HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT

A Pilot Trial of Abatacept Based Graft-Versus-Host Disease Prophylaxis Following Omidubicel Hematopoietic Cell Transplantation

This study is a single-center, non-randomized, single-arm pilot trial of omidubicel hematopoietic stem cell transplantation (HCT) for hematologic malignancies with myeloablative conditioning chemotherapy of physician's choice followed by abatacept/tacrolimus/mycophenolate mofetil (ABA/Tac/MMF) graft-versus-host disease (GVHD) prophylaxis. The primary objective is to assess the safety and feasibility of abatacept/tacrolimus/mycophenolate mofetil GVHD prophylaxis following omidubicel HCT.

Target enrollment is 10 participants. Subjects are adults with a diagnosis of hematologic malignancy with an available cord blood unit for omidubicel product manufacturing. Patients will be followed for a total of 18 months and will have research blood draws and Abatacept pharmacokinetics, as well as standard of care assessments that will be reviewed for this study.

It is estimated that 36 months of accrual will be necessary to enroll the targeted sample size with an accrual rate of approximately 1 participant every 3 months. Accrual will be reported by race, ethnicity, gender, and age. Descriptive analyses are planned given the sample size.

Study Overview

• Status: Recruiting
• Conditions: Hematologic Malignancy
• Intervention / Treatment: Drug: Abatacept

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Lauren Hill; Phone Number: 9196682369; Email: lauren.hill@duke.edu
• Study Contact Backup: Name: Sanghee Hong, MD; Phone Number: 9196848694; Email: sanghee.hong@duke.edu

Study Locations

• United States
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Recruiting
      • Duke University Health System
      • Contact: Jennifer Tichon; Phone Number: 919-660-7262; Email: jennifer.tichon@duke.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. A diagnosis of hematologic malignancy with an available cord blood unit for omidubicel product manufacturing
2. Adult patients (≥18 at the time of enrollment)

3. Adequate organ function for transplant defined as:

   1. Left ventricular ejection fraction ≥ 40%;
   2. DLCO, FEV1, FVC > 50% predicted;
   3. Total bilirubin ≤ 2.5 mg/dL except for patients with Gilbert's syndrome or hemolysis, and ALT, AST, and alkaline phosphatase all < 5 x upper limit of normal (ULN);
   4. Serum creatinine within normal range, or if serum creatinine outside normal range, must have measured or estimated creatinine clearance > 40 mL/min/1.73m2;
   5. Karnofsky performance score ≥ 70; and
   6. If applicable, > 6 months since a previous autologous transplant.
4. Female patients (unless postmenopausal or surgically sterilized) and male patients (even if surgically sterilized) must agree to practice two effective methods of contraception at the same time, or agree to completely abstain from heterosexual intercourse from the time of signing informed consent through 100 days post-transplant. Fertility preservation method will be left to treating physician's discretion.

Exclusion Criteria:

1. Patients with known sensitivity to dimethyl sulfoxide, dextran 40, gentamicin, human serum albumin or bovine material
2. Presence of a donor-specific antibodies with MFI >2000
3. Uncontrolled bacterial, fungal or viral infection
4. Treatment with any other investigational medical product (medications without any known FDA approved indication) needs to be discussed with the PI for patient eligibility.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Abatacept/tacrolimus/mycophenolate mofetil (ABA/Tac/MMF) following omidubicel HCT; Abatacept 10 mg/kg is given on day -1, +5, +14, and +28 in combination with standard of care tacrolimus and mycophenolate mofetil-based GVHD prophylaxis. This regimen will follow an omidubicel transplantation.	Drug: Abatacept; Abatacept is a monoclonal antibody that suppresses T-cell activation through costimulatory blockade. In 2021, abatacept was FDA approved to prevent acute GVHD following allogeneic HCT.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as evaluated by frequency of adverse events.	Adverse events are defined by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. Only events related to Abatacept (and not solely expected toxicities of omidubicel HCT) will be recorded.	6 months post-HCT
Safety of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as evaluated by severity of adverse events.	Adverse events are defined by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. Only events related to Abatacept (and not solely expected toxicities of omidubicel HCT) will be recorded.	6 months post-HCT
Feasibility of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as evaluated by number of subjects who receive minimum dose of ABA.	Minimum dose is 4 doses of minimum 10mg/kg of abatacept prophylaxis following omidubicel transplant.	Day 28 post-HCT

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by rates of acute GVHD.	Cumulative incidences of Grade II-IV and III-IV acute GVHD will be determined per Glucksberg criteria.	18 months post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by rates of chronic GVHD - mild, moderate, and severe per NIH criteria.	The cumulative incidence of chronic GVHD will be determined per NIH Consensus Conference Criteria.	18 months post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by rates of severe infections at 6 months post-transplant.	The incidence of definite and probable viral, fungal, and bacterial infections will be tabulated.	6 months post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by rates of viral reactivations at 6 months post-transplant.	The cumulative incidence of treated CMV and HHV6 reactivation in the first 6 months post-transplant will be described.	6 months post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by donor cell engraftment.	Donor cell engraftment will be assessed by donor/recipient chimerism studies. Chimerism may be evaluated in bone marrow, whole unfractionated blood, or blood cell fractions, including CD3 and CD33 or CD15 fraction.	Day 28, 60, and 90 post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by disease relapse or progression.	Relapse or progression will be diagnosed by bone marrow assessment.	Day 90 post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by non-relapse mortality (NRM).	NRM is defined as death without evidence of disease progression or recurrence.	Day 100, 180, and 365 post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by disease-free survival (DFS).	DFS is defined as the time from date of transplant to death or relapse/progression, whichever comes first.	18 months post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by overall survival (OS).	OS is defined as the time interval between date of transplant and death from any cause.	18 months post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by Severe GVHD-Free and Progression-Free Survival (SRFS).	Severe GVHD-Free and Progression-Free Survival (SRFS) as a time to event outcome is defined as the first event of Grade III-IV acute GVHD or chronic GVHD requiring systemic immune suppression, with underlying disease progression or relapse, and death by any cause.	18 months post-HCT
Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by hematologic recovery.	Hematologic recovery is defined by achieving both neutrophil and platelet count recovery after transplant. Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) greater than or equal to 500/mm3 for three consecutive measurements on three different days. Platelet recovery is defined as the first day of a sustained platelet count (1) greater than or equal to 20,000/mm3 or (2) greater than or equal to 50,000/mm3 with no platelet transfusions in the preceding seven days.	30 days post-HCT

Collaborators and Investigators

• Sponsor: Duke University
• Investigators: Principal Investigator: Sanghee Hong, MD, Duke Health

Study record dates

Study Major Dates

• Study Start (Actual): November 12, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): July 1, 2028

Study Registration Dates

• First Submitted: December 9, 2024
• First Submitted That Met QC Criteria: December 9, 2024
• First Posted (Actual): December 12, 2024

Study Record Updates

• Last Update Posted (Estimated): January 16, 2026
• Last Update Submitted That Met QC Criteria: January 14, 2026
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Hematologic Neoplasms; Immunoconjugates; Amino Acids, Peptides, and Proteins; Proteins; Antibodies; Immunoglobulins; Blood Proteins; Serum Globulins; Globulins; Abatacept
• Other Study ID Numbers: Pro00116015

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes