Phase 2 Abatacept Dosing for Graft Versus Host Disease Prophylaxis

This study is testing two different dosing schedules of abatacept to prevent Graft Versus Host Disease (GVHD) in people receiving a stem cell transplant. GVHD is a serious complication where the donor's immune cells attack the patient's body. You might be able to join if you are at least 2 years old, weigh 10 kg, and are receiving a transplant from a specific type of unrelated donor. All participants will receive abatacept for the first four doses. Then, you will be randomly assigned to either continue with four more doses of abatacept or receive a placebo (an inactive substance) for those four doses. The main goal is to see if one dosing schedule is better at preventing severe GVHD and relapse (the return of the original disease) for at least two years after transplant. The study aims to enroll 160 participants.

Study design
This is a Phase 2, multicenter, randomized, double-blind study. It plans to enroll 160 participants.
What's involved
All patients will receive 4 doses of abatacept. Then, you will be randomly assigned to receive either 4 more doses of abatacept or 4 doses of placebo.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for at least one year post-transplant, with the primary outcome measured at two years.

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NCT04380740

Extended vs Short-term Abatacept Dosing for Graft Versus Host Disease Prophylaxis

Recruiting
PHASE2Ages 2+InterventionalTreatment
Boston Children's Hospital
~160 participants
Updated 2026-05-11 on ClinicalTrials.gov
What's tested:PlaceboAbatacept

At a glance

Recruiting sites
4 of 15 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Severe AGVHD-free, severe CGVHD-free, relapse-free survival (SGRFS)
Measured over 2 years
Graft Vs Host Disease

NCT04380740

Where you'd take part

This study runs at 15 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Boston Children's Hospital

    Boston, Massachusettsstudy coordinator listed

    Recruiting

  • Children's Healthcare of Atlanta

    Atlanta, Georgiastudy coordinator listed

    Recruiting

  • University of Rochester

    Rochester, New Yorkstudy coordinator listed

    Recruiting

  • University of Chicago

    Chicago, Illinoisno site contact published

    Recruiting

  • Beth Israel Deaconess Medical Center

    Boston, Massachusettsno site contact published

    Active, not recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvaniano site contact published

    Withdrawn

  • Cincinnati Children's Hospital

    Cincinnati, Ohiono site contact published

    Active, not recruiting

  • City Of Hope National Medical Center

    Duarte, Californiano site contact published

    Active, not recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

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Eligibility criteria

Exclusion

If the patient does not meet defined eligibility requirements, the PI/study committee must be contacted to determine eligibility.
  • Severe AGVHD-free, severe CGVHD-free, relapse-free survival (SGRFS)2 years

    SGRFS will be modeled as a time-to-event outcome, and as such, failures that occur beyond one year and before study end will be considered in the analysis.