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

NCT07244419 | Recruiting Early Phase 1 DMC FDA Drug

• 1 other identifier: 2024-0167
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• Preliminary efficacy of Emapalumab

  Measured by the incidence of graft rejection in the treatment cohort.

  100 days

Secondary Outcomes (13)

• Maximum plasma concentration (Cmax) of emapalumab after 10 mg/kg prophylactic dosing

  Until day 42 or time of rescue dose, whichever is sooner

• Maximum plasma concentration of emapalumab after 3 mg/kg prophylactic dosing

  Until day 42 or time of rescue dose, whichever is sooner

• Maximum plasma concentration of emapalumab after rescue dosing

  Until day 42 or 1 week after rescue dose, whichever is later

• Number of patients in 10 mg/kg prophylactic dosing arm who maintain CXCL9 levels below the upper limit of normal for the test (</= 647 pg/mL).

  Until day 42 or 1 week after rescue dose, whichever is later

• Number of patients in 3 mg/kg prophylactic dosing arm who maintain CXCL9 levels below the upper limit of normal for the test (</= 647 pg/mL).

  Until day 42 or 1 week after rescue dose, whichever is later

Study Arms (2)

Emapalumab 3 mg/kg

ACTIVE COMPARATOR

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.

Drug: Emapalumab 3 mg/kg

Emapalumab 10 mg/kg

ACTIVE COMPARATOR

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.

Drug: Emapalumab 10 mg/kg

Interventions

Emapalumab 10 mg/kg DRUG

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

Emapalumab 3 mg/kg DRUG

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 3 mg/kg

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Known hypersensitivity to any constituent of the study medication.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Sobi, Inc.: collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

MeSH Terms

Interventions

Emapalumab

Study Officials

• Anthony Sabulski, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jessica Anderson, BSN, RN, CCRC

CONTACT

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

Manisha Pathak, MS

CONTACT

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

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 30, 2024
• First Posted: November 24, 2025
• Study Start: January 7, 2026
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): August 1, 2029
• Last Updated: January 22, 2026

Record last verified: 2026-01

Locations

US (1)