Chronic Graft Versus Host DiseasePhase 1/2NCT01954979
Abatacept to Treat Steroid Refractory Chronic Graft Versus Host Disease (cGVHD)
A Phase 1/2 study testing Abatacept in people with Chronic Graft Versus Host Disease, run by Beth Israel Deaconess Medical Center at 2 sites in United States.
Trial summary
The participant is invited to take part in this study because they have chronic Graft versus Host Disease (cGVHD) that is not responding to standard treatment with steroids. This research study is a way of gaining new knowledge about the treatment of patients with cGVHD. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Graft Versus Host Disease
- What is being tested?
- Abatacept
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 2 sitesUnited States
- Study dates
- 27 Mar 2014to 16 Jan 2024
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must meet the following criteria on screening examination to be eligible to participate in the study:
- Participants must be recipients of an allogeneic bone marrow or stem cell transplantation with myeloablative or reduced intensity conditioning regimens.
- Participants must be at least 100 days after the transplantation or a donor lymphocyte infusion.
- Participants must have cGVHD (as defined by the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease)
- Participants may have either extensive or limited cGVHD requiring systemic treatment
- Participants must have steroid refractory cGVHD, defined as having persistent signs and symptoms of chronic GVHD despite the use of prednisone at ≥ 0. 5 mg/kg/day (or equivalent) for at least 4 weeks in the preceding 12 months. Patients may remain on steroids while enrolled in the study.
Show all 30Show fewer
- No addition or subtraction of other immunosuppressive medications for at least 4 weeks prior to starting treatment.
- On stable immunosuppressive regimen for 2 weeks prior to enrollment. Adjustment of immunosuppressive medications to maintain a therapeutic level is permitted.
- Age ≥ 18 years at the time of signing the informed consent form.
- Reproductive Status: Definition of Women of Child-Bearing Potential (WOCBP). WOCBP comprises women who have experienced menarche and who have not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or who are not post-menopausal (see definition below).
- Post-menopause is defined as:
- Women who have had amenorrhea for ≥ 12 consecutive months (without another cause) and who have a documented serum follicle-stimulating hormone (FSH) level > 35 mIU/mL.
- Women who have irregular menstrual periods and a documented serum FSH level > 35 mIU/mL.
- Women who are taking hormone replacement therapy (HRT).
- The following women are WOCBP:
- Women using the following methods to prevent pregnancy: Oral contraceptives, other hormonal contraceptives (vaginal products, skin patches, or implanted or injectable products), or mechanical products such as intrauterine devices or barrier methods (diaphragm, condoms, spermicides).
- Women who are practicing abstinence.
- Women who have a partner who is sterile (eg, due to vasectomy). WOCBP must be using an acceptable method of contraception to avoid pregnancy throughout the study and for up to 10 weeks after the last dose of study drug in such a manner that the risk of pregnancy is minimized.
- WOCBP must have a negative serum or urine pregnancy test result (minimum sensitivity 25 IU/L or equivalent units of HCG) within 0 to 48 hours before the first dose of study drug.
- Women must not be breast-feeding.
- Sexually active fertile men must use effective birth control if their partners are WOCBP.
- Life expectancy of greater than > 3 months.
- ECOG performance status ≤ 2 (see Appendix A).
- Laboratory test results within these ranges:
- Absolute neutrophil count ≥ 1500/mm³
- Serum creatinine ≤ 2.0 mg/dL
- Renal function assessed by calculated creatinine clearance ≥ 60ml/min by Cockcroft-Gault formula (see Appendix B: Cockcroft-Gault estimation of CrCl).
- Total bilirubin ≤ 1.5 x ULN (unless hepatic dysfunction is caused by cGVHD)AST (SGOT) and ALT (SGPT) ≤ 3 x ULN (unless hepatic dysfunction is caused by cGVHD).
- Patients much have a negative PPD skin test and a negative Quantiferon assay.
- Must possess the ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- Participants who exhibit any of the following conditions at screening will not be eligible for admission into the study.
- Any serious medical condition (including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia), laboratory abnormality, or psychiatric illness/ social situation that would prevent the subject from signing the informed consent form or limit compliance with study requirements.
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- Use of any other experimental drug or therapy within 28 days of starting treatment with abatacept.
