Abatacept for Graft-versus-Host Disease Prevention
(ASCENT Trial)

Recruiting in Palo Alto (17 mi)

+8 other locations

Overseen byJohn Horan, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Emory University

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents receiving unrelated donor (URD) hematopoietic stem cell transplantation (HSCT), without compromising their engraftment or reconstitution of protective immunity to infection. The study will enroll 30 pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing URD HSCT. The trial will include patients with 7/8 donors and those with 8/8 (matched) donors. All participants will receive 8 doses of abatacept. Recruitment is expected to last for about 2 years and participants will be followed for up to 3 years.

Eligibility Criteria

This trial is for children and adolescents with serious non-malignant hematologic diseases who are undergoing stem cell transplantation from an unrelated donor. Participants must be aged between 0-20.99 years, depending on their condition, and have a matched or nearly matched donor. They cannot join if they have certain organ dysfunctions, HIV, uncontrolled infections, recent clinical stroke, or are pregnant.

Inclusion Criteria

I have a diagnosed artery condition in my brain shown by specific tests.

I have had a stroke confirmed by brain scans showing damage.

I have a rare blood disorder or immune system condition.

+15 more

Exclusion Criteria

My lung function is significantly reduced, below 40% of what is expected.

I do not have any ongoing serious infections.

I have not had a stroke in the last 6 months.

+11 more

Participant Groups

The study tests whether extended use of abatacept can prevent acute and chronic graft-versus-host disease in pediatric patients receiving stem cell transplants without affecting engraftment or immune reconstitution. All participants will receive eight doses of abatacept along with standard medications tacrolimus and mycophenolate mofetil.

1Treatment groups

Experimental Treatment

Group I: Participants Receiving AbataceptExperimental Treatment1 Intervention

Pediatric participants who are undergoing URD HSCT for serious NMHD will receive 8 doses of abatacept in addition to conventional GVHD prophylaxis.

Abatacept is already approved in European Union, United States, Canada, Japan for the following indications:

🇪🇺 Approved in European Union as Orencia for: