Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant
Status:
Recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
The ASCENT Trial is a single arm, multi-center, phase II study. The primary objective is to
determine the rejection-free, severe graft-versus-host disease (GVHD)-free survival in
pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing
unrelated donor (URD) hematopoietic stem cell transplantation (HSCT) with abatacept added to
conventional GVHD prophylaxis. The secondary objective is to characterize the impact of
abatacept on infection and the reconstitution of protective immunity to infection.
Transplanted patients will be followed for 3 years. Weight-based peripheral blood samples
will be drawn longitudinally through two years to evaluate immune reconstitution.
The study will enroll 28 pediatric patients with serious NMHD undergoing URD HSCT. The trial
will include two strata, based on donor matching. Stratum 1 (n=14) will be for patients with
7/8 donors and stratum 2 (n=14) will be for those with 8/8 (matched) donors. All participants
will receive 8 doses of abatacept (10 mg/kg intravenously on days -1, +5, +14, +28, +56, +84,
+112, and +150). Recruitment is expected to last for about 2 years and participants will be
followed for up to 3 years.
This trial will test the hypothesis that extended abatacept administration (combined with a
standard regimen of tacrolimus and mycophenolate mofetil) will effectively prevent acute and
chronic GVHD in children and adolescents receiving URD HSCT, without compromising their
engraftment or reconstitution of protective immunity to infection.
Phase:
Phase 2
Details
Lead Sponsor:
Emory University
Collaborators:
Aflac Cancer and Blood Disorders Center Bristol-Myers Squibb Sickle Cell Transplant Advocacy & Research Alliance (STAR) Thrasher Research Fund