Graft Vs Host DiseasePhase 2NCT05621759
Cyclophosphamide, Abatacept, and Tacrolimus for the Prevention of GvHD
A Phase 2 study testing Cyclophosphamide, Abatacept and Tacrolimus in people with Graft Vs Host Disease, run by NYU Langone Health at 1 site in United States.
Trial summary
This is a single arm, open label, phase II clinical trial. Adult patients with hematological malignancies undergoing allogeneic HSCT from first- or second-degree haploidentical donor are eligible for the study if they meet the standard criteria defined in our institutional standard operation procedures (SOPs), meet… Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Graft Vs Host Disease
- What is being tested?
- Cyclophosphamide, Abatacept and Tacrolimus
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Sep 2022to 30 Jan 2027
- 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 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years
- Karnofsky score ≥ 70%
- No evidence of progressive bacterial, viral, or fungal infection
- Creatinine clearance > 50 mL/min/1.72m2
- Total bilirubin, Alanine Aminotransferase and Aspartate Aminotransferase < 2 x the upper limit of normal (except for diagnosed Gilbert's syndrome)
- Alkaline phosphatase ≤ 250 IU/L
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- Left Ventricular Ejection Fraction (LVEF) > 45%
- Adjusted Carbon Monoxide Diffusing Capacity (DLCO) > 60%
- Negative HIV serology
- Negative pregnancy test: confirmation per negative serum β-human chorionic gonadotropin (β-hCG) for women of childbearing age and potential.
Who cannot join
- Donors are excluded in case of donor-specific HLA antibodies or positive cross-match.
- Pregnant or nursing females or women of child bearing age or potential, who are unwilling to completely abstain from heterosexual sex or practice 2 effective methods of contraception from the first dose of conditioning regimen through day +180. A woman of reproductive capability is one who has not undergone a hysterectomy (removal of the womb), has not had both ovaries removed, or has not been post-menopausal (stopped menstrual periods) for more than 24 months in a row.
- Male subjects who refuse to practice effective barrier contraception during the entire study treatment period and through a minimum of 90 days after the last dose of study drug, or completely abstain from heterosexual intercourse. This must be done even if they are surgically sterilized (i.e., post-vasectomy).
- Inability to provide informed consent.
- Patient had myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure (see Appendix E), uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Prior to study entry, any ECG abnormality at screening must be documented by the investigator as not medically relevant.
- Known allergies to any of the components of the investigational treatment regimen.
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- Serious medical or psychiatric illness likely to interfere with participation in this clinical study.
- Participation in clinical trials with other investigational agents not included in this trial, within 14 days of the start of this trial and throughout the duration of this trial.
- Prisoners
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Single-Arm Open-Label Study Of Reduced-Dose Post-Transplant Cyclophosphamide, Abatacept, and Short-Duration Tacrolimus for the Prevention of Graft-Versus-Host Disease (GVHD) Following Haploidentical Hematopoietic Stem Cell Transplantation (HSCT)
This is a single arm, open label, phase II clinical trial. Adult patients with hematological malignancies undergoing allogeneic HSCT from first- or second-degree haploidentical donor are eligible for the study if they meet the standard criteria defined in our institutional standard operation procedures (SOPs), meet all inclusion criteria, and do not satisfy any exclusion criteria. Patients will receive non-myeloablative, reduced-intensity or myeloablative conditioning regimen followed by peripheral blood hematopoietic stem cells. Patients will receive dosed reduced cyclophosphamide, abatacept, and short-duration tacrolimus for GvHD prophylaxis.
- Conditions
- Graft Vs Host Disease
- Being tested
- Cyclophosphamide, Abatacept, Tacrolimus
- Keywords
- Haploidentical Hematopoietic Stem Cell Transplantation
- 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.
CyclophosphamideDrug
Also called Cytoxan
Nitrogen mustard alkylating agent produced by Bristol-Myers Squibb.
AbataceptDrug
Also called Prograf
Calcineurin-inhibitor produced by Astellas.
