Graft Versus Host Disease (cGvHD)Phase 1EVENTNCT06991361
Ex Vivo-Expanded Regulatory T Cells Plus Low-Dose Interleukin-2 for Steroid-Refractory Chronic GVHD
A Phase 1 study testing EVE-Treg in people with Graft Versus Host Disease (cGvHD), run by Sarah Nikiforow, MD at 1 site in United States.
Trial summary
Hematopoietic Stem Cell Transplant (HCT) has been used to treat children and adults who have leukemia, lymphoma and other cancers of blood and immune cells since the 1970s. For many of these forms of cancer, HCT works well. However, HCT can cause serious, sometimes life-threatening complications. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Graft Versus Host Disease (cGvHD)
- What is being tested?
- EVE-Treg
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 29 May 2026to 1 May 2031
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Recipient of 7-8/8 HLA-matched allogeneic hematopoietic stem cell transplantation or recipient of haploidentical allogenic hematopoietic stem cell transplantation.
- Age 2 and up.
- Must have steroid-refractory chronic GVHD that is still active despite at least 4 weeks of treatment with low-dose subcutaneous (SC) IL-2.
- Stable dose of glucocorticoids for 2 weeks prior to enrollment
- no addition or subtraction of other immunosuppressive medications for 4 weeks prior to enrollment.
- Must have adequate organ and marrow function.
- Ability to understand and willingness to sign a written informed consent form.
- Donor who is willing and cleared to donate starting material for manufacture of EVE-Treg.
Who cannot join
- Recipient of umbilical cord blood stem cell graft.
- Ongoing prednisone requirement greater than 1 mg/kg/day (or equivalent).
- Karnofsky/Lansky performance score less than 40%.
- Concurrent use of methotrexate, azathioprine, or a calcineurin-inhibitor plus sirolimus.
- Other investigational agents within 4 weeks prior to enrollment.
- Participants with post-transplant exposure to donor lymphocyte infusion, or T-cell or IL-2 targeted medications within 100 days prior to enrollment.
Show all 11Show fewer
- Participants with new immunosupprssive medication, extra-corporeal photopheresis or rituximab therapy initiated int he 4 weeks prior to enrollment.
- Participants with active malignant relapse or recrudescence of their prior hematologic disorder.
- Uncontrolled intercurrent illness unrelated to cGVHD.
- Participants with psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant patients are excluded.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Brandi Bratrude, 617-919-2197, brandi.bratrude@childrens.harvard.edu
Sarah Nikiforow, MD, PhD
About this study
Official title: A Phase I Trial of Ex Vivo-Expanded (EVE) Regulatory T Cells in Combination With Low-Dose Interleukin-2 for Steroid-Refractory Chronic Graft-versus-Host-Disease
Hematopoietic Stem Cell Transplant (HCT) has been used to treat children and adults who have leukemia, lymphoma and other cancers of blood and immune cells since the 1970s. For many of these forms of cancer, HCT works well. However, HCT can cause serious, sometimes life-threatening complications.
One of the most serious and common complications of HCT is graft-versus-host disease (GVHD). GVHD can appear early after the transplant, usually about 3 to 4 weeks after the transplant is given. This is called acute GVHD (aGVHD), because it usually happens over a couple of days. If successfully treated, acute GVHD quickly goes away. Sometimes GVHD happens months after the transplant. Then it is called chronic GVHD (cGVHD), because it happens gradually and goes away slowly.
The investigators are doing this study to see if one dose of Ex vivo-Expanded Regulatory T cells (EVE-Treg) can be used together with the daily Interleukin 2 (IL-2) to treat cGVHD, that has not responded to steroid treatment or low-dose IL-2.
- Conditions
- Graft Versus Host Disease (cGvHD)
- Being tested
- EVE-Treg
- Medical subject headings
- Graft vs Host Disease, Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EVE-TregBiological
Ex Vivo-Expanded (EVE) Regulatory T cells.
| Study group | Role | What they receive |
|---|---|---|
| Dose Level A | Experimental | EVE Treg Cell dose level A Receives: Biological: EVE-Treg |
| Dose Level B | Experimental | EVE Treg Cell dose level B Receives: Biological: EVE-Treg |
| Dose Level C | Experimental | EVE Treg Cell dose level C. Receives: Biological: EVE-Treg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of EVE-Treg
Safety of EVE-Treg, in combination with physician-prescribed daily low-dose IL-2, for the treatment of chronic GVHD refractory to steroids and low-dose IL-2. Safety will be based on adverse events at each Dose Level of EVE-Treg
Measured: 5 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 May 2025
- Study start29 May 2026
- Primary completion1 May 2029 (estimated)
- Study completion1 May 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft Versus Host Disease (cGvHD)Studies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarah Nikiforow, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Sarah Nikiforow, MD, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06991361
- Sponsor's protocol ID
- IRB00025318
- Acronym
- EVENT
- Status last verified
- Jul 2026
- First submitted
- 20 May 2025
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Trial of Ex Vivo-Expanded (EVE) Regulatory T Cells in Combination With Low-Dose Interleukin-2 for Steroid-Refractory Chronic Graft-versus-Host-Disease
- ClinicalTrials.gov ID
- NCT06991361
- Acronym / sponsor ID
- EVENT / IRB00025318
- Conditions
- Graft Versus Host Disease (cGvHD)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Sarah Nikiforow, MDUniversity, hospital or other
- Study start
- 29 May 2026 (actual)
- Primary completion
- 1 May 2029 (estimated)
- Study completion
- 1 May 2031 (estimated)
- First posted
- 27 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Hematopoietic Stem Cell Transplant (HCT) has been used to treat children and adults who have leukemia, lymphoma and other cancers of blood and immune cells since the 1970s. For many of these forms of cancer, HCT works well. However, HCT can cause serious, sometimes life-threatening complications.
One of the most serious and common complications of HCT is graft-versus-host disease (GVHD). GVHD can appear early after the transplant, usually about 3 to 4 weeks after the transplant is given. This is called acute GVHD (aGVHD), because it usually happens over a couple of days. If successfully treated, acute GVHD quickly goes away. Sometimes GVHD happens months after the transplant. Then it is called chronic GVHD (cGVHD), because it happens gradually and goes away slowly.
The investigators are doing this study to see if one dose of Ex vivo-Expanded Regulatory T cells (EVE-Treg) can be used together with the daily Interleukin 2 (IL-2) to treat cGVHD, that has not responded to steroid treatment or low-dose IL-2.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose Level A | Experimental | Biological: EVE-TregEVE Treg Cell dose level A |
| Dose Level B | Experimental | Biological: EVE-TregEVE Treg Cell dose level B |
| Dose Level C | Experimental | Biological: EVE-TregEVE Treg Cell dose level C. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Recipient of 7-8/8 HLA-matched allogeneic hematopoietic stem cell transplantation or recipient of haploidentical allogenic hematopoietic stem cell transplantation.
- Age 2 and up.
- Must have steroid-refractory chronic GVHD that is still active despite at least 4 weeks of treatment with low-dose subcutaneous (SC) IL-2.
- Stable dose of glucocorticoids for 2 weeks prior to enrollment
- no addition or subtraction of other immunosuppressive medications for 4 weeks prior to enrollment.
Key exclusion criteria (11 listed)
- Recipient of umbilical cord blood stem cell graft.
- Ongoing prednisone requirement greater than 1 mg/kg/day (or equivalent).
- Karnofsky/Lansky performance score less than 40%.
- Concurrent use of methotrexate, azathioprine, or a calcineurin-inhibitor plus sirolimus.
- Other investigational agents within 4 weeks prior to enrollment.
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 |
|---|---|---|
| EVE-TregEx Vivo-Expanded (EVE) Regulatory T cells. | Biological | Dose Level A, Dose Level B, Dose Level C |
Primary outcome
Safety of EVE-Treg
Time frame: 5 weeks
Safety of EVE-Treg, in combination with physician-prescribed daily low-dose IL-2, for the treatment of chronic GVHD refractory to steroids and low-dose IL-2. Safety will be based on adverse events at each Dose Level of EVE-Treg
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Graft Versus Host Disease (cGvHD)
RecruitingPhase 3
A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease
Chronic Graft-versus-host-disease
Not yet recruiting
Evaluating and Promoting Resilience in Adolescents With Tourette Syndrome
Tourette Syndrome, Tic Disorders
Active, not recruitingEarly Phase 1
Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide
Chronic Granulomatous Disease
Recruiting
Uncovering the Mechanism of Pain Relief by Peripheral Transcutaneous Magnetic Stimulation
Post-Surgical Pain, Chronic, Post-traumatic Pain, Chronic
Not yet recruitingPhase 1/2
A Study of Sequential Therapy With AHB-137 Injection Followed by TVAX-008 Injection in Participants Living With Chronic Hepatitis B (CHB)
Chronic Hepatitis B
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EVE-Treg in people with Graft Versus Host Disease (cGvHD), run by Sarah Nikiforow, MD at 1 site 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 11 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: looking for participants now, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Brandi Bratrude (617-919-2197, brandi.bratrude@childrens.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of EVE-Treg (time frame: 5 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Oct 2026.