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

Graft Vs Host Disease Clinical Trial

Official title:

An Open Label Phase 2, Study to Evaluate the Safety and Efficacy of RGI-2001 for the Prevention of Acute Graft-vs-Host Disease Compared to Contemporary Controls in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04014790
• Other study ID #: RGI-2001-003
• Status: Completed
• Phase: Phase 2
• Start date: November 25, 2019
• Est. completion date: April 3, 2023

Study information

• Verified date: April 2024
• Source: Regimmune Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 49
• Est. completion date: April 3, 2023
• Est. primary completion date: July 5, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 64 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Drug:
• RGI-2001
Subjects will receive 6 weekly doses of RGI 2001, 100 µg/kg via IV administration after completion of alloHSCT
• Standard of Care
Standard of care prophylaxis regimen will be administered according to institutional guidelines

Locations

Country	Name	City	State
United States	University of Maryland Greenebaum Comprehensive Cancer Center	Baltimore	Maryland
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Ohio State University Wexner Medical Center	Columbus	Ohio
United States	Henry Ford Health System	Detroit	Michigan
United States	UCLA Medical Center	Los Angeles	California
United States	University of Miami, Sylvester Comprehensive Cancer Center	Miami	Florida
United States	Columbia University Irving Medical Center	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Regimmune Corporation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Grades II-IV aGVHD	Acute GVHD will be graded and assessed within 180 days post-transplant	Day 180 post-transplant
Secondary	Non-relapse Mortality (NRM) Rates	The probability of mortality not preceded by relapse of the underlying malignancy will be estimated	1 year post-transplant
Secondary	Disease-free Survival (DFS)	The probability of survival without relapse of the underlying malignancy will be estimated	1 year post-transplant
Secondary	GvHD-free, Relapse Free Survival (GRFS)	The probability of survival without relapse of the underlying malignancy, without severe (grades 3-4) acute GVHD, and without chronic GVHD requiring systemic immunosuppression will be estimated	1 year post-transplant
Secondary	Overall Survival (OS)	The probability of survival will be estimated	1 year post-transplant