Graft Vs Host DiseasePhase 2ABA3NCT04380740
Extended vs Short-term Abatacept Dosing for Graft Versus Host Disease Prophylaxis
A Phase 2 study testing Abatacept in people with Graft Vs Host Disease, run by Boston Children's Hospital at 15 sites in United States.
Trial summary
This is a multicenter randomized, double blind, Phase 2 trial for patients receiving transplants from 7 of 8 HLA matched donors, in which an extended dosing regimen of abatacept, and a short-term dosing regimen + placebo, when added to standard calcineurin inhibitor + methotrexate-based prophylaxis, will be compared… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Graft Vs Host Disease
- What is being tested?
- Abatacept
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 160 participants
- Where
- 15 sitesUnited States
- Study dates
- 30 Mar 2022to Jun 2028
- Phase
- Phase 2
- 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
- Must be at least 2 years old and weigh 10 kg.
- Must have a willing unrelated adult donor (bone marrow or peripheral blood). Donors may have a single mismatch (i.e. be a 7/8) and this mismatch may be at the allele or antigen level; however, donors with allele level disparity should be given preference over those with antigen level disparity. Patients for whom a donor is available with disparity only in the host versus graft direction (because of recipient homozygosity), will not be eligible, since this mismatching does not increase the risk for GVHD. Centers may perform extended typing (e.g. DQB1 and DPB1) according to institutional practices and use these results in selecting donors; however, it is recommended that this extending typing be used only to select between donors who are equally well matched with the recipient at the A, B, C and DRB1.
- All patients and/or their parents or legal guardians must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
- Must have a hematologic malignancy treatable by HCT (except for those stipulated below under study Exclusion Criteria), which is in remission by standard testing (no patients in relapse will be included).
- Patients with an inherited predisposition to leukemia or otherwise hematologic malignancies that have not been associated with predisposition to transplant morbidities or non-hematologic cancers.
- Karnofsky performance score or Lanskey Play-Performance Scale score >/= 80.
- If the patient does not meet defined eligibility requirements, the PI/study committee must be contacted to determine eligibility.
Who cannot join
- Patients with the following hematologic malignancies will be excluded: Chronic Lymphocytic Leukemia, Myeloma and Primary Myelofibrosis.
- Active Relapse (>5% blasts) of their primary malignancy.
- For patients with Acute Lymphocytic Leukemia (ALL) with pre-transplant MRD testing performed as standard practice at the treating institution, patients with MRD >0.01% will be ineligible.
- For patients with Acute Myeloid Leukemia (AML) with pre-transplant MRD testing as standard of practice at the treating institution, patients with any MRD status are eligible and should be enrolled at the discretion of provider.
- For patients with MDS, those with >5% blasts will be excluded.
- Prior allogeneic HCT.
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- Uncontrolled viral, bacterial, fungal or protozoal infection at the time of study enrollment.
- HIV infection.
- Serious psychiatric disease including schizophrenia, bipolar disorder and severe depression.
- Prisoners or others who are compulsorily detained.
- Any patient with a known or suspected inherited predisposition to cancer should be discussed with the study team prior to screening for eligibility.
- Patients with a known inherited or constitutional predisposition to transplant morbidities, including, but not limited to Fanconi Anemia, Dyskeratosis Congenita, Shwachman-Diamond Syndrome and Down Syndrome will be excluded.
- Patients with known inherited or constitutional predisposition to non-hematologic cancers including, but not limited to Li-Fraumeni syndrome, BRCA1 and BRCA2 mutations will be excluded.
- Patients with active non-hematological malignancies (except non-melanoma skin cancers) or those with non-hematological malignancies (except non-melanoma skin cancers) who have been rendered with no evidence of disease, and are disease free for <2 years.
- Incompletely treated active tuberculosis Infection.
- Pregnancy (positive serum b-HCG) or breastfeeding.
- Estimated GFR of < 50 mL/min/1.73m2.
- Cardiac ejection fraction < 50 (using M-Mode if assessment is done by ECHO)
- T.bilirubin > 2 × upper limit of normal or ALT > 4 × upper limit of normal or unresolved veno-occlusive disease.
- Pulmonary disease with FVC, FEV1 or DLCO parameters <45% predicted (corrected for hemoglobin) or requiring supplemental oxygen. Children who are developmentally unable to perform pulmonary function testing will be assessed solely on their need for supplemental oxygen.
- Presence of antibodies to a mismatched donor HLA antigen (please refer to Section 3.4.g).
- Patients who have developed severe AGVHD, severe CGVHD or relapse will be excluded at the time of randomization.
- Exclusion Criteria Prior to Randomization (prior to 5th dose of abatacept/placebo):
- Severe allergic reaction during the first 4 doses of abatacept
- If any clinical events occur that preclude further dosing of abatacept, those patients will be deemed ineligible for randomization
Trial locations and contacts
15 sites in 1 country. Contact the study team or a site near you to ask about taking part.
