NCT07244419

Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients

Emapalumab for the Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The investigators hypothesize that graft rejection after hematopoietic stem cell transplant (HSCT) is primarily driven by interferon gamma, and prophylactic interferon gamma inhibition in high-risk patients will prevent graft rejection. Additionally, knowledge of emapalumab PK/PD and in vitro mechanistic effects of emapalumab in this novel setting will guide optimization of dosing regimens and treatment approaches in future studies.

Detailed description

Graft rejection is a devastating and understudied complication of hematopoietic stem cell transplant (HSCT) due to the lack of available interventions outside of re-transplantation. Re-transplantation is challenging and is associated with increased morbidity and mortality. The purpose of this study is to learn more about emapalumab and its ability to prevent graft rejection in hematopoietic stem cell transplant (HSCT) recipients. Specifically, the study doctors would like to learn more about the efficacy and treatment of emapalumab as a prophylactic intervention for graft rejection.

Conditions

• Hematopoietic Stem Cell Transplantation
• Graft Failure

Interventions

Type	Name	Description
DRUG	Emapalumab 3 mg/kg	Subjects will be randomized to either receive a 3mg/kg or 10mg/kg intravenous dose of emapalumab once and may receive up to two additional doses if clinical concern for impending graft rejection develops.
DRUG	Emapalumab 10 mg/kg	Subjects will be randomized to either receive a 3mg/kg or 10mg/kg intravenous dose of emapalumab once and may receive up to two additional doses if clinical concern for impending graft rejection develops.

Timeline

• Start date: 2026-01-07
• Primary completion: 2029-06-01
• Completion: 2029-08-01
• First posted: 2025-11-24
• Last updated: 2026-01-22

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study