TacrolimusDrug
Also called Orencia
Selective T cell co-stimulation modulator produced by Bristol-Myers Squibb.
| Study group | Role | What they receive |
|---|---|---|
| Reduced-Dose Post-Transplant Cyclophosphamide, Abatacept, and Short-Duration Tacrolimus | Experimental | Participants to receive:
* Cyclophosphamide 25 mg/kg IV over 1 hour on Day 3 and Day 4 following transplant
* Abatacept 10 mg/kg IV on Day 5, Day 14, Day 28, and Day 56 following transplant
* Tacrolimus 0.02 mg/kg IV by continuous infusion, starting on Day 5 following transplant. May switch to oral administration when tolerated, adjusted to maintain a drug level between 5-12ng/mL. Tacrolimus treatment is continued until Day 60 and then tapered over a period of 4 weeks in the absence of GvHD. Receives: Drug: Cyclophosphamide; Drug: Abatacept; Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative Incidence of Grades II-IV Acute GvHD
The first day of acute GvHD of any grade is used to calculate the cumulative incidence for that grade. The diagnosis of acute GvHD is based on clinical and pathological evaluation by the principal investigator in collaboration with the treating physician.
Measured: Up to Day 120
Secondary outcomes (8)
Cumulative Incidence of Chronic GvHD
The first day of chronic GvHD is used to calculate the cumulative incidence of chronic GvHD. The diagnosis of chronic GvHD is based on clinical and pathological evaluation by the principal investigator in collaboration with the treating physician.
Measured: Up to Day 365
Number of Participants Presenting with Primary Graft Failure
Primary graft failure is defined as failure to achieve neutrophil engraftment by Day 28 after transplant or lack of donor chimerism greater than 50% by Day 45, not due to the underlying malignancy.
Measured: Up to Day 45
Number of Participants Presenting with Poor Graft Function
Poor graft function is defined by at least 2 of the following 3 criteria: Hemoglobin less than 8 g/dL, absolute neutrophil count less than 0.5 x 109/L, and platelets less than 20 x 109/L. The cytopenia must be unexplained (such as by disease relapse) and unresponsive to cytokines and must last at least 4 weeks.
Measured: Up to Day 30
Number of Participants Presenting with Secondary Graft Failure
Secondary graft failure is defined as poor graft function associated with donor chimerism less than 5%.
Measured: Up to Day 730
Treatment-Related Mortality (TRM) Rate
The proportion of participant deaths not attributable to disease relapse or progression.
Measured: Up to Day 730
Relapse Rate (RR)
The proportion of participants in whom the disease for which transplant was performed is evident by methods of disease detection.
Measured: Up to Day 730
GvHD and Relapse-Free Survival (GRFS) Rate
The proportion of participants who are without reported grade III-IV acute GvHD, chronic GvHD requiring systemic therapy and have not experienced relapse or death.
Measured: Up to Day 730
Overall Survival (OS) Rate
The proportion of participants who are alive at the end of the study's evaluation period.
Measured: Up to Day 730
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2022
- Study start7 Sep 2022
- Primary completion30 Jan 2027 (estimated)
- Study completion30 Jan 2027 (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 NYU Langone HealthThe 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mohammad Maher Abdul Hay, Principal investigator, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05621759
- Sponsor's protocol ID
- 22-00674
- Status last verified
- Jan 2026
- First submitted
- 10 Nov 2022
- Last updated
- 29 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Only available to study team members.
Study at a glance
From the registry record, last updated by the sponsor on 29 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Single-Arm Open-Label Study Of Reduced-Dose Post-Transplant Cyclophosphamide, Abatacept, and Short-Duration Tacrolimus for the Prevention of Graft-Versus-Host Disease (GVHD) Following Haploidentical Hematopoietic Stem Cell Transplantation (HSCT)
- ClinicalTrials.gov ID
- NCT05621759
- Acronym / sponsor ID
- 22-00674
- Conditions
- Graft Vs Host Disease
- Keywords
- Haploidentical Hematopoietic Stem Cell Transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 27 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- 7 Sep 2022 (actual)
- Primary completion
- 30 Jan 2027 (estimated)
- Study completion
- 30 Jan 2027 (estimated)
- First posted
- 18 Nov 2022 (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 single arm, open label, phase II clinical trial. Adult patients with hematological malignancies undergoing allogeneic HSCT from first- or second-degree haploidentical donor are eligible for the study if they meet the standard criteria defined in our institutional standard operation procedures (SOPs), meet all inclusion criteria, and do not satisfy any exclusion criteria. Patients will receive non-myeloablative, reduced-intensity or myeloablative conditioning regimen followed by peripheral blood hematopoietic stem cells. Patients will receive dosed reduced cyclophosphamide, abatacept, and short-duration tacrolimus for GvHD prophylaxis.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Reduced-Dose Post-Transplant Cyclophosphamide, Abatacept, and Short-Duration Tacrolimus | Experimental | Drug: Cyclophosphamide, Drug: Abatacept, Drug: TacrolimusParticipants to receive: * Cyclophosphamide 25 mg/kg IV over 1 hour on Day 3 and Day 4 following transplant * Abatacept 10 mg/kg IV on Day 5, Day 14, Day 28, and Day 56 following transplant * Tacrolimus 0.02 mg/kg IV by continuous infusion, starting on Day 5 following transplant. May switch to oral administration when tolerated, adjusted to maintain a drug level between 5-12ng/mL. Tacrolimus treatment is continued until Day 60 and then tapered over a period of 4 weeks in the absence of GvHD. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age ≥ 18 years
- Karnofsky score ≥ 70%
- No evidence of progressive bacterial, viral, or fungal infection
- Creatinine clearance > 50 mL/min/1.72m2
- Total bilirubin, Alanine Aminotransferase and Aspartate Aminotransferase < 2 x the upper limit of normal (except for diagnosed Gilbert's syndrome)
Key exclusion criteria (9 listed)
- Donors are excluded in case of donor-specific HLA antibodies or positive cross-match.
