Mycophenolate After Stem Cell Transplant for Systemic Sclerosis

Официальное название

Immunomodulatory Maintenance Therapy Post-Autologous Hematopoietic Stem Cell Transplantation for Prevention of Systemic Sclerosis Relapse: A Pilot Randomized Trial

Обзор

A pilot single center randomized trial to test whether a full-scale RCT evaluating the role of post-AHSCT maintenance immunosuppression with MMF in preventing disease relapse is feasible . We are studying whether a brief course of treatment with MMF after AHSCT will serve to prevent disease relapse.

Подробное описание

This study is performed determine the feasibility of a larger pragmatic RCT to evaluate the effectiveness of post- Autologous Hemopoietic Stem Cell Transplant (AHSCT) maintenance therapy with mycophenolate mofetil (MMF) in the prevention of Systemic sclerosis (SSc) relapse.

The study will include 6 participants who will be randomized at 1:1 ratio to treatment (MMF) or control arm (no treatment) for 6 months.

The participants will have 6 visits in total : An initial clinic visit will occur prior to stem cell collection. Post-AHSCT, in-person follow-up visits are scheduled on days 30, 90, 180, 270, and 300, with windows of +/- 1 week for days 30 and 90 and +/- 2 weeks for days 180, 270 and 300.

These visits will be coordinated at an established multidisciplinary (Rheumatology + Cell Therapy) clinic that are part of the patient's clinical care. At each visit, the participants will be asked to complete health questionnaires and laboratory tests will be performed as part of the standard of care of AHSCT recipients.

Вмешательства

• Препарат Mycophenolate Mofetil 1000 mg twice daily
  MMF 1000mg p.o. b.i.d. starting at day 90 post-AHSCT and continued for 6 months.

Первичные конечные точки

• Feasibility: [Срок оценки: 18 months]
• Efficacy : [Срок оценки: At day 0,90,180 and 270]
• Safety : AEs/SAEs [Срок оценки: Day 90 (treatment start) , Day 180, day 270]
• Efficacy: [Срок оценки: At day 0,90,180 and 270]

Вторичные конечные точки (8)

• Feasibility: Consent rate: [Срок оценки: Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.]
• Feasibility: Retention rate: Proportion of participants retained [Срок оценки: Day 270 (clinic visit #5)]
• Feasibility: Study completion rate: [Срок оценки: Day 270]
• Feasibility: Adherence rate: [Срок оценки: Day 180 and Day 270]
• Clinical outcomes: [Срок оценки: day 0, 90, 180 and 270]
• Auto-antibody testing (clinical) [Срок оценки: Prior to AHSCT and Day 180 (visit# 4)]
• Clinical outcome: [Срок оценки: day 0,90,180 and 270]
• Clinical outcome: [Срок оценки: Day 0,90,180 and 270]

Критерии участия

Критерии включения

• Consenting participants 18 years of age or older who are initiating AHSCT at The Ottawa Hospital for management of SSc.

Критерии исключения

• Exclusion criteria are focused on patient safety, namely inability to receive treatment with

MMF due to contraindications as determine at time of randomization (day 90 / clinic visit #3) including:

• Renal insufficiency (eGFR <25 mL/minute/1.73m2) or
• Neutropenia (ANC <1.0 x 103/mcL) or
• Thrombocytopenia (platelet count <50 000/mcL)
• Previously documented intolerance.
• Pregnant or breastfeeding - Women of childbearing potential who are not willing to use a highly effective method of contraception for the duration of study participation will be excluded

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

Открытое

Основная цель

Профилактика

Центры проведения

Канада · 1 центр

• The Ottawa Hospital — Ottawa

Идентификаторы

NCT: NCT07734012 · 20260406-01H

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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