Evaluate the Safety and Efficacy of Allogeneic Umbilical Cord Mesenchymal Stem Cells in Patients With Multiple Sclerosis

Официальное название

A Seamless Phase I/IIa Clinical Study to Evaluate the Safety and Efficacy of Allogeneic Umbilical Cord Mesenchymal Stem Cells in Patients With Multiple Sclerosis

Обзор

This study is to identify the safety and efficacy of repeat IV(Intravenous) and IT(Intrathecal) administrations of UMSC01 in patients with MS. While anti-inflammatory drugs are routinely used for the treatment of MS by inhibiting immune responses, their effects on axon remyelination or neuroregeneration are limited. The combined systemic delivery of UCMSCs via intravenous injection and local administration of the cells by IT was to have safety and therapeutic efficacy for patients with MS.

Подробное описание

There is single arm in Phase I part: 6 patients will be enrolled sequentially for safety considerations. The patient will receive UMSC01 via IV followed by IT at day 28 as described in above. After all patients in Phase I complete the safety assessment by SMC without any major safety issue 4 weeks after the last UMSC01 administration, the Phase IIa part will be initiated. There are 2 arms in Phase IIa part: Sham-controlled with conventional treatment control and administration of UMSC01 with conventional treatment.

Вмешательства

• Биопрепарат Allogeneic umbilical cord mesenchymal stem cells
  UMSC01 cells will be IV infusion followed by IT infusion with 12 months of follow up after treatment.
• Биопрепарат Control group
  Normal saline will be IV infusion followed by sham-IT infusion with 12 months of follow up after treatment.

Первичные конечные точки

• Primary Endpoint for Phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Primary Endpoint for Phase IIa portion [Срок оценки: from visit 2 to 12-month follow-up period]

Вторичные конечные точки (12)

• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoint for phase I portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoints for phase IIa portion [Срок оценки: from visit 2 to 6-month follow-up period]
• Efficacy endpoints for phase IIa portion [Срок оценки: from visit 2 to 12-month follow-up period]
• Efficacy endpoints for phase IIa portion [Срок оценки: from visit 2 to 12-month follow-up period]

Критерии участия

Критерии включения

• Patients are willing to sign informed consent.
• Male or female are age between 20 to 65 years old on date of consent.
• Diagnosis of Relapsing-Remitting MS (RRMS) (≥1 clinically documented relapse in the past 12 months, ≥2 clinically documented relapses in the last 24 months or ≥ 1 gadolinium enhanced lesion or T2 new lesion in the last 12 months) or Secondary Progressive MS (SPMS) (EDSS increase ≥1.0 point (baseline EDSS ≤ 5.0) or ≥ 0.5 point (baseline EDSS ≥5.5), and ≥1 clinical relapse or ≥1 gadolinium enhanced lesion in the last 12 months)
• MS diagnosis established between 2 to 15 years and EDSS score between 2.0 to 6.5 before enrollment
• Patient has appropriated blood clotting function as assessed by the following laboratory requirements: PT, APTT ≤ 1.5X upper limit of normal (ULN).
• Treatment failure (either ≥ 1 relapse, ≥ 1 new T2 lesion, ≥ one gadolinium enhanced lesion or EDSS deterioration) with at least one of MS disease modifying therapy as Interferon-β, Glatiramer acetate (Copaxone), Dimethyl fumarate (Tecfidera), Teriflunomide (Aubagio), Fingolimod (Gilenya), Ozanimod (Zeposia), Cladribine (Mavenclad), Siponimod (Mayzent), Ofatumumab (Kesimpta), or Natalizumab (Tysabri) for more than 6 months
• All male patients and female patients with child-bearing potential (between puberty and 2 years after menopause) should use appropriate contraception method(s) for at least 4 weeks after UMSC01 treatment

Критерии исключения

• Pregnancy, lactation, and those who are not pregnant but did not, or unwilling to, take effective contraceptives measures 4 weeks before and after the treatment.
• Patients with uncontrolled diabetes (fasting blood glucose > 250 mg/dL)
• Patients with inadequate hepatic and renal function: AST and ALT > 5X ULN; eGFR < 30 mL/min.
• Patients who are unable to undergo Brain MRI examination for any reason.
• Patients who have medical history or current clinically active malignant tumor, peripheral neuropathy, myopathy or other clinically significant neurological diseases that will confound the evaluation of this study.
• Patients who have immuno-compromised condition or is with known clinically significantly autoimmune conditions other than MS or is receiving immunosuppressive treatments other than MS treatment within 6 months.
• With active infection that required systemic treatment
• Patients who are participating in other clinical trials with an investigational product within 1 month.
• Patients who were treated with cytotoxic medications during the last 1 month prior to the infusion.
• Relapse of MS within1 month before UMSC01 infusion.
• With anti-CD20 therapy, such as rituximab
• Patients not suitable to participate the trial as judged by the Investigator(s)

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

Двойное слепое

Основная цель

Лечение

Центры проведения

Тайвань · 1 центр

• China Medical University Hospital — Taichung

Идентификаторы

NCT: NCT05532943 · ES-CMSC01-D1101

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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