The Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis

Официальное название

A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis

Обзор

A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects with Amyotrophic Lateral Sclerosis

Вмешательства

• Препарат Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)
  Injection, once, 6 months
• Препарат Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)
  Injection, every two weeks for four doses, 6 months

Первичные конечные точки

• The incidence of XS228CN-related adverse events (AEs) and serious adverse events (SAEs) within 28 days after the last administration in ALS patients [Срок оценки: within 28 days after the last administration]

Критерии участия

Критерии включения

• 18-75 years of age (inclusive of 18 and 75 years), regardless of gender;
• Diagnosed of definite or probable ALS according to the revised EI Escorial criteria;
• Respiratory function FVC at baseline was ≥70% of the predicted value (FVC%);
• Patients with birth-potential (both male and female) must agree to use effective non-drug contraceptive measures from the time of signing the informed consent until 6 months after the conclusion of the trial;
• Volunteer to participate in the clinical study, understand and sign the informed consent form.

Критерии исключения

• Subject has signs and symptoms of neuromuscular weakness, and other causes of muscle weakness cannot be ruled out;
• Baseline body mass index (BMI) < 18.5 kg/m²;
• Primary lateral sclerosis presenting only with upper motor neuron symptoms;
• Significant psychiatric disorders that the investigator assesses may affect evaluation;
• Diseases causing neurological or muscular dysfunction, such as metabolic muscle diseases or myasthenia gravis;
• Diagnosed autoimmune diseases with uncontrolled severe arthritis or other conditions (e.g., lameness) that the investigator assesses may affect evaluation;
• Acute active infections requiring antibiotics, antivirals, or antifungals that occurred within the 2 weeks prior to screening and are not controlled;
• Subject diagnosed with active pulmonary tuberculosis or treated for suspected tuberculosis;
• Diagnosed severe pulmonary diseases that the investigator assesses may affect evaluation;
• Poorly controlled hypertension;
• Previous or detected cardiac abnormalities;
• A history of cirrhosis, chronic hepatitis, or liver function at screening;
• A history of chronic kidney disease;
• Previous history of bleeding, abnormal clotting, or being treated with anticoagulation;
• Active hepatitis B, active hepatitis C, human immunodeficiency virus (HIV) antibody or treponema pallidum antibody positive at screening;
• History of severe trauma or surgery and may affect the assessment judged by investigator;
• Subjects with contraindications to lumbar puncture, including menifestations of injection site infection or high intracranial pressure.
• Those who have had a malignant tumor within 5 years prior to screening or are undergoing antitumor therapy;
• Have participated in other clinical trials within 3 months prior to screening;
• Pregnant or breastfeeding women;
• Subject who is judged by the investigator to be unsuitable for the clinical trial.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Peking University Third Hospital — Пекин

Идентификаторы

NCT: NCT07118319 · XS228-Allo-ALS-CN1/2-P01 · 2025LP01221

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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