NCT04473911

Haplo Peripheral Blood Sct In GVHD Prevention

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Zachariah Michael DeFilipp · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	FLUDARABINE	predetermined dose, intravenously, a predetermined times per cycle Given in both pre stem cell and post stem cell cycles
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
RADIATION	TBI	Total body irradiation (TBI) once per cycle.
DRUG	Melphalan	Melphalan, infusion, determined dosage, once per cycle
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
DRUG	Mycophenolate mofetil	◦Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle
DRUG	RGI-2001	IV, predetermined dose, weekly to 6 total doses
DRUG	CYCLOPHOSPHAMIDE	◦Given in Post Stem Cell cycle of Regimen #1 and #2 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion

Timeline

• Start date: 2020-08-14
• Primary completion: 2023-10-22
• Completion: 2024-08-19
• First posted: 2020-07-16
• Last updated: 2025-07-20

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study