ATLG Levels in Autologous HSCT for Multiple Sclerosis

Официальное название

ATLG Levels and Immune Reconstitution Kinetics in Autologous HSCT for Multiple Sclerosis

Обзор

This is a prospective, observational, biological, multicenter study to investigate IR profile, ATLG dynamics, main HSCT and disease outcomes in MS. This study will provide a preliminary descriptive evaluation of key study parameters, including ATLG pharmacokinetics and immune reconstitution trends, as well as an initial assessment of variability (e.g., dispersion of immunological biomarkers), to support the interpretation of results and the design of future studies. Since this is an observational study, no clinical decision will be made, and the interim analysis will not have an impact of the study conduction, and it will not be a stopping rule.

Вмешательства

• Препарат ATLG administration
  Patients will receive standard conditioning regimen (BEAM or cyclophosphamide) and ATLG (total dose 30 mg/kg over 3 days: 10 mg/kg on days -3, -2 and -1).

Первичные конечные точки

• Investigate immune status in correlation with ATLG [Срок оценки: before mobilization, before conditioning, +1/3/6/12 months after HSCT.]
• Investigate immune status in correlation with ATLG [Срок оценки: before and 30' after the end of ATLG dose, day0, day1, weekly for 4 weeks.]

Вторичные конечные точки (7)

• Assess clinically relevant viral infections in correlation with viral specific IR [Срок оценки: before mobilization, +1 month after HSCT within the first year after HSCT.]
• To assess HSCT and neurological outcomes [Срок оценки: at 100 days and 1-year]
• Assessment of Progression Free Survival (PFS) [Срок оценки: at 1-year]
• Assessment of Overall survival (OS) [Срок оценки: At 1 year]
• neurological status- Expanded Disability Status Scale (EDSS) [Срок оценки: at 100 days, 6 months and 1-year]
• neurological status - neurological progression [Срок оценки: at 1 year]
• neurological status -No Evidence of Disease Activity [Срок оценки: At 1 year]

Критерии участия

Критерии включения

• Participant is willing and able to give informed consent for participation in the study.
• Adult patients (age >/= 18y)
• Diagnosis of MS
• Confirmed program of autologous HSCT according to standard EBMT indications (any conditioning regimen considered standard, BEAM or Cyclophosphamide)
• ATLG (total dose 30 mg/kg over 3 days: 10 mg/kg on days -3, -2 and -1) in the conditioning regimen.

Критерии исключения

• Subjects that did not accept to sign the informed consent.
• Use of ATG.
• Contraindications to HSCT procedures (including pregnancy and breast feeding, uncontrolled active infections)

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Только случаи

Центры проведения

Италия · 1 центр

• IRCCS Ospedale San Raffaele — Milan

Публикации

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• Retiere C, Willem C, Guillaume T, Vie H, Gautreau-Rolland L, Scotet E, Saulquin X, Gagne K, Bene MC, Imbert BM, Clemenceau B, Peterlin P, Garnier A, Chevallier P. Impact on early outcomes and immune reconstitution of high-dose post-transplant cyclophosphamide vs anti-thymocyte globulin after reduced intensity conditioning peripheral blood stem cell allogeneic transplantation. Oncotarget. 2018 Jan PMID 29545911
• Naeije L, Kariminia A, Abdossamadi S, Azadpour S, Subrt P, Kuzeljevic B, Irvine MA, Walker I, Schultz KR. Anti-Thymocyte Globulin Prophylaxis Induces a Decrease in Naive Th Cells to Inhibit the Onset of Chronic Graft-versus-Host Disease: Results from the Canadian Bone Marrow Transplant Group (CBMTG) 0801 Study. Biol Blood Marrow Transplant. 2020 Mar;26(3):438-444. doi: 10.1016/j.bbmt.2019.11.015. PMID 31756535
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Идентификаторы

NCT: NCT07635693 · ATLG-MS · 4332

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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