Actively Recruiting
Emapalumab for the Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant Recipients
Led by Children's Hospital Medical Center, Cincinnati · Updated on 2026-01-22
20
Participants Needed
1
Research Sites
8 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital Medical Center, Cincinnati
Lead Sponsor
S
Sobi, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are investigating whether blocking interferon gamma with emapalumab can prevent graft rejection in patients receiving hematopoietic stem cell transplants (HSCT). This study focuses on high-risk patients, as graft rejection is a serious complication that currently can only be treated by re-transplantation, which carries higher risks. The trial also aims to understand how emapalumab behaves in the body to help improve dosing and treatment approaches in future research. Participants are randomly assigned to receive one of two intravenous doses of emapalumab (3 mg/kg or 10 mg/kg) on the day after their transplant. If signs of acute graft rejection appear, up to two additional rescue doses of 10 mg/kg may be given, with timing determined by the lead investigator. Because emapalumab binds to circulating interferon gamma, the drug may be consumed quickly, so rescue doses can be administered as soon as 24 hours after the prior dose. During the study, participants will be closely monitored through blood tests measuring drug levels, markers like CXCL9, and immune responses up to 42 days after dosing or rescue treatment. The primary outcome is to assess the preliminary effectiveness of emapalumab over the first 100 days post-transplant. Researchers will also track survival, infections, and graft function. The total study duration extends to 100 days after HSCT to evaluate safety and outcomes.
CONDITIONS
Brief Title
Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients undergoing allogeneic hematopoietic stem cell transplant assessed for graft rejection risk
- High risk for graft rejection defined by having two or more of the following: mismatched or haploidentical donor, ex vivo t-cell depleted graft, prior history of graft rejection
You will not qualify if you...
- Known hypersensitivity to any component of the study medication
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 6 weeks or until rescue dosing decisions are made
Participants receive an intravenous dose of emapalumab on day +1 after hematopoietic stem cell transplant. Up to two additional rescue doses may be given if signs of acute graft rejection develop.
1 baseline visit and additional visits as needed for up to two rescue doses
Duration - Approximately 14 weeks
Participants are monitored for up to 100 days after transplant to assess efficacy, survival, infections, and graft status.
Weekly visits for up to 14 weeks
Trial Site Locations
Total: 1 location
1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Research Team
J
Jessica Anderson, BSN, RN, CCRC
M
Manisha Pathak, MS
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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