- Use of biologic antibody therapy for cGVHD with rituximab, alemtuzumab, or ATG within 6 months of starting treatment with abatacept.
- Use of TNF alpha inhibitors within four weeks prior to study entry.
Show all 14Show fewer
- Ongoing prednisone requirement >1 mg/kg/day (or equivalent)
- New immunosuppressive medication or ECP within 28 days of starting treatment with abatacept.
- Donor lymphocyte infusion within 100 days prior to enrollment.
- Active malignant disease relapse or other active malignancy with the exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast.
- Participants who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier.
- Known seropositive for or positive viral load for HIV or positive viral loads for infectious hepatitis, type B (HBV) or C (HCV).
- Uncontrolled intercurrent active infection. Controlled infection on long term suppressive or maintenance therapy is permissible.
- Use of live vaccines within four weeks of starting abatacept.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
About this study
Official title: A Phase I/II Study of Abatacept in the Treatment of Patients With Steroid Refractory Chronic Graft Versus Host Disease (cGVHD)
The participant is invited to take part in this study because they have chronic Graft versus Host Disease (cGVHD) that is not responding to standard treatment with steroids. This research study is a way of gaining new knowledge about the treatment of patients with cGVHD. This research study is evaluating a drug called abatacept.
Abatacept is a drug that alters and suppresses the immune system. Abatacept is approved by the Food and Drug Administration (FDA) for the treatment of moderate to severe active rheumatoid arthritis in adults and of severe juvenile idiopathic arthritis (JIA) in patients who have failed prior therapy with disease-modifying anti-rheumatic drugs (DMARDs). These are autoimmune conditions, ie caused by an overactive immune system that attacks normal tissues and organs. It is currently being tested in a variety of other autoimmune conditions. In this case it is considered experimental.
Read more
cGVHD is caused by the donor cells attacking various organs of the recipient. The investigators try to minimize this immune attack by using corticosteroids such as prednisone. In severe cases prednisone is not sufficient and other immunosuppressive medications are used in addition in order to more efficiently control cGVHD and to limit the dose and consequently the multiple side-effects of corticosteroids. This study is being done to determine if the use of abatacept is safe in patients with cGVHD and if it can facilitate a better control of cGVHD.
During this study the participants will be evaluated for side effects from the treatment with abatacept, and for response of the cGVHD to the treatment. There will be two groups of participants in the study. The first group will be treated at a relatively low dose of abatacept. If this is found to be safe then the second group will be treated at a higher dose. Three to four tablespoons of blood will be drawn at every 2 week visit in order to determine your blood counts, kidney and liver function. Some of the blood will be used in a research lab in order to study measures of your immune system and how they might be affected by the treatment.
Read the detailed description
Before the research starts (screening): After signing the consent form, the participant will be asked to undergo some screening tests or procedures to find out if they can be in the research study. Many of these tests and procedures are likely to be part of regular cGVHD care and may be done even if it turns out that the participant does not take part in the research study. If the participant has had some of these tests or procedures recently, they may or may not have to be repeated.
- Complete Medical History and Physical Examination
- Blood Collection: 3-4 tablespoons of blood will be drawn to measure the participants complete blood counts, kidney, liver and thyroid function
- Disease Assessment: Depending on what organs are affected by their cGVHD the participant may undergo blood tests or a skin exam or an eye exam or a dental exam.
- Electrocardiogram (EKG): a noninvasive test that measures the electrical activity of the participant's heart
- A tuberculin skin test and a quantiferon blood test to rule out tuberculosis
- Pregnancy testing if applicable
- HIV and Hepatitis blood test: 2-3 teaspoons of blood will be collected to perform an HIV test. The participant will be asked to sign a separate consent form for this test. The participant's doctor will discuss the results of this test with the participant and the results will become a part of their permanent medical record. The participant may seek private HIV testing prior to consenting, and based on the results may choose whether or not to participate in this study or have the HIV test become part of their medical records.
If these tests show that the participant is eligible to participate in the research study, the participant will begin the study treatment. If the participant does not meet the eligibility criteria, the participant will not be able to participate in this research study.
Additional research procedures to be performed at the time of screening:
- Research blood testing: to study measures of the participant's immune system
After the screening procedures confirm that the participant is eligible to participate in the research study:
- Since the investigators are looking for the highest dose of the study drug that can be administered safely without severe or unmanageable side effects in participants that have abatacept, not everyone who participates in this research study will receive the same dose of the study drug. The dose the participant gets will depend on the number of participants who have been enrolled in the study before the participant and how well they have tolerated their doses.
- The total duration of treatment on this study is approximately 113 days or 6 treatments. Doses 1-3 will be administered every two weeks. One month following Dose 3, abatacept will be administered every four weeks for the remaining three doses (Doses 4-6.) The treatment will be given intravenously on these days. One month following your last dose of treatment, the participant will be seen in clinic. The participant will be seen in the clinic weekly through dose 4, then every 2 weeks for follow up even if the participant doesn't have treatment.
- The first group of participants will be treated with a lower dose of abatacept. If this dose is determined to be safe then the second group will be treated at a higher dose.
During treatment the participant will be seen in the clinic every two weeks. At every visit the following will be performed:
- Complete Medical History and Physical Examination
- Blood Collection: 3-4 tablespoons of blood will be drawn to measure their complete blood counts, kidney, liver and thyroid function
- Research blood testing: to study measures of the participant's immune system
- On the days of treatment the participant will undergo an assessment of cGVHD depending on what organs are affected
After the final dose of the study drug:
After the participant has completed taking the drug on the study, the following procedures and tests will take place:
- Safety assessment 28 days after the last dose of the study drug.
- Complete Medical History and Physical Examination
- Blood Collection: 3-4 tablespoons of blood will be drawn to measure the participant's complete blood counts, kidney, liver and thyroid function
- Disease Assessment: by physical exam and blood tests if the participant has leukemia or multiple myeloma, or physical exam and PET/CT or CT scan if the participant has lymphoma. These will be done at the safety assessment visit and at the 3 and 6 month follow up visits.
- Electrocardiogram
- Research blood testing: to study measures of your immune system
- Once the participant has completed treatment on study they will be followed monthly for 6 months
- Conditions
- Chronic Graft Versus Host Disease
- Being tested
- Abatacept
- Keywords
- Graft versus Host Disease, ABATACEPT
- Medical subject headings
- Bronchiolitis Obliterans Syndrome, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AbataceptDrug
The study will follow a standard 3+3 design with two escalating doses of abatacept to determine the maximum tolerated dose (MTD): 3 mg/kg (dose level 1) and 10 mg/kg (dose level 2). Dose-limiting toxicities (DLTs) are defined as any Grade 3 or 4 toxicities judged to be probably or definitely related to abatacept.
| Study group | Role | What they receive |
|---|---|---|
| Abatacept | Experimental | Abatacept will be administered for a total of 6 doses. Doses 1-3 will be administered at two week intervals (+/-2 days). One month following Dose 3, abatacept will be administered, and given at four-week intervals (+/-2 days) for three doses (Doses 4-6.) Patients will be followed for toxicity for 28 days following last dose of Abatacept. Patients will then be seen monthly for six months. Receives: Drug: Abatacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Dose-Limiting Toxicities (DLTs)
The study will follow a standard 3+3 design with two escalating doses of abatacept to determine the maximum tolerated dose (MTD): 3 mg/kg (dose level 1) and 10 mg/kg (dose level 2). Dose-limiting toxicities (DLTs) are defined as any Grade 3 or 4 toxicities judged to be probably or definitely related to abatacept.
Measured: The first 8 weeks of treatment
Secondary outcomes (2)
Determination of the Efficacy of a 141 Day/6 Dose Course of Abatacept in Patients With Steroid Refractory cGVHD (Phase 2 Cohort Only)
Following identification of MTD, the study expanded to a phase 2 cohort. One outcome of the phase 2 cohort was efficacy. Determination of the efficacy (in terms of cGVHD symptoms, score and steroid dose) of a 141 day/6 dose course of abatacept in patients with steroid refractory cGVHD.
Measured: 1 month following 6th dose of abatacept
Examination of the Immunologic Effects (Changes in % of Cells Expressing CD4 and PD1) Associated With the Administration of Abatacept in Patients With Steroid Refractory cGVHD (Phase 2 Cohort Only)
Following identification of MTD, the study expanded to a phase 2 cohort. One outcome of the phase 2 cohort was immunologic effects of abatacept. This was evaluated through flow cytometry to measure changes in % of cells expressing CD4 and PD1.
Measured: 1 month after 6th dose of abatacept
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2013 (estimated)
- Study start27 Mar 2014
- Primary completion1 Jun 2023
- Study completion16 Jan 2024
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 (2)Papers the sponsor has linked to this study.
- Other trials for Chronic Graft Versus Host DiseaseStudies of the same condition, open and under way.
- More trials run by Beth Israel Deaconess Medical CenterThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Beth Israel Deaconess Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Bristol-Myers Squibb, Dana-Farber Cancer Institute
- Responsible party
- Principal investigator, Jacalyn Rosenblatt, Beth Israel Deaconess Medical Center
- Investigators
- Jacalyn Rosenblatt, MD, Principal investigator, Beth Israel Deaconess Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01954979
- Sponsor's protocol ID
- 13-358
- Status last verified
- May 2026
- First submitted
- 27 Sep 2013
- Last updated
- 29 May 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Koshy AG, Kim HT, Liegel J, Arnason J, Ho VT, Antin JH, Joyce R, Cutler C, Gooptu M, Nikiforow S, Logan EK, Elavalakanar P, Narcis M, Stroopinsky D, Avigan ZM, Boussi L, Stephenson S, El Banna H, Bindal P, Cheloni G, Avigan DE, Soiffer RJ, Rosenblatt J. Phase 2 clinical trial evaluating abatacept in patients with steroid-refractory chronic graft-versus-host disease. Blood. 2023 Jun 15;141(24):2932-2943. doi: 10.1182/blood.2022019107.
PubMed 36862975Nahas MR, Soiffer RJ, Kim HT, Alyea EP 3rd, Arnason J, Joyce R, Antin JH, Ho VT, Stroopinsky D, Li S, Levine JD, McMasters M, Jain S, Hamdan A, Tzachanis D, Bryant MP, Logan EK, Bazemore J, Stewart J, Joyce A, Stephenson S, Washington A, Cole L, Pyzer A, Leaf RK, Avigan DE, Rosenblatt J. Phase 1 clinical trial evaluating abatacept in patients with steroid-refractory chronic graft-versus-host disease. Blood. 2018 Jun 21;131(25):2836-2845. doi: 10.1182/blood-2017-05-780239. Epub 2018 Mar 16.
PubMed 29549175
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of Abatacept in the Treatment of Patients With Steroid Refractory Chronic Graft Versus Host Disease (cGVHD)
- ClinicalTrials.gov ID
- NCT01954979
- Acronym / sponsor ID
- 13-358
- Conditions
- Chronic Graft Versus Host Disease
- Keywords
- Graft versus Host Disease, ABATACEPT
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Beth Israel Deaconess Medical CenterUniversity, hospital or other
- Collaborators
- Bristol-Myers Squibb, Dana-Farber Cancer Institute
- Study start
- 27 Mar 2014 (actual)
- Primary completion
- 1 Jun 2023 (actual)
- Study completion
- 16 Jan 2024 (actual)
- First posted
- 7 Oct 2013 (estimated)
- Results
- Posted 29 May 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The participant is invited to take part in this study because they have chronic Graft versus Host Disease (cGVHD) that is not responding to standard treatment with steroids. This research study is a way of gaining new knowledge about the treatment of patients with cGVHD. This research study is evaluating a drug called abatacept.
Abatacept is a drug that alters and suppresses the immune system. Abatacept is approved by the Food and Drug Administration (FDA) for the treatment of moderate to severe active rheumatoid arthritis in adults and of severe juvenile idiopathic arthritis (JIA) in patients who have failed prior therapy with disease-modifying anti-rheumatic drugs (DMARDs). These are autoimmune conditions, ie caused by an overactive immune system that attacks normal tissues and organs. It is currently being tested in a variety of other autoimmune conditions. In this case it is considered experimental.
cGVHD is caused by the donor cells attacking various organs of the recipient. The investigators try to minimize this immune attack by using corticosteroids such as prednisone. In severe cases prednisone is not sufficient and other immunosuppressive medications are used in addition in order to more efficiently control cGVHD and to limit the dose and consequently the multiple side-effects of corticosteroids. This study is being done to determine if the use of abatacept is safe in patients with cGVHD and if it can facilitate a better control of cGVHD.
