Recruiting
Early Phase 1

Emapalumab

Sponsor:

Children's Hospital Medical Center, Cincinnati

Code:

NCT07244419

Conditions

Hematopoietic Stem Cell Transplantation

Graft Failure

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Emapalumab 3 mg/kg

Emapalumab 10 mg/kg

Study Details

Brief 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.

Conditions

Hematopoietic Stem Cell Transplantation

Graft Failure

Study ID

NCT07244419

Start date

Jan 7, 2026

Status verified date

Jan, 2026

Completion date

Aug, 2029

Anticipated

Primary completion date

Jun, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • All patients undergoing allogeneic HSCT at our institution will be evaluated for graft rejection risk factors. Patients deemed high risk for graft rejection will have 2 or more of the following: mismatched or haploidentical donor, ex vivo t-cell depleted graft, prior history of graft rejection.

Exclusion Criteria:

  • Known hypersensitivity to any constituent of the study medication.

Study Design

Enrollment

20 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: Emapalumab 3 mg/kg

Patients randomized to this arm will receive 3mg/kg of emapalumab intravenously (IV) once on day +1 after HSCT. Up to two additional, 10mg/kg rescue doses may be administered if patients developed signs and symptoms of acute graft rejection. Rescue dose administration decisions will be made in consultation with the lead study investigator. Emapalumab is a ligand-based therapy, which means high levels of circulating ligand (i.e. interferon gamma) will rapidly consume the drug. For these reasons, rescue doses may be given as early as 24 hours from the prior dose.

active comparator: Emapalumab 10 mg/kg

Patients randomized to this arm will receive 10mg/kg of emapalumab intravenously (IV) once on day +1 after HSCT. Up to two additional, 10mg/kg rescue doses may be administered if patients developed signs and symptoms of acute graft rejection. Rescue dose administration decisions will be made in consultation with the lead study investigator. Emapalumab is a ligand-based therapy, which means high levels of circulating ligand (i.e. interferon gamma) will rapidly consume the drug. For these reasons, rescue doses may be given as early as 24 hours from the prior dose.

Interventions

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.

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.

Primary outcome measure

  • Preliminary efficacy of Emapalumab [ Time Frame: 100 days ]

Central Contacts and Locations

Central contacts

Jessica Anderson, BSN, RN, CCRC

513-636-4200Jessica.Anderson@cchmc.org

Manisha Pathak, MS

513-636-4200Manisha.Pathak@cchmc.org

Locations

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Jessica Anderson, BSN, RN, CCRC

513-636-4200Jessica.Anderson@cchmc.org

Manisha Pathak, MS

513-636-4200Manisha.Pathak@cchmc.org

Principal Investigator:

Anthony Sabulski, MD

More Information

Sponsor

Children's Hospital Medical Center, Cincinnati

Last update posted

Jan 22, 2026

Last verified

Jan, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Children's Hospital Medical Center, Cincinnati on 2026-01-22.