FMT for Pediatric SR-aGVHD

Официальное название

Fecal Microbiota Transplantation for the Treatment of Steroid-refractory Graft-versus-host Disease.

Обзор

This is a pilot, prospective, non-profit, multicenter, uncontrolled, open-label study to evaluate the safety and feasibility of FMT in patients aged between 3 months and 25 years suffering from acute intestinal GVHD resistant to conventional steroid therapy. Eligible patients will receive 1-3 FMT via naso-jejunal tube or endoscopy.

Вмешательства

• Биопрепарат Fecal Microbial Transplantation
  The administration of the fecal preparation (from a related donor or a third party donor) will be carried out via esophagogastroduodenoscopy, with the release of the fecal preparation into the duodenum; or via a nasoduodenal tube, with the release of the fecal preparation into the duodenum at a 'dose' of 7-12 ml/kg, up to a maximum of 250 ml/administration; or via colonoscopy. In the latter case, mucosal biopsies will not be performed to reduce the risk of bacterial translocation. In some subjec

Первичные конечные точки

• Safety (AE and treatment-related AE, according to CTCAE v5.0) [Срок оценки: 28 days]

Вторичные конечные точки (5)

• Efficacy: Overall Response Rate (ORR) [Срок оценки: 28 days]
• Microbiota modification [Срок оценки: 56 days]
• Cumulative incidence of infections [Срок оценки: 28 days]
• Efficacy on other GVHD target organs [Срок оценки: 56 days]
• Immune reconstitution [Срок оценки: 90 days]

Критерии участия

Критерии включения

• Diagnosis of hematological disease, malignant or non-malignant;
• Patients undergoing allogeneic stem cell transplantation from a familial or unrelated donor;
• Presence of acute GVHD with intestinal involvement (grade II-IV), steroid-resistant (i.e., with progression after 3 days of high-dose steroid therapy (methylprednisolone > 2 mg/kg), no response after 7 days, progression during steroid tapering, or failure to achieve remission on day 28 from the start of steroid therapy), for those who have no indication for other second- or third-line therapies;
• Signed informed consent.

Критерии исключения

• Presence of concurrent bacterial infections requiring systemic antibiotic therapy;
• Positivity for anti-HIV or anti-HCV antibodies, or for HbsAg with HBV-DNA positive by PCR;
• Presence of severe mucositis;
• History of chronic inflammatory bowel disease.

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

Италия · 3 центра

• Pediatric Hematology and Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna — Bologna
• UOC Clinica di Oncoematologia Pediatrica, Azienda Ospedale-Università Padova — Padova
• Department of Hematology/Oncology, Cell and Gene Therapy, IRCCS Bambino Gesù Children's Ho — Roma

Публикации

• van Lier YF, Davids M, Haverkate NJE, de Groot PF, Donker ML, Meijer E, Heubel-Moenen FCJI, Nur E, Zeerleder SS, Nieuwdorp M, Blom B, Hazenberg MD. Donor fecal microbiota transplantation ameliorates intestinal graft-versus-host disease in allogeneic hematopoietic cell transplant recipients. Sci Transl Med. 2020 Aug 12;12(556):eaaz8926. doi: 10.1126/scitranslmed.aaz8926. PMID 32801142
• Kakihana K, Fujioka Y, Suda W, Najima Y, Kuwata G, Sasajima S, Mimura I, Morita H, Sugiyama D, Nishikawa H, Hattori M, Hino Y, Ikegawa S, Yamamoto K, Toya T, Doki N, Koizumi K, Honda K, Ohashi K. Fecal microbiota transplantation for patients with steroid-resistant acute graft-versus-host disease of the gut. Blood. 2016 Oct 20;128(16):2083-2088. doi: 10.1182/blood-2016-05-717652. Epub 2016 Jul 26. PMID 27461930

Идентификаторы

NCT: NCT07602751 · 3444_OPBG_2024

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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