15 sites in 1 country
United States15 sites, 4 recruiting
- Active, not recruiting
University of Michigan
Ann Arbor, Michigan, 48109
- Active, not recruiting
Emory University/Winship Cancer Center
Atlanta, Georgia, 30322
- Recruiting
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
Muna Qayed, MD, 404-785-1112, mqayed@emory.edu
Muna Qayed, MD
- Active, not recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
Brandi Bratrude, BA, 6179192197, brandi.bratrude@childrens.harvard.edu
Leslie S Kean, MD, PhD
- Active, not recruiting
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
- Active, not recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
- Recruiting
University of Chicago
Chicago, Illinois, 60637
James LaBelle, MD
James LaBelle, MD
- Active, not recruiting
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
- Active, not recruiting
City Of Hope National Medical Center
Duarte, California, 91010
- Withdrawn
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Active, not recruiting
Oregon Health and Sciences University
Portland, Oregon, 97239
- Recruiting
University of Rochester
Rochester, New York, 14642
Eric Huselton, MD, Eric_Huselton@URMC.Rochester.edu
Eric Huselton, MD
- Withdrawn
Washington University St. Louis
St Louis, Missouri, 63110
- Active, not recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
About this study
Official title: A Randomized Double-Blind Trial of Abatacept Extended Dosing Versus Abatacept Short-term Dosing for Graft Versus Host Disease Prophylaxis: "ABA3"
This is a multicenter randomized, double blind, Phase 2 trial for patients receiving transplants from 7 of 8 HLA matched donors, in which an extended dosing regimen of abatacept, and a short-term dosing regimen + placebo, when added to standard calcineurin inhibitor + methotrexate-based prophylaxis, will be compared for their ability to improve outcomes in patients with a minimum follow-up of one year post-transplant. All patients will receive 4 doses of abatacept (Days -1, +5, +14, +28). Prior to the fifth dose, patients will be randomly assigned to the 4-dose abatacept arm and receive 4 doses of placebo or 8-dose abatacept arm and receive 4 more doses of abatacept. The primary endpoint of the study will be severe AGVHD-free, severe CGVHD-free, relapse-free survival (SGRFS). The study will end when the last patient has reached 2 years after transplant. Results will first be calculated and the study unblinded when the last patient has reached one year post-transplant.
- Conditions
- Graft Vs Host Disease
- Being tested
- Placebo, Abatacept
- 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.
PlaceboDrug
Also called saline
During the extended dosing of abatacept, those randomized to receive 4 doses will receive a placebo consisting of an equal volume of normal saline solution.
AbataceptDrug
Also called orencia
Investigational prophylaxis with extended-dosing abatacept, a calcineurin inhibitor and methotrexate.
| Study group | Role | What they receive |
|---|---|---|
| Standard GVHD Prophylaxis + Abatacept + Placebo | Placebo comparator | Standard GVHD prophylaxis of calcineurin inhibitor (cyclosporine or tacrolimus) and methotrexate + 4 doses of Abatacept (investigational product) + 4 doses of Placebo. Receives: Drug: Placebo; Drug: Abatacept |
| Standard GVHD Prophylaxis + Abatacept Extended dosing | Experimental | Standard GVHD prophylaxis of calcineurin inhibitor (cyclosporine or tacrolimus) and methotrexate + 8 doses of Abatacept. Receives: Drug: Abatacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Severe AGVHD-free, severe CGVHD-free, relapse-free survival (SGRFS)
SGRFS will be modeled as a time-to-event outcome, and as such, failures that occur beyond one year and before study end will be considered in the analysis.
Measured: 2 years
Secondary outcomes (3)
Severe Chronic GVHD
We will compare the cause-specific hazards of severe chronic GVHD, including overlap syndrome (based on adjudicated events), using the NIH consensus criteria, between the two arms of the study.
Measured: 2 years
Relapse-Free survival
We will compare the hazards of failure (earliest of relapse or any-cause death) for relapse-free survival (RFS), which will be defined as survival without relapse of underlying malignancy.
Measured: 2 years
Non-relapse mortality
We will compare the cause-specific hazards of non-relapse mortality (NRM), which will be defined as death without a prior relapse, with relapse defined as either morphological or standard cytogenetic evidence of acute leukemia or MDS consistent with pre-transplant features, or radiologic evidence of lymphoma, documented or not by biopsy.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2020
- Study start30 Mar 2022
- Primary completionMay 2027 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft Vs Host DiseaseStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 160 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Responsible party
- Principal investigator, Leslie Kean, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04380740
- Sponsor's protocol ID
- IRB-P00035528
- Other IDs
- 20-227
- Acronym
- ABA3
- Status last verified
- May 2026
- First submitted
- 5 May 2020
- Last updated
- 11 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Dana-Farber/Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscripts may only be shared under the terms of a Data Use Agreement. Requests may be directed to aba3study@childrens.harvard.edu. The protocol and statistical analysis plan will be made available on clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Double-Blind Trial of Abatacept Extended Dosing Versus Abatacept Short-term Dosing for Graft Versus Host Disease Prophylaxis: "ABA3"
- ClinicalTrials.gov ID
- NCT04380740
- Acronym / sponsor ID
- ABA3 / IRB-P00035528
- Conditions
- Graft Vs Host Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 160 participants (estimated)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Study start
- 30 Mar 2022 (actual)
- Primary completion
- May 2027 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 8 May 2020 (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.
