Graft Versus Host DiseasePhase 1NCT01795573
Ex-vivo Expanded Donor Regulatory T Cells for Prevention of Acute Graft-Versus-Host Disease
A Phase 1 study testing Cultured Treg cells in people with Graft Versus Host Disease, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
Trial summary
Clinical trial of allospecific regulatory t cells (Tregs) for prevention of acute graft-versus-host disease (GVHD) in human leukocyte antigen (HLA) identical sibling transplants. Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Graft Versus Host Disease
- What is being tested?
- Cultured Treg cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Oct 2014to 14 Aug 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Signed informed consent
- Diagnoses:
- Hematologic malignancies - Acute myelogenous leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS), chronic lymphocytic leukemia (CLL), non-Hodgkin lymphoma (NHL), Hodgkin lymphoma (HL), multiple myeloma (MM) - in complete remission (CR). Complete remission is defined per morphologic, cytogenetic, FISH, molecular, and radiographic imaging studies appropriate for each condition listed.
- AML, ALL: Normal values for absolute neutrophil count (>1000/microL) and platelet count (>100,000/microL); Absence of extramedullary leukemia; Less than 5 percent blast cells present in the bone marrow
- MDS: Bone marrow with ≤5 percent myeloblasts with normal maturation of all cell lines; Peripheral blood demonstrates hemoglobin ≥11 g/dL, platelets ≥100 x 10^9/L, neutrophils ≥1 x 10^9/L, and no circulating blasts
- CLL: Absence of constitutional symptoms attributable to CLL; No lymph nodes >1.5 cm in diameter on computed tomography; No hepatomegaly or splenomegaly by computed tomography; Absolute neutrophil count >1500/microL; Platelet count >100,000/microL; No clonal lymphocytes in the peripheral blood by immunophenotyping; Bone marrow with no evidence of clonal CLL (by flow cytometry and/or immunohistochemistry
Show all 21Show fewer
- NHL: No clinical evidence of disease or disease-related symptoms; Typically FDG-avid lymphomas: a post-treatment residual mass of any size is permitted as long as it is PET negative; Variably FDG-avid lymphoma/FDG avidity unknown: all lymph nodes normal size by CT; Spleen and liver non-palpable and without nodules; If pretreatment bone marrow biopsy was positive, repeat bone marrow biopsy must be negative; if morphologically indeterminate, immunohistochemistry should be negative If pretreatment bone marrow biopsy was positive, repeat bone marrow biopsy must be negative; if morphologically indeterminate, immunohistochemistry should be negative
- HL: No clinical evidence of disease or disease-related symptoms; A post-treatment residual mass of any size is permitted as long as it is PET negative; Spleen and liver must be non-palpable and without nodules; If a pre-treatment bone marrow biopsy was positive, an adequate bone marrow biopsy from the same site must be cleared of infiltrate; if this is indeterminate by morphology, immunohistochemistry should be negative
- MM: Absence of monoclonal protein in serum and urine by immunofixation with no current evidence of soft tissue plasmacytoma; Bone marrow aspirate and biopsy must demonstrate less than 5 percent clonal plasma cells; In patients who lack measurable M proteins in the serum and urine being monitored using the FLC levels, the definition of CR requires a normalization of the free light chain (FLC) ratio in addition to the above criteria
- MDS: May have achieved CR through either hypomethylating agent therapy, induction chemotherapy, or other therapy
- MDS: Low/intermediate-1 IPSS risk category patients are eligible only if they have failed prior therapy or are transfusion-dependent
- Peripheral blood white blood count (WBC) greater than 2,000 per microliter (required for collection of dendritic cell precursors)
- Adequate vital organ function: Left ventricular ejection fraction (LVEF) ≥ 45% by multigated acquisition (MUGA) scan or echocardiogram; Forced expiratory volume in one second (FEV1), forced vital capacity (FVC), and diffusing lung capacity oxygenation (DLCO) ≥ 50% of predicted values on pulmonary function tests; Transaminases (AST, ALT) < 3 times upper limit of normal values; Creatinine clearance ≥ 50cc/min
- Infectious disease criteria:
- No active infection; infection controlled with antimicrobial therapy is not excluded
- HIV negative by ELISA or reverse transcription polymerase chain reaction (RT-PCR) [if ELISA is positive and RT-PCR is negative, the ELISA is considered false positive]
- Hepatitis B and C negative by serology or RT-PCR
- Must complete full screening panel: HIV 1, 2 serology and RT-PCR; human T cell lymphotropic virus types 1/2 (HTLV-1/2) serology; rapid plasma reagin (RPR) serology; Epstein-Barr virus (EBV) serology; Cytomegalovirus (CMV) serology; herpes simplex virus (HSV) serology; Varicella-. Zoster Virus (VZV) serology
- Performance status: Karnofsky Performance Status Score ≥ 60%.