- Pregnant or nursing females or women of child bearing age or potential, who are unwilling to completely abstain from heterosexual sex or practice 2 effective methods of contraception from the first dose of conditioning regimen through day +180. A woman of reproductive capability is one who has not undergone a hysterectomy (removal of the womb), has not had both ovaries removed, or has not been post-menopausal (stopped menstrual periods) for more than 24 months in a row.
- Male subjects who refuse to practice effective barrier contraception during the entire study treatment period and through a minimum of 90 days after the last dose of study drug, or completely abstain from heterosexual intercourse. This must be done even if they are surgically sterilized (i.e., post-vasectomy).
- Inability to provide informed consent.
- Patient had myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure (see Appendix E), uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Prior to study entry, any ECG abnormality at screening must be documented by the investigator as not medically relevant.
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 |
|---|---|---|
| CyclophosphamideAlso: CytoxanNitrogen mustard alkylating agent produced by Bristol-Myers Squibb. | Drug | Reduced-Dose Post-Transplant Cyclophosphamide, Abatacept, and Short-Duration Tacrolimus |
| AbataceptAlso: PrografCalcineurin-inhibitor produced by Astellas. | Drug | Reduced-Dose Post-Transplant Cyclophosphamide, Abatacept, and Short-Duration Tacrolimus |
| TacrolimusAlso: OrenciaSelective T cell co-stimulation modulator produced by Bristol-Myers Squibb. | Drug | Reduced-Dose Post-Transplant Cyclophosphamide, Abatacept, and Short-Duration Tacrolimus |
Primary outcome
Cumulative Incidence of Grades II-IV Acute GvHD
Time frame: Up to Day 120
The first day of acute GvHD of any grade is used to calculate the cumulative incidence for that grade. The diagnosis of acute GvHD is based on clinical and pathological evaluation by the principal investigator in collaboration with the treating physician.
8 secondary outcomes
Cumulative Incidence of Chronic GvHD
Time frame: Up to Day 365
Number of Participants Presenting with Primary Graft Failure
Time frame: Up to Day 45
Number of Participants Presenting with Poor Graft Function
Time frame: Up to Day 30
Number of Participants Presenting with Secondary Graft Failure
Time frame: Up to Day 730
Treatment-Related Mortality (TRM) Rate
Time frame: Up to Day 730
Relapse Rate (RR)
Time frame: Up to Day 730
GvHD and Relapse-Free Survival (GRFS) Rate
Time frame: Up to Day 730
Overall Survival (OS) Rate
Time frame: Up to Day 730
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 Jan 2026.
- Individual participant dataSharing planned: No. Only available to study team members.
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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. This study is not recruiting at the moment.
Mohammad Maher Abdul Hay principal investigator
NYU Langone Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide, Abatacept and Tacrolimus in people with Graft Vs Host Disease, run by NYU Langone Health 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 10 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: under way, not looking for participants, as of the sponsor's last update on 29 Jan 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 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?
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?
Cumulative Incidence of Grades II-IV Acute GvHD (time frame: Up to Day 120). 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 Jan 2026.