HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT

NCT06731504 | Recruiting Early Phase 1 FDA Drug

• 1 other identifier: Pro00116015
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hematologic Malignancy

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (11)

• Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by Severe GVHD-Free and Progression-Free Survival (SRFS).

  18 months post-HCT

• Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by rates of acute GVHD.

  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.

  18 months post-HCT

• Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by hematologic recovery.

  30 days post-HCT

• Efficacy of ABA/Tac/MMF GVHD prophylaxis following omidubicel HCT as measured by rates of severe infections at 6 months post-transplant.

  6 months post-HCT

Study Arms (1)

Abatacept/tacrolimus/mycophenolate mofetil (ABA/Tac/MMF) following omidubicel HCT

EXPERIMENTAL

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

Interventions

Abatacept DRUG

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.

Abatacept/tacrolimus/mycophenolate mofetil (ABA/Tac/MMF) following omidubicel HCT

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A diagnosis of hematologic malignancy with an available cord blood unit for omidubicel product manufacturing
• Adult patients (≥18 at the time of enrollment)
• Adequate organ function for transplant defined as:
• Left ventricular ejection fraction ≥ 40%;
• DLCO, FEV1, FVC \> 50% predicted;
• 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);
• Serum creatinine within normal range, or if serum creatinine outside normal range, must have measured or estimated creatinine clearance \> 40 mL/min/1.73m2;
• Karnofsky performance score ≥ 70; and
• If applicable, \> 6 months since a previous autologous transplant.
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Duke University: lead

Study Sites (1)

Duke University Health System

Durham, North Carolina, 27705, United States

RECRUITING

MeSH Terms

Conditions

Hematologic Neoplasms

Interventions

Abatacept

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Immunoconjugates; Antibodies; Immunoglobulins; Serum Globulins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Globulins

Study Officials

• Sanghee Hong, MD

  Duke Health

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sanghee Hong, MD

CONTACT

9196848694; sanghee.hong@duke.edu

Lauren Hill

CONTACT

9196682369; lauren.hill@duke.edu

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 9, 2024
• First Posted: December 12, 2024
• Study Start: November 12, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): July 1, 2028
• Last Updated: January 16, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)