Reduced Intensity Haploidentical Peripheral Blood Stem Cell Transplantation With Post-transplant Cyclophosphamide and Sirolimus/Mycophenolate Mofetil/RGI-2001 Based GVHD Prevention: a Pilot Study

Zachariah Michael DeFilipp1 个研究点分布在 1 个国家目标入组 25 人开始时间: 2020年8月14日最近更新: 2025年7月20日

适应症

干预措施

试验速览

阶段: 1 期
状态: 已完成
发起方
入组人数: 25
试验地点: 1
主要终点: Number of patients achieving successful donor engraftment

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This research study is studying the RGI-2001 for preventing Graft-vs-Host Disease (GVHD) in people with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), chronic myelomonocytic leukemic (CMML), chemosensitive hodgkin lymphoma (HL), or Non-Hodgkin lymphoma (NHL).who will have a blood stem cell transplantation.

GVHD is a condition in which cells from the donor's tissue attack the organs.
RGI-2001 is an investigational treatment

详细描述

This is a pilot study in subjects undergoing reduced-intensity haploidentical peripheral blood stem cell transplantation who will receive graft-versus-host disease prevention with post-transplant cyclophosphamide, followed by sirolimus, mycophenolate mofetil, and RGI-2001.
The research study procedures include screening for eligibility and study treatment including evaluations and follow up visits.
The standard of care drugs of fludarabine, cyclophosphamide, melphalan, radiation, sirolimus, and mycophenolate mofetil are all FDA approved.
Eligible Participants will be placed in 1 of 2 groups, per physicians discretion:
Regimen #1 :
Before stem cell transplant:Fludarabine + Cyclophosphamide + Radiation
After stem cell transplant: Cyclophosphamide + Sirolimus +Mycophenolate mofetil + RGI-2001
Regimen #2
Before stem cell transplant: fludarabine + melphalan + radiation
After stem cell transplant: cyclophosphamide + sirolimus +Mycophenolate mofetil + RGI-2001
A total of 20 participants will be enrolled to this trial
The U.S. Food and Drug Administration (FDA) has not approved RGI-2001 as a treatment for any disease.

研究设计

研究类型: Interventional
分配方式: Non Randomized
干预模型: Parallel
主要目的: Treatment
盲法: None

入排标准

年龄范围: 18 Years 至 80 Years（Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Men or women ≥ 18 and ≤ 80 years old
•Diagnosis of hematological malignancy:
•Acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) in morphologic complete remission
•Myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), or chronic myelomonocytic leukemic (CMML) with < 5% blasts in blood or bone marrow
•Chemosensitive Hodgkin lymphoma (HL) or Non-Hodgkin lymphoma (NHL)
•Patients must be undergoing haploidentical allogeneic hematopoietic cell transplantation, defined as 1st or 2nd degree relative with at least 5/10 matching at HLA-A, -B, -C, DR, and DQ.
•ECOG performance status ≤2
•Patients with adequate physical function as measured by:
•Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening fraction >25%
•Bilirubin ≤ 2.5 mg/dL, except for patients with Gilbert's syndrome or hemolysis
•ALT, AST, and Alkaline Phosphatase < 5 x ULN
•Renal: Serum creatinine within normal range, or if serum creatinine is outside normal range, then renal function (measured or estimated creatinine clearance or GFR) ≥ 40mL/min/1.73m2
•Pulmonary: DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted
•Ability to understand and the willingness to sign a written informed consent document

排除标准

•Prior allogeneic hematopoietic stem cell transplantation. (Patients may have received a prior autologous hematopoietic stem cell transplant.)
•Participants who are receiving any other investigational agents within 14 days prior to RGI-2001 dosing. Thus, participants must stop investigational agents by Day -9 prior to transplant.
•Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, recent myocardial infarction or stroke, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
•Patients with active or uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy.
•Planned use of prophylactic donor lymphocyte infusion (DLI) therapy.
•Pregnant and breast-feeding women are ineligible because they are not eligible for hematopoietic stem cell transplantation.
•HIV-positive participants and patients with active Hepatitis B or C are ineligible