RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation

NCT04014790 | Completed Phase 2 Results FDA Drug

• 1 other identifier: RGI-2001-003
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 7

Brief Summary

This Phase II open label study will evaluate the safety and efficacy of repeat doses of RGI-2001 in combination with standard of care treatment for the prevention of acute graft-vs-host-disease (aGvHD) in subjects following Allogeneic Hematopoietic Stem Cell Transplantation (alloHSCT). These subjects will be compared to contemporary controls.

Conditions

Graft Vs Host Disease; Graft-versus-host-disease; Acute-graft-versus-host Disease; Prevention of aGVHD

Outcome Measures

Primary Outcomes (1)

• Grades II-IV aGVHD

  Acute GVHD will be graded and assessed according to modified KEYSTONE criteria and the severity grade (Grade II through IV) were captured up through the first 100 days post-transplant

  Day 100 post-transplant

Secondary Outcomes (5)

• Grades II-IV aGVHD

  Day 180 post-transplant

• Non-relapse Mortality (NRM) Rates

  1 year post-transplant

• Disease-free Survival (DFS)

  1 year post-transplant

• GvHD-free, Relapse Free Survival (GRFS)

  1 year post-transplant

• Overall Survival (OS)

  1 year post-transplant

Study Arms (1)

RGI-2001

EXPERIMENTAL

Subjects will be administered RGI 2001 in combination with standard of care treatment

Drug: RGI-2001; Drug: Standard of Care

Interventions

RGI-2001 DRUG

Subjects will receive 6 weekly doses of RGI 2001, 100 μg/kg via IV administration after completion of alloHSCT

Also known as: KRN-7000, RGI-7000

RGI-2001

Standard of Care DRUG

Standard of care prophylaxis regimen will be administered according to institutional guidelines

RGI-2001

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Ages ≥ 18 and ≤ 65 years of age
• Has a hematologic malignancy which includes Acute myelogenous leukemia (AML), T or B cell acute lymphoblastic leukemia (ALL) Myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), myeloproliferative disorder (MPD) including myeloid metaplasia and CML
• Must have adequate organ function
• Transplant Donor: Matched related donor or Unrelated donor
• Is a candidate for anti-graft-vs-host-disease (GvHD) prophylaxis that includes a calcineurin inhibitor
• Ability to understand and willingness to sign a written informed consent form
• If female of childbearing potential, must have had a negative serum pregnancy test prior to enrollment and must have agreed to use a double barrier method of contraception for 30 days after RGI-2001 administration
• If male, must be sterile or willing to use an approved method of contraception from the time of informed consent to 90 days after last dose of RGI-2001 administration

You may not qualify if:

• Has had any other prior organ transplantation
• Planned procedure to deplete regulatory T cells from donor transplant materials
• Planned reduced intensity conditioning
• Has had prior treatment with anti-CD3, other T cell depleting antibodies, or anti-thymocyte globulin within 12 months prior to alloHSCT procedure
• Has progressive underlying malignant disease including post-transplant lymphoproliferative disease
• Has evidence of active central nervous system (CNS) disease including known brain or leptomeningeal disease (CT or MRI scan of the brain required only in case of clinical suspicion of CNS involvement)
• Is female and pregnant or lactating
• Has a documented history of uncontrolled autoimmune disease or on active treatment
• History of myocardial infarction, unstable angina, or acute coronary syndrome within 6 months prior to receiving study drug

Sponsors & Collaborators

• Regimmune Corporation: lead

Study Sites (7)

UCLA Medical Center

Los Angeles, California, 90095, United States

Location

University of Miami, Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States

Location

University of Maryland Greenebaum Comprehensive Cancer Center

Baltimore, Maryland, 21201, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Columbia University Irving Medical Center

New York, New York, 10087-6453, United States

Location

Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

Related Publications (1)

• DeFilipp Z, Choe H, Efebera YA, Saad A, Farhan S, Lekakis L, Yared JA, Schiller G, Mapara MY, Assal A, Gooley T, Bui JD, Lee D, Lane H, Chen YB. RGI-2001 for the prophylaxis of acute graft-versus-host disease after allogeneic HCT. Blood. 2025 Oct 23;146(17):2037-2046. doi: 10.1182/blood.2025029584.

  PMID: 40680268 DERIVED

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

KRN 7000; Standard of Care

Condition Hierarchy (Ancestors)

Immune System Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Chief Executive Officer
• Organization: REGiMMUNE

kenzo@regimmune.com

+81-80-7022-7536

Study Officials

• Yi-Bin Chen, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2019
• First Posted: July 10, 2019
• Study Start: November 25, 2019
• Primary Completion: July 5, 2022
• Study Completion: April 3, 2023
• Last Updated: May 28, 2024
• Results First Posted: April 17, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)