Graft Vs Host DiseasePhase 2NCT04014790
RGI-2001 for the Prevention of Acute Graft-vs-Host Disease in Subjects Following Allogeneic Hematopoietic Stem Cell Transplantation
A Phase 2 study testing RGI-2001 in people with Graft Vs Host Disease and Graft-versus-host-disease, run by Regimmune Corporation at 7 sites in United States.
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). Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Graft Vs Host Disease, Graft-versus-host-disease, Acute-graft-versus-host Disease
- What is being tested?
- RGI-2001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 7 sitesUnited States
- Study dates
- 25 Nov 2019to 3 Apr 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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)
Show all 9Show fewer
- 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
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
University of Maryland Greenebaum Comprehensive Cancer Center
Baltimore, Maryland, 21201
Massachusetts General Hospital
Boston, Massachusetts, 02114
Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
Henry Ford Health System
Detroit, Michigan, 48202
UCLA Medical Center
Los Angeles, California, 90095
University of Miami, Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
Columbia University Irving Medical Center
New York, New York, 10087-6453
About this study
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
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.
Read the 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
- Being tested
- RGI-2001, Standard of Care
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RGI-2001Drug
Also called KRN-7000, RGI-7000
Subjects will receive 6 weekly doses of RGI 2001, 100 μg/kg via IV administration after completion of alloHSCT
Standard of CareDrug
Standard of care prophylaxis regimen will be administered according to institutional guidelines
| Study group | Role | What they receive |
|---|---|---|
| RGI-2001 | Experimental | Subjects will be administered RGI 2001 in combination with standard of care treatment Receives: Drug: RGI-2001; Drug: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Day 100 post-transplant
Secondary outcomes (5)
Grades II-IV aGVHD
Acute GVHD will be graded and assessed within 180 days post-transplant
Measured: Day 180 post-transplant
Non-relapse Mortality (NRM) Rates
The probability of mortality not preceded by relapse of the underlying malignancy will be estimated
Measured: 1 year post-transplant
Disease-free Survival (DFS)
The probability of survival without relapse of the underlying malignancy will be estimated
Measured: 1 year post-transplant
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
Measured: 1 year post-transplant
Overall Survival (OS)
The probability of survival will be estimated
Measured: 1 year post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2019
- Study start25 Nov 2019
- Primary completion5 Jul 2022
- Study completion3 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Graft Vs Host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regimmune Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yi-Bin Chen, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04014790
- Sponsor's protocol ID
- RGI-2001-003
- Status last verified
- Apr 2024
- First submitted
- 8 Jul 2019
- Last updated
- 28 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 40680268
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04014790
- Acronym / sponsor ID
- RGI-2001-003
- Conditions
- Graft Vs Host Disease, Graft-versus-host-disease, Acute-graft-versus-host Disease, Prevention of aGVHD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Regimmune CorporationIndustry (pharma, biotech, devices)
- Study start
- 25 Nov 2019 (actual)
- Primary completion
- 5 Jul 2022 (actual)
- Study completion
- 3 Apr 2023 (actual)
- First posted
- 10 Jul 2019 (actual)
- Results
- Posted 17 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RGI-2001 | Experimental | Drug: RGI-2001, Drug: Standard of CareSubjects will be administered RGI 2001 in combination with standard of care treatment |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RGI-2001Also: KRN-7000, RGI-7000Subjects will receive 6 weekly doses of RGI 2001, 100 μg/kg via IV administration after completion of alloHSCT | Drug | RGI-2001 |
| Standard of CareStandard of care prophylaxis regimen will be administered according to institutional guidelines | Drug | RGI-2001 |
Primary outcome
Grades II-IV aGVHD
Time frame: Day 100 post-transplant
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
5 secondary outcomes
Grades II-IV aGVHD
Time frame: Day 180 post-transplant
Non-relapse Mortality (NRM) Rates
Time frame: 1 year post-transplant
Disease-free Survival (DFS)
Time frame: 1 year post-transplant
GvHD-free, Relapse Free Survival (GRFS)
Time frame: 1 year post-transplant
Overall Survival (OS)
Time frame: 1 year post-transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40680268DeFilipp 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yi-Bin Chen, MD principal investigator
Massachusetts General Hospital
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RGI-2001 in people with Graft Vs Host Disease and Graft-versus-host-disease, run by Regimmune Corporation at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Grades II-IV aGVHD (time frame: Day 100 post-transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04014790, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2024; AzTrials last refreshed its copy on 7 Nov 2026.