above the lower limit of detection; positive for syphilis antigen or antibody;
• Presence of other uncontrolled active infections;
• History of major organ transplantation (e.g., heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation;
• Pregnant or breastfeeding women;
• Receipt of any mRNA-LNP products or other LNP-based drugs within the past two years;
• History of any of the following cardiovascular conditions within 6 months prior to screening: New York Heart Association (NYHA) Class III or IV heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmias, any ventricular arrhythmias, or other clinically significant cardiac disease;
• History of ≥Grade 2 bleeding events within 30 days prior to screening, or requiring long-term continuous anticoagulation therapy (e.g., warfarin, low molecular weight heparin, Xa factor inhibitors);
• History of live vaccination within 30 days prior to screening;
• Severe central nervous system diseases or pathological changes, including but not limited to: cerebrovascular accident, aneurysm, epilepsy, seizures/convulsions, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disorders, organic brain syndromes, or psychosis;
• History of asthma or severe allergies;
• Any condition that, in the investigator's opinion, may increase the patient's risk or interfere with study assessments.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• The Affiliated Hospital of Xuzhou Medical University — Xuzhou

Идентификаторы

NCT: NCT07413835 · HN2302-N04

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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