During this study the participants will be evaluated for side effects from the treatment with abatacept, and for response of the cGVHD to the treatment. There will be two groups of participants in the study. The first group will be treated at a relatively low dose of abatacept. If this is found to be safe then the second group will be treated at a higher dose. Three to four tablespoons of blood will be drawn at every 2 week visit in order to determine your blood counts, kidney and liver function. Some of the blood will be used in a research lab in order to study measures of your immune system and how they might be affected by the treatment.
Detailed description
Before the research starts (screening): After signing the consent form, the participant will be asked to undergo some screening tests or procedures to find out if they can be in the research study. Many of these tests and procedures are likely to be part of regular cGVHD care and may be done even if it turns out that the participant does not take part in the research study. If the participant has had some of these tests or procedures recently, they may or may not have to be repeated.
- Complete Medical History and Physical Examination
- Blood Collection: 3-4 tablespoons of blood will be drawn to measure the participants complete blood counts, kidney, liver and thyroid function
- Disease Assessment: Depending on what organs are affected by their cGVHD the participant may undergo blood tests or a skin exam or an eye exam or a dental exam.
- Electrocardiogram (EKG): a noninvasive test that measures the electrical activity of the participant's heart
- A tuberculin skin test and a quantiferon blood test to rule out tuberculosis
- Pregnancy testing if applicable
- HIV and Hepatitis blood test: 2-3 teaspoons of blood will be collected to perform an HIV test. The participant will be asked to sign a separate consent form for this test. The participant's doctor will discuss the results of this test with the participant and the results will become a part of their permanent medical record. The participant may seek private HIV testing prior to consenting, and based on the results may choose whether or not to participate in this study or have the HIV test become part of their medical records.
If these tests show that the participant is eligible to participate in the research study, the participant will begin the study treatment. If the participant does not meet the eligibility criteria, the participant will not be able to participate in this research study.
Additional research procedures to be performed at the time of screening:
- Research blood testing: to study measures of the participant's immune system
After the screening procedures confirm that the participant is eligible to participate in the research study:
- Since the investigators are looking for the highest dose of the study drug that can be administered safely without severe or unmanageable side effects in participants that have abatacept, not everyone who participates in this research study will receive the same dose of the study drug. The dose the participant gets will depend on the number of participants who have been enrolled in the study before the participant and how well they have tolerated their doses.
- The total duration of treatment on this study is approximately 113 days or 6 treatments. Doses 1-3 will be administered every two weeks. One month following Dose 3, abatacept will be administered every four weeks for the remaining three doses (Doses 4-6.) The treatment will be given intravenously on these days. One month following your last dose of treatment, the participant will be seen in clinic. The participant will be seen in the clinic weekly through dose 4, then every 2 weeks for follow up even if the participant doesn't have treatment.
- The first group of participants will be treated with a lower dose of abatacept. If this dose is determined to be safe then the second group will be treated at a higher dose.
During treatment the participant will be seen in the clinic every two weeks. At every visit the following will be performed:
- Complete Medical History and Physical Examination
- Blood Collection: 3-4 tablespoons of blood will be drawn to measure their complete blood counts, kidney, liver and thyroid function
- Research blood testing: to study measures of the participant's immune system
- On the days of treatment the participant will undergo an assessment of cGVHD depending on what organs are affected
After the final dose of the study drug:
After the participant has completed taking the drug on the study, the following procedures and tests will take place:
- Safety assessment 28 days after the last dose of the study drug.
- Complete Medical History and Physical Examination
- Blood Collection: 3-4 tablespoons of blood will be drawn to measure the participant's complete blood counts, kidney, liver and thyroid function
- Disease Assessment: by physical exam and blood tests if the participant has leukemia or multiple myeloma, or physical exam and PET/CT or CT scan if the participant has lymphoma. These will be done at the safety assessment visit and at the 3 and 6 month follow up visits.
- Electrocardiogram
- Research blood testing: to study measures of your immune system
- Once the participant has completed treatment on study they will be followed monthly for 6 months
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Abatacept | Experimental | Drug: AbataceptAbatacept will be administered for a total of 6 doses. Doses 1-3 will be administered at two week intervals (+/-2 days). One month following Dose 3, abatacept will be administered, and given at four-week intervals (+/-2 days) for three doses (Doses 4-6.) Patients will be followed for toxicity for 28 days following last dose of Abatacept. Patients will then be seen monthly for six months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (30 listed)
- Participants must meet the following criteria on screening examination to be eligible to participate in the study:
- Participants must be recipients of an allogeneic bone marrow or stem cell transplantation with myeloablative or reduced intensity conditioning regimens.
