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

NCT ID: NCT04014790

Last Updated: 2024-05-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 49 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-11-25
• Study Completion Date: 2023-04-03

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.

Detailed Description

This is an open-label, multi-center, single-arm study to evaluate six weekly doses of RGI-2001 in combination with standard of care treatment for the prevention of aGvHD in subjects following alloHSCT. The study will include a Safety Run-in Phase to assess the safety and tolerability of 6 weekly doses of RGI-2001 followed by an Expansion Phase in which the potential efficacy of 6 weekly doses of RGI-2001 in addition to standard of care for GvHD prophylaxis will be assessed. Comparison will be made to a contemporaneous control group.

Conditions

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

RGI-2001

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

Group Type: EXPERIMENTAL

RGI-2001

Intervention Type: DRUG

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

Standard of Care

Intervention Type: DRUG

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

Interventions

RGI-2001

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

Intervention Type: DRUG

Standard of Care

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

Intervention Type: DRUG

Other Intervention Names

KRN-7000; RGI-7000

Eligibility Criteria

Inclusion Criteria

1. Ages ≥ 18 and ≤ 65 years of age

2. 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

3. Must have adequate organ function

4. Transplant Donor: Matched related donor or Unrelated donor

5. Is a candidate for anti-graft-vs-host-disease (GvHD) prophylaxis that includes a calcineurin inhibitor

6. Ability to understand and willingness to sign a written informed consent form

7. 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

8. 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

Exclusion Criteria

1. Has had any other prior organ transplantation

2. Planned procedure to deplete regulatory T cells from donor transplant materials

3. Planned reduced intensity conditioning

4. Has had prior treatment with anti-CD3, other T cell depleting antibodies, or anti-thymocyte globulin within 12 months prior to alloHSCT procedure

5. Has progressive underlying malignant disease including post-transplant lymphoproliferative disease

6. 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)

7. Is female and pregnant or lactating

8. Has a documented history of uncontrolled autoimmune disease or on active treatment

9. History of myocardial infarction, unstable angina, or acute coronary syndrome within 6 months prior to receiving study drug

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 64 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Regimmune Corporation

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Yi-Bin Chen, MD

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

UCLA Medical Center

Los Angeles, California, United States

University of Miami, Sylvester Comprehensive Cancer Center

Miami, Florida, United States

University of Maryland Greenebaum Comprehensive Cancer Center

Baltimore, Maryland, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Henry Ford Health System

Detroit, Michigan, United States

Columbia University Irving Medical Center

New York, New York, United States

Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Countries

United States

References

DeFilipp Z, Choe HK, Efebera YA, Saad A, Farhan S, Lekakis LJ, Yared JA, Schiller GJ, Mapara MY, Assal A, Gooley TA, Bui JD, Lee DD, Lane H, Chen YB. RGI-2001 for the Prophylaxis of Acute Graft-Versus-Host Disease Following Allogeneic HCT. Blood. 2025 Jul 18:blood.2025029584. doi: 10.1182/blood.2025029584. Online ahead of print.

Reference Type: DERIVED

PMID: 40680268 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

RGI-2001-003

Identifier Type: -

Identifier Source: org_study_id