- Agreement to utilize effective contraceptive methods during the study (for one year)
- Eligible donors will include siblings age ≥ 18 matched with the recipient at HLA-A, B, C, and DRB1
Who cannot join
- Antithymocyte globulin (ATG) as part of the conditioning regimen
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I Trial of Ex-vivo Expanded Donor Regulatory T Cells for Prevention of Acute Graft-Versus-Host Disease
Clinical trial of allospecific regulatory t cells (Tregs) for prevention of acute graft-versus-host disease (GVHD) in human leukocyte antigen (HLA) identical sibling transplants.
Read the detailed description
To evaluate the safety of sirolimus based immune suppression and ex-vivo expanded donor regulatory T cells for the prevention of acute graft-versus-host disease following allogeneic hematopoietic cell transplantation.
- Conditions
- Graft Versus Host Disease
- Being tested
- Cultured Treg cells
- Keywords
- GVHD, AML, ALL, MDS, CLL, NHL, HL, MM
- 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.
Cultured Treg cellsBiological
Co-culturing of recipient dendritic cells and donor Treg. Treg administration will occur 2 days before the allogeneic stem cell transplant (i.e. day -2 with reference of day 0 as stem cell infusion date).
| Study group | Role | What they receive |
|---|---|---|
| Cultured Treg cells | Other | Co-culturing of recipient dendritic cells and donor Treg cells given prior to allogeneic stem cell transplant Receives: Biological: Cultured Treg cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximally Tolerated dose (MTD)
MTD of donor Treg in combination with standard dose SIR/TAC immune suppression. The occurrence of dose-limiting toxicity in >= 33% serves as the boundary for the MTD of donor Treg.
Measured: Up to 1 year
Secondary outcomes (4)
Acute GVHD incidence
Clinical evidence of acute GVHD will be recorded per standard grading scheme. GVHD grade will be reported weekly from day 0-100 both for site-specific involvement, as well as an overall composite score.
Measured: Up to day 100
Relapse Free Survival
Defined as time from transplantation (day 0 as day of stem cell infusion per standard nomenclature) to relapse or death from any cause.
Measured: Up to 1 year
Non-relapse Mortality
Defined as mortality while underlying malignancy is in remission.
Measured: Up to 1 year
Overall Survival (OS)
Defined as time from transplantation (day 0 as day of stem cell infusion per standard nomenclature) to death from any cause.
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2013 (estimated)
- Study start29 Oct 2014
- Primary completion14 Aug 2020
- Study completion14 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Graft Versus Host DiseaseStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Joseph Pidala, MD, PhD, Principal investigator, Moffitt Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01795573
- Sponsor's protocol ID
- MCC-17263
- Status last verified
- Nov 2022
- First submitted
- 18 Feb 2013
- Last updated
- 3 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Pidala JA, Cieri N, Schell MJ, Song X, Shao Y, Cao B, Du D, Thapa R, Betts BC, Janssen W, Cubitt C, Yoder SJ, O'Leary MF, Beato F, Veerapathran A, Menges MA, Burton M, Bejanyan N, Castaneda-Puglianini OA, Elmariah H, Faramand RG, Fernandez HF, Hansen DK, Jain MD, Khimani F, Lazaryan A, Liu HD, Locke FL, Mirza AS, Mishra A, Nieder M, Nishihori T, Ochoa L, Perez LE, Perna F, Ramlal R, Torres NE, Anasetti C. Expanded antigen-specific donor regulatory T cells for GVHD prevention. Blood. 2026 Jun 18:blood.2025030663. doi: 10.1182/blood.2025030663. Online ahead of print.
