Trial Outcomes & Findings for Cyclophosphamide, Abatacept, and Tacrolimus for GvHD Prevention (NCT NCT04503616)

Last Updated: 2024-10-16

Results Overview

The first day of grade II-IV acute GvHD will be used to calculate the cumulative incidence. The cumulative incidence will be defined as the percentage of participants with grade II-IV acute GvHD. The diagnosis of acute GvHD is based on clinical and pathological evaluation by the principal investigator in collaboration with the treating physician. Overall Grades of Acute GvHD: 0 = none; 1. = mild; 2. = moderate; 3. = severe; 4. = life threatening

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

46 participants

Primary outcome timeframe

120 days

Results posted on

2024-10-16

Participant Flow

Participant milestones

Participant milestones
Measure	HSCT Patients Adult patients with hematological malignancies undergoing HLA-haploidentical HSCT from first-or second-degree family donors. Cyclophosphamide, Abatacept, and Tacrolimus for GvHD Prevention: Cyclophosphamide 50 mg/kg IV over 2 hours on Day +3 and +4 Abatacept 10 mg/kg IV on days +5, +14, and +28 Tacrolimus 0.02 mg/kg IV by continuous infusion, starting on day +5. May switch to oral when tolerated, adjusted to maintain a drug level between 5-12ng/mL. Treatment is discontinued on day +60 after a 4 week-taper
Overall Study STARTED	46
Overall Study COMPLETED	46
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cyclophosphamide, Abatacept, and Tacrolimus for GvHD Prevention

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	HSCT Patients n=46 Participants Adult patients with hematological malignancies undergoing HLA-haploidentical HSCT from first-or second-degree family donors. Cyclophosphamide, Abatacept, and Tacrolimus for GvHD Prevention: Cyclophosphamide 50 mg/kg IV over 2 hours on Day +3 and +4 Abatacept 10 mg/kg IV on days +5, +14, and +28 Tacrolimus 0.02 mg/kg IV by continuous infusion, starting on day +5. May switch to oral when tolerated, adjusted to maintain a drug level between 5-12ng/mL. Treatment is discontinued on day +60 after a 4 week-taper
Age, Continuous	60 years n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants
Sex: Female, Male Male	25 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	34 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants
Race (NIH/OMB) White	39 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	46 participants n=5 Participants

PRIMARY outcome

Timeframe: 120 days

Outcome measures

Outcome measures
Measure	HSCT Patients n=46 Participants Adult patients with hematological malignancies undergoing HLA-haploidentical HSCT from first-or second-degree family donors. Cyclophosphamide, Abatacept, and Tacrolimus for GvHD Prevention: Cyclophosphamide 50 mg/kg IV over 2 hours on Day +3 and +4 Abatacept 10 mg/kg IV on days +5, +14, and +28 Tacrolimus 0.02 mg/kg IV by continuous infusion, starting on day +5. May switch to oral when tolerated, adjusted to maintain a drug level between 5-12ng/mL. Treatment is discontinued on day +60 after a 4 week-taper
Percentage of Participants With Grade II-IV Acute GvHD by Day +120	17.4 Percentage of participants Interval 9.2 to 32.9

SECONDARY outcome

Timeframe: Day 365

The first day of chronic GvHD will be used to calculate the cumulative incidence of chronic GvHD. This endpoint will be evaluated through day +365 post-transplant.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 45

Graft failure is defined as failure to achieve neutrophil engraftment by day +28 or lack of donor chimerism \> 50% by day 45, not due to the underlying malignancy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 28

Poor Graft Function defined by at least 2 of the following 3 criteria: Hemoglobin \< 8 g/dL, ANC \< 0.5 x 109/L, and platelets \< 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 730

Evaluated following engraftment through day +730. Secondary Graft Failure defined as poor graft function associated with donor chimerism \< 5%.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 730

Number of participant deaths not attributable to disease relapse or progression. Will be evaluated to day +730.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 730

Evaluated to day +730 and defined as the percentage of patients in whom the disease for which transplant is performed becomes evident by methods of disease detection after the transplant.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 730

Evaluated to day +730 and defined as the percentage of participants who are without reported grade III-IV acute GvHD, without chronic GvHD requiring systemic therapy, and have not experienced relapse or death.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 730

Evaluated to day +730 and defined as percentage of participants alive at the end of the study's evaluation period.