- Participants must be at least 100 days after the transplantation or a donor lymphocyte infusion.
- Participants must have cGVHD (as defined by the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease)
- Participants may have either extensive or limited cGVHD requiring systemic treatment
Key exclusion criteria (14 listed)
- Participants who exhibit any of the following conditions at screening will not be eligible for admission into the study.
- Any serious medical condition (including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia), laboratory abnormality, or psychiatric illness/ social situation that would prevent the subject from signing the informed consent form or limit compliance with study requirements.
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- Use of any other experimental drug or therapy within 28 days of starting treatment with abatacept.
- Use of biologic antibody therapy for cGVHD with rituximab, alemtuzumab, or ATG within 6 months of starting treatment with abatacept.
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 |
|---|---|---|
| AbataceptThe study will follow a standard 3+3 design with two escalating doses of abatacept to determine the maximum tolerated dose (MTD): 3 mg/kg (dose level 1) and 10 mg/kg (dose level 2). Dose-limiting toxicities (DLTs) are defined as any Grade 3 or 4 toxicities judged to be probably or definitely related to abatacept. | Drug | Abatacept |
Primary outcome
Number of Participants With Dose-Limiting Toxicities (DLTs)
Time frame: The first 8 weeks of treatment
The study will follow a standard 3+3 design with two escalating doses of abatacept to determine the maximum tolerated dose (MTD): 3 mg/kg (dose level 1) and 10 mg/kg (dose level 2). Dose-limiting toxicities (DLTs) are defined as any Grade 3 or 4 toxicities judged to be probably or definitely related to abatacept.
2 secondary outcomes
Determination of the Efficacy of a 141 Day/6 Dose Course of Abatacept in Patients With Steroid Refractory cGVHD (Phase 2 Cohort Only)
Time frame: 1 month following 6th dose of abatacept
Examination of the Immunologic Effects (Changes in % of Cells Expressing CD4 and PD1) Associated With the Administration of Abatacept in Patients With Steroid Refractory cGVHD (Phase 2 Cohort Only)
Time frame: 1 month after 6th dose of abatacept
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36862975Koshy AG, Kim HT, Liegel J, Arnason J, Ho VT, Antin JH, Joyce R, Cutler C, Gooptu M, Nikiforow S, Logan EK, Elavalakanar P, Narcis M, Stroopinsky D, Avigan ZM, Boussi L, Stephenson S, El Banna H, Bindal P, Cheloni G, Avigan DE, Soiffer RJ, Rosenblatt J. Phase 2 clinical trial evaluating abatacept in patients with steroid-refractory chronic graft-versus-host disease. Blood. 2023 Jun 15;141(24):2932-2943. doi: 10.1182/blood.2022019107.
- Derived from this study: PubMed 29549175Nahas MR, Soiffer RJ, Kim HT, Alyea EP 3rd, Arnason J, Joyce R, Antin JH, Ho VT, Stroopinsky D, Li S, Levine JD, McMasters M, Jain S, Hamdan A, Tzachanis D, Bryant MP, Logan EK, Bazemore J, Stewart J, Joyce A, Stephenson S, Washington A, Cole L, Pyzer A, Leaf RK, Avigan DE, Rosenblatt J. Phase 1 clinical trial evaluating abatacept in patients with steroid-refractory chronic graft-versus-host disease. Blood. 2018 Jun 21;131(25):2836-2845. doi: 10.1182/blood-2017-05-780239. Epub 2018 Mar 16.
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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.
Jacalyn Rosenblatt, MD principal investigator
Beth Israel Deaconess Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Abatacept in people with Chronic Graft Versus Host Disease, run by Beth Israel Deaconess Medical Center at 2 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 30 inclusion and 14 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 29 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 2 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 combined 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.
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 29 May 2026). 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?
Number of Participants With Dose-Limiting Toxicities (DLTs) (time frame: The first 8 weeks of treatment). 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 NCT01954979, which also lists 2 linked publications.
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 May 2026.