PubMed 42322116
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Trial of Ex-vivo Expanded Donor Regulatory T Cells for Prevention of Acute Graft-Versus-Host Disease
- ClinicalTrials.gov ID
- NCT01795573
- Acronym / sponsor ID
- MCC-17263
- Conditions
- Graft Versus Host Disease
- Keywords
- GVHD, AML, ALL, MDS, CLL, NHL, HL, MM
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Study start
- 29 Oct 2014 (actual)
- Primary completion
- 14 Aug 2020 (actual)
- Study completion
- 14 Aug 2020 (actual)
- First posted
- 20 Feb 2013 (estimated)
- 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.
Clinical trial of allospecific regulatory t cells (Tregs) for prevention of acute graft-versus-host disease (GVHD) in human leukocyte antigen (HLA) identical sibling transplants.
Detailed description
To evaluate the safety of sirolimus based immune suppression and ex-vivo expanded donor regulatory T cells for the prevention of acute graft-versus-host disease following allogeneic hematopoietic cell transplantation.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cultured Treg cells | Other | Biological: Cultured Treg cellsCo-culturing of recipient dendritic cells and donor Treg cells given prior to allogeneic stem cell transplant |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Signed informed consent
- Diagnoses:
- Peripheral blood white blood count (WBC) greater than 2,000 per microliter (required for collection of dendritic cell precursors)
- Adequate vital organ function: Left ventricular ejection fraction (LVEF) ≥ 45% by multigated acquisition (MUGA) scan or echocardiogram; Forced expiratory volume in one second (FEV1), forced vital capacity (FVC), and diffusing lung capacity oxygenation (DLCO) ≥ 50% of predicted values on pulmonary function tests; Transaminases (AST, ALT) < 3 times upper limit of normal values; Creatinine clearance ≥ 50cc/min
- Infectious disease criteria:
Key exclusion criteria (1 listed)
- Antithymocyte globulin (ATG) as part of the conditioning regimen
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 |
|---|---|---|
| Cultured Treg cellsCo-culturing of recipient dendritic cells and donor Treg. Treg administration will occur 2 days before the allogeneic stem cell transplant (i.e. day -2 with reference of day 0 as stem cell infusion date). | Biological | Cultured Treg cells |
Primary outcome
Maximally Tolerated dose (MTD)
Time frame: Up to 1 year
MTD of donor Treg in combination with standard dose SIR/TAC immune suppression. The occurrence of dose-limiting toxicity in >= 33% serves as the boundary for the MTD of donor Treg.
4 secondary outcomes
Acute GVHD incidence
Time frame: Up to day 100
Relapse Free Survival
Time frame: Up to 1 year
Non-relapse Mortality
Time frame: Up to 1 year
Overall Survival (OS)
Time frame: Up to 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2022.
- Derived from this study: PubMed 42322116Pidala JA, Cieri N, Schell MJ, Song X, Shao Y, Cao B, Du D, Thapa R, Betts BC, Janssen W, Cubitt C, Yoder SJ, O'Leary MF, Beato F, Veerapathran A, Menges MA, Burton M, Bejanyan N, Castaneda-Puglianini OA, Elmariah H, Faramand RG, Fernandez HF, Hansen DK, Jain MD, Khimani F, Lazaryan A, Liu HD, Locke FL, Mirza AS, Mishra A, Nieder M, Nishihori T, Ochoa L, Perez LE, Perna F, Ramlal R, Torres NE, Anasetti C. Expanded antigen-specific donor regulatory T cells for GVHD prevention. Blood. 2026 Jun 18:blood.2025030663. doi: 10.1182/blood.2025030663. Online ahead of print.
- H. Lee Moffitt Cancer Center \& Research InstituteLink provided by the sponsor.
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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.
Joseph Pidala, MD, PhD principal investigator
Moffitt Cancer Center
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 Cultured Treg cells in people with Graft Versus Host Disease, run by H. Lee Moffitt Cancer Center and Research Institute 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 21 inclusion and 1 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 3 Nov 2022. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Maximally Tolerated dose (MTD) (time frame: Up to 1 year). 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 NCT01795573, 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 3 Nov 2022; AzTrials last refreshed its copy on 7 Nov 2026.