Outcome measures

Outcome data not reported

Adverse Events

HSCT Patients

Serious events: 19 serious events

Other events: 24 other events

Deaths: 7 deaths

Serious adverse events

Serious adverse events
Measure	HSCT Patients n=46 participants at risk Adult patients with hematological malignancies undergoing HLA-haploidentical HSCT from first-or second-degree family donors. Cyclophosphamide, Abatacept, and Tacrolimus for GvHD Prevention: Cyclophosphamide 50 mg/kg IV over 2 hours on Day +3 and +4 Abatacept 10 mg/kg IV on days +5, +14, and +28 Tacrolimus 0.02 mg/kg IV by continuous infusion, starting on day +5. May switch to oral when tolerated, adjusted to maintain a drug level between 5-12ng/mL. Treatment is discontinued on day +60 after a 4 week-taper
Renal and urinary disorders Acute kidney injury	10.9% 5/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Bacteremia	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Hepatobiliary disorders Blood bilirubin increased	6.5% 3/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Cytomegalovirus (CMV) reactivation	6.5% 3/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Colitis	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Diarrhea	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Epstein-Barr Virus (EBV) reactivation	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
General disorders Febrile neutropenia	13.0% 6/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
General disorders Graft-versus-host disease (GvHD)	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Hematochezia with colostomy	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Metabolism and nutrition disorders Hyperglycemia	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Hypoxia	6.5% 3/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Meningitis	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
General disorders Multiorgan failture	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Cardiac disorders Pericardial effusion	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Cardiac disorders Pericardial tamponade	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Pulmonary airspace opacities	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Pulmonary infiltrates	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Respiratory failure	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Hepatobiliary disorders Sinusoidal obstruction syndrome	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Stridor	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Blood and lymphatic system disorders Thrombotic microangiopathy	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Toxoplasmosis infection	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Urinary tract infection	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Wheezing	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.

Other adverse events

Other adverse events
Measure	HSCT Patients n=46 participants at risk Adult patients with hematological malignancies undergoing HLA-haploidentical HSCT from first-or second-degree family donors. Cyclophosphamide, Abatacept, and Tacrolimus for GvHD Prevention: Cyclophosphamide 50 mg/kg IV over 2 hours on Day +3 and +4 Abatacept 10 mg/kg IV on days +5, +14, and +28 Tacrolimus 0.02 mg/kg IV by continuous infusion, starting on day +5. May switch to oral when tolerated, adjusted to maintain a drug level between 5-12ng/mL. Treatment is discontinued on day +60 after a 4 week-taper
Gastrointestinal disorders Abdominal pain	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Anorexia	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Bacteremia	8.7% 4/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Hepatobiliary disorders Blood bilirubin increased	6.5% 3/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations CMV reactivation	15.2% 7/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Colitis	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Nervous system disorders Confusion	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Eye disorders Conjunctival hemorrhage	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Cough	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Renal and urinary disorders Creatinine increase	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
General disorders Cytokine release syndrome	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Nervous system disorders Delirium	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Diarrhea	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Dyspnea	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Renal and urinary disorders Dysuria	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations EBV reactivation	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Blood and lymphatic system disorders Epitaxis	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Nervous system disorders Facial nerve disorder	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
General disorders Fatigue	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
General disorders Fever	8.7% 4/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
General disorders Flu like symptoms	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Nervous system disorders Headache	8.7% 4/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Renal and urinary disorders Hematuria	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Vascular disorders Hypertension	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Metabolism and nutrition disorders Hyponatremia	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Respiratory, thoracic and mediastinal disorders Hypoxia	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Injury, poisoning and procedural complications Infusion related reaction	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Nervous system disorders Lethargy	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Mucositis	13.0% 6/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Nausea	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Skin and subcutaneous tissue disorders Papulopustular rash	4.3% 2/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Pneumonia	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Gastrointestinal disorders Rectal ulcer	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Nervous system disorders Tremor	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.
Infections and infestations Viremia	2.2% 1/46 • 120 Days Collected during study visits and interviews of a study participant presenting for medical care, or upon review by a study monitor.

Additional Information

Kelli Cole, NP

NYU Langone Health

Phone: 646-501-4848

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place