This is a multicenter randomized, double blind, Phase 2 trial for patients receiving transplants from 7 of 8 HLA matched donors, in which an extended dosing regimen of abatacept, and a short-term dosing regimen + placebo, when added to standard calcineurin inhibitor + methotrexate-based prophylaxis, will be compared for their ability to improve outcomes in patients with a minimum follow-up of one year post-transplant. All patients will receive 4 doses of abatacept (Days -1, +5, +14, +28). Prior to the fifth dose, patients will be randomly assigned to the 4-dose abatacept arm and receive 4 doses of placebo or 8-dose abatacept arm and receive 4 more doses of abatacept. The primary endpoint of the study will be severe AGVHD-free, severe CGVHD-free, relapse-free survival (SGRFS). The study will end when the last patient has reached 2 years after transplant. Results will first be calculated and the study unblinded when the last patient has reached one year post-transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard GVHD Prophylaxis + Abatacept + Placebo | Placebo comparator | Drug: Placebo, Drug: AbataceptStandard GVHD prophylaxis of calcineurin inhibitor (cyclosporine or tacrolimus) and methotrexate + 4 doses of Abatacept (investigational product) + 4 doses of Placebo. |
| Standard GVHD Prophylaxis + Abatacept Extended dosing | Experimental | Drug: AbataceptStandard GVHD prophylaxis of calcineurin inhibitor (cyclosporine or tacrolimus) and methotrexate + 8 doses of Abatacept. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Must be at least 2 years old and weigh 10 kg.
- Must have a willing unrelated adult donor (bone marrow or peripheral blood). Donors may have a single mismatch (i.e. be a 7/8) and this mismatch may be at the allele or antigen level; however, donors with allele level disparity should be given preference over those with antigen level disparity. Patients for whom a donor is available with disparity only in the host versus graft direction (because of recipient homozygosity), will not be eligible, since this mismatching does not increase the risk for GVHD. Centers may perform extended typing (e.g. DQB1 and DPB1) according to institutional practices and use these results in selecting donors; however, it is recommended that this extending typing be used only to select between donors who are equally well matched with the recipient at the A, B, C and DRB1.
- All patients and/or their parents or legal guardians must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
- Must have a hematologic malignancy treatable by HCT (except for those stipulated below under study Exclusion Criteria), which is in remission by standard testing (no patients in relapse will be included).
- Patients with an inherited predisposition to leukemia or otherwise hematologic malignancies that have not been associated with predisposition to transplant morbidities or non-hematologic cancers.
Key exclusion criteria (25 listed)
- Patients with the following hematologic malignancies will be excluded: Chronic Lymphocytic Leukemia, Myeloma and Primary Myelofibrosis.
- Active Relapse (>5% blasts) of their primary malignancy.
- For patients with Acute Lymphocytic Leukemia (ALL) with pre-transplant MRD testing performed as standard practice at the treating institution, patients with MRD >0.01% will be ineligible.
- For patients with Acute Myeloid Leukemia (AML) with pre-transplant MRD testing as standard of practice at the treating institution, patients with any MRD status are eligible and should be enrolled at the discretion of provider.
- For patients with MDS, those with >5% blasts will be excluded.
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 |
|---|---|---|
| PlaceboAlso: salineDuring the extended dosing of abatacept, those randomized to receive 4 doses will receive a placebo consisting of an equal volume of normal saline solution. | Drug | Standard GVHD Prophylaxis + Abatacept + Placebo |
| AbataceptAlso: orenciaInvestigational prophylaxis with extended-dosing abatacept, a calcineurin inhibitor and methotrexate. | Drug | Standard GVHD Prophylaxis + Abatacept + Placebo, Standard GVHD Prophylaxis + Abatacept Extended dosing |
Primary outcome
Severe AGVHD-free, severe CGVHD-free, relapse-free survival (SGRFS)
Time frame: 2 years
SGRFS will be modeled as a time-to-event outcome, and as such, failures that occur beyond one year and before study end will be considered in the analysis.
3 secondary outcomes
Severe Chronic GVHD
Time frame: 2 years
Relapse-Free survival
Time frame: 2 years
Non-relapse mortality
Time frame: 2 years
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 11 May 2026.
- Individual participant dataSharing planned: Yes. The Dana-Farber/Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscripts may only be shared under the terms of a Data Use Agreement. Requests may be directed to aba3study@childrens.harvard.edu. The protocol and statistical analysis plan will be made available on clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
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Graft vs Host Disease
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.
15 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 Abatacept in people with Graft Vs Host Disease, run by Boston Children's Hospital at 15 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 7 inclusion and 25 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 11 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 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. Some participants in this study may receive a placebo. 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?
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 M Bratrude, BA (6179192197, 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?
Severe AGVHD-free, severe CGVHD-free, relapse-free survival (SGRFS) (time frame: 2 years). 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 11 May 2026.