Back to resultsRGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation
Primary Purpose
Graft Vs Host Disease, Graft-versus-host-disease, Acute-graft-versus-host Disease
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
RGI-2001
Standard of Care
Sponsored by
About this trial
This is an interventional prevention trial for Graft Vs Host Disease
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Sites / Locations
- UCLA Medical Center
- University of Miami, Sylvester Comprehensive Cancer Center
- University of Maryland Greenebaum Comprehensive Cancer Center
- Massachusetts General Hospital
- Henry Ford Health System
- Columbia University Irving Medical Center
- Ohio State University Wexner Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
RGI-2001
Arm Description
Subjects will be administered RGI 2001 in combination with standard of care treatment
Outcomes
Primary Outcome Measures
Grades II-IV aGVHD
Acute GVHD will be graded and assessed within the first 100 days post-transplant
Secondary Outcome Measures
Grades II-IV aGVHD
Acute GVHD will be graded and assessed within 180 days post-transplant
Non-relapse mortality (NRM) rates
The probability of mortality not preceded by relapse of the underlying malignancy will be estimated
Disease-free survival (DFS)
The probability of survival without relapse of the underlying malignancy will be estimated
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
Overall survival (OS)
The probability of survival will be estimated
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04014790
Brief Title
RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation
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
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
November 25, 2019 (Actual)
Primary Completion Date
July 5, 2022 (Actual)
Study Completion Date
April 3, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Regimmune Corporation
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Graft Vs Host Disease, Graft-versus-host-disease, Acute-graft-versus-host Disease, Prevention of aGVHD
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
49 (Actual)
8. Arms, Groups, and Interventions
Arm Title
RGI-2001
Arm Type
Experimental
Arm Description
Subjects will be administered RGI 2001 in combination with standard of care treatment
Intervention Type
Drug
Intervention Name(s)
RGI-2001
Other Intervention Name(s)
KRN-7000, RGI-7000
Intervention Description
Subjects will receive 6 weekly doses of RGI 2001, 100 μg/kg via IV administration after completion of alloHSCT
Intervention Type
Drug
Intervention Name(s)
Standard of Care
Intervention Description
Standard of care prophylaxis regimen will be administered according to institutional guidelines
Primary Outcome Measure Information:
Title
Grades II-IV aGVHD
Description
Acute GVHD will be graded and assessed within the first 100 days post-transplant
Time Frame
Day 100 post-transplant
Secondary Outcome Measure Information:
Title
Grades II-IV aGVHD
Description
Acute GVHD will be graded and assessed within 180 days post-transplant
Time Frame
Day 180 post-transplant
Title
Non-relapse mortality (NRM) rates
Description
The probability of mortality not preceded by relapse of the underlying malignancy will be estimated
Time Frame
Day 100, 6 months and 1 year post-transplant
Title
Disease-free survival (DFS)
Description
The probability of survival without relapse of the underlying malignancy will be estimated
Time Frame
6 months and 1 year post-transplant
Title
GvHD-free, relapse free survival (GRFS)
Description
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
Time Frame
6 months and 1 year post-transplant
Title
Overall survival (OS)
Description
The probability of survival will be estimated
Time Frame
6 months and 1 year post-transplant
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yi-Bin Chen, MD
Organizational Affiliation
Massachusetts General Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
UCLA Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
University of Miami, Sylvester Comprehensive Cancer Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
University of Maryland Greenebaum Comprehensive Cancer Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Henry Ford Health System
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Columbia University Irving Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10087-6453
Country
United States
Facility Name
Ohio State University Wexner Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation
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