NCT04380740

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

A Randomized Double-Blind Trial of Abatacept Extended Dosing Versus Abatacept Short-term Dosing for Graft Versus Host Disease Prophylaxis: "ABA3"

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 160 (estimated)
• Sponsor: Boston Children's Hospital · Academic / Other
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter randomized, double blind, Phase 2 trial for patients receiving transplants from 7 of 8 HLA matched donors, in which an extended dosing regimen of abatacept, and a short-term dosing regimen + placebo, when added to standard calcineurin inhibitor + methotrexate-based prophylaxis, will be compared for their ability to improve outcomes in patients with a minimum follow-up of one year post-transplant. All patients will receive 4 doses of abatacept (Days -1, +5, +14, +28). Prior to the fifth dose, patients will be randomly assigned to the 4-dose abatacept arm and receive 4 doses of placebo or 8-dose abatacept arm and receive 4 more doses of abatacept. The primary endpoint of the study will be severe AGVHD-free, severe CGVHD-free, relapse-free survival (SGRFS). The study will end when the last patient has reached 2 years after transplant. Results will first be calculated and the study unblinded when the last patient has reached one year post-transplant.

Conditions

• Graft Vs Host Disease

Interventions

Type	Name	Description
DRUG	Placebo	During the extended dosing of abatacept, those randomized to receive 4 doses will receive a placebo consisting of an equal volume of normal saline solution.
DRUG	Abatacept	Investigational prophylaxis with extended-dosing abatacept, a calcineurin inhibitor and methotrexate.

Timeline

• Start date: 2022-03-30
• Primary completion: 2027-05-01
• Completion: 2028-06-01
• First posted: 2020-05-08
• Last updated: 2026-02-19

Locations

14 sites across 1 country: United States

Regulatory

• FDA-regulated drug study