Haplo Peripheral Blood Sct In GVHD Prevention

NCT ID: NCT04473911

Last Updated: 2025-07-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-08-14
• Study Completion Date: 2024-08-19

Brief Summary

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

Detailed Description

• 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.

Conditions

GVHD; AML; ALL; MDS; MPN; CMML; Hodgkin Lymphoma; Non Hodgkin Lymphoma; Blood Stem Cell Transplant Failure; Graft Vs Host Disease; Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndromes; Myeloproliferative Disorder; Chronic Myelomonocytic Leukemia; Chemosensitive Hodgkin Lymphoma

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Regimen 1: Fludarabine, Cyclophosphamide, and TBI

-. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician

• Pre- stem cell transplant:

• Fludarabine predetermined dose, intravenously, 4 times per cycle
• Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion
• Total body irradiation (TBI) once during treatment cycle
• Post stem cell transplant:

• Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion
• Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral.
• Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle
• RGI-2001: IV, predetermined dose, weekly to 6 total doses

Group Type: EXPERIMENTAL

FLUDARABINE

Intervention Type: DRUG

predetermined dose, intravenously, a predetermined times per cycle Given in both pre stem cell and post stem cell cycles

CYCLOPHOSPHAMIDE

Intervention Type: DRUG

◦Cyclophosphamide predetermined dose, predetermined number of times in Given in pre-stem cell Regimen #1 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion

TBI

Intervention Type: RADIATION

Total body irradiation (TBI) once per cycle.

Sirolimus

Intervention Type: DRUG

Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral: Please note that doses of sirolimus can be adjusted at the treating physician's discretion given the multiple drugs and other situations which affect its metabolism

Mycophenolate mofetil

Intervention Type: DRUG

◦Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle

RGI-2001

Intervention Type: DRUG

IV, predetermined dose, weekly to 6 total doses

CYCLOPHOSPHAMIDE

Intervention Type: DRUG

◦Given in Post Stem Cell cycle of Regimen #1 and #2 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion

Regimen 2: Fludarabine, Melphalan, and TBI

-. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician

• Pre- stem cell transplant:

• Fludarabine predetermined dose, intravenously 3 times per cycle
• Melphalan, infusion, determined dosage, once per cycle
• Total body irradiation (TBI) once per cycle.
• Post stem cell transplant

• Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion
• Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral:
• Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle
• RGI-2001: IV, predetermined dose, weekly to 6 total doses

Group Type: EXPERIMENTAL

FLUDARABINE

Intervention Type: DRUG

predetermined dose, intravenously, a predetermined times per cycle Given in both pre stem cell and post stem cell cycles

TBI

Intervention Type: RADIATION

Total body irradiation (TBI) once per cycle.

Melphalan

Intervention Type: DRUG

Melphalan, infusion, determined dosage, once per cycle

Sirolimus

Intervention Type: DRUG

Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral: Please note that doses of sirolimus can be adjusted at the treating physician's discretion given the multiple drugs and other situations which affect its metabolism

Mycophenolate mofetil

Intervention Type: DRUG

◦Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle

RGI-2001

Intervention Type: DRUG

IV, predetermined dose, weekly to 6 total doses

CYCLOPHOSPHAMIDE

Intervention Type: DRUG

◦Given in Post Stem Cell cycle of Regimen #1 and #2 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion

Interventions

FLUDARABINE

predetermined dose, intravenously, a predetermined times per cycle Given in both pre stem cell and post stem cell cycles

Intervention Type: DRUG

CYCLOPHOSPHAMIDE

◦Cyclophosphamide predetermined dose, predetermined number of times in Given in pre-stem cell Regimen #1 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion

Intervention Type: DRUG

TBI

Total body irradiation (TBI) once per cycle.

Intervention Type: RADIATION

Melphalan

Melphalan, infusion, determined dosage, once per cycle

Intervention Type: DRUG

Sirolimus

Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral: Please note that doses of sirolimus can be adjusted at the treating physician's discretion given the multiple drugs and other situations which affect its metabolism

Intervention Type: DRUG

Mycophenolate mofetil

◦Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle

Intervention Type: DRUG

RGI-2001

IV, predetermined dose, weekly to 6 total doses

Intervention Type: DRUG

CYCLOPHOSPHAMIDE

◦Given in Post Stem Cell cycle of Regimen #1 and #2 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion

Intervention Type: DRUG

Other Intervention Names

Fludara®; Cytoxan®; Neosar®; Alkeran®; L-PAM; L-Sarcolysin; Phenylalanine Mustard; Rapamune; CellCept; Myfortic; Cytoxan®; Neosar®

Eligibility Criteria

Inclusion Criteria

• 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%
• Hepatic:

• 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

Exclusion Criteria

• 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Regimmune Corporation

INDUSTRY

Sponsor Role: collaborator

Zachariah Michael DeFilipp

OTHER

Sponsor Role: lead

Responsible Party

Zachariah Michael DeFilipp

Sponsor Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Zachariah DeFilipp, MD

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

Massachusetts General Hospital

Boston, Massachusetts, United States

Countries

United States

Other Identifiers

19-336

Identifier Type: -

Identifier Source: org_study_id