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NCT Number: NCT07244419

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

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.

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Key information

Conditions

Hematopoietic Stem Cell Transplantation Graft Failure

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location status: Recruiting

Location contact

Anthony Sabulski, MD

PRINCIPAL_INVESTIGATOR

Jessica Anderson, BSN, RN, CCRC

CONTACT

[email protected]

513-636-4200

Manisha Pathak, MS

CONTACT

[email protected]

513-636-4200

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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 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.

Primary outcomes

  1. Preliminary efficacy of Emapalumab

    Time frame: 100 days

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

Secondary outcomes

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

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

    • Measured by the Cmax (ng/mL) of emapalumab after 10mg/kg dose
    • Cmax values will be calculated using every 48 hour blood collections beginning at the time of emapalumab administration (day 1 after HSCT) until day 21.

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

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

    • Measured by the Cmax (ng/mL) of emapalumab after 3 mg/kg dose
    • Cmax values will be calculated using every 48 hour blood collections beginning at the time of emapalumab administration (day 1 after HSCT) until day 21.

  3. Maximum plasma concentration of emapalumab after rescue dosing

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

    • Measured by the Cmax (ng/mL) of emapalumab after 10mg/kg rescue dose(s).
    • Cmax values will be calculated using every 48 hour blood collections beginning at the time of emapalumab rescue dose administration and continuing for 1 week after the rescue dose.

  4. 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).

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

    • Measured by plasma CXCL9 levels (pg/mL)
    • CXCL9 levels will be measured every 48 hours beginning at the time of prophylactic emapalumab administration (day 1 after HSCT) and until day 21 after HSCT. Additionally, weekly CXCL9 levels will be obtained from day 21 until day 42 after HSCT.

  5. 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).

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

    • Measured by plasma CXCL9 levels (pg/mL)
    • CXCL9 levels will be measured every 48 hours beginning at the time of prophylactic emapalumab administration (day 1 after HSCT) and until day 21 after HSCT. Additionally, weekly CXCL9 levels will be obtained from day 21 until day 42 after HSCT.

  6. Number of patients in 10 mg/kg prophylactic dosing arm who maintain CXCL9 levels below 2.6x the upper limit of normal for the test.

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

    • Measured by plasma CXCL9 levels (pg/mL). This cutoff was chosen based on our prior publication which showed CXCL9 levels above this threshold were associated with graft rejection.
    • CXCL9 levels will be measured every 48 hours beginning at the time of prophylactic emapalumab administration (day 1 after HSCT) and until day 21 after HSCT. Additionally, weekly CXCL9 levels will be obtained from day 21 until day 42 after HSCT.

  7. Number of patients in 3 mg/kg prophylactic dosing arm who maintain CXCL9 levels below 2.6x the upper limit of normal for the test.

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

    • Measured by plasma CXCL9 levels (pg/mL). This cutoff was chosen based on our prior publication which showed CXCL9 levels above this threshold were associated with graft rejection.
    • CXCL9 levels will be measured every 48 hours beginning at the time of prophylactic emapalumab administration (day 1 after HSCT) and until day 21 after HSCT. Additionally, weekly CXCL9 levels will be obtained from day 21 until day 42 after HSCT.

  8. Emapalumab half-life after 10 mg/kg prophylactic dosing

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

    • Measured using the volume of distribution (L) and clearance (L/h) of emapalumab after 10mg/kg prophylactic dose
    • Half-life will be calculated using every 48 hour sample collections beginning at the time of emapalumab administration (day 1 after HSCT) until day 21. Additionally, weekly blood samples will be obtained from day 21 until day 42 after HSCT.

  9. Emapalumab half-life after 3 mg/kg prophylactic dosing

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

    • Measured using the volume of distribution (L) and clearance (L/h) of emapalumab after 3mg/kg prophylactic dose
    • Half-life will be calculated using every 48 hour sample collections beginning at the time of emapalumab administration (day 1 after HSCT) until day 21. Additionally, weekly blood samples will be obtained from day 21 until day 42 after HSCT.

  10. Emapalumab half-life after 10mg/kg rescue dosing

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

    • Measured using the volume of distribution (L) and clearance (L/h) of emapalumab after 10mg/kg dose(s)
    • Half-life will be calculated using every 48 hour blood collections beginning at the time of emapalumab rescue dose administration and continuing for 1 week after the rescue dose.

  11. Overall survival

    Time frame: 100 days after HSCT.

    • Measured by overall survival of patients who receive prophylactic emapalumab.

  12. Number of patients who develop infections

    Time frame: 100 days after HSCT.

    Measured by the incidence of infection in patients who receive prophylactic emapalumab.

  13. Number of patients who develop mixed chimerism.

    Time frame: 100 days after HSCT.

    Measured by the incidence of mixed chimerism in patients who receive prophylactic emapalumab.

Study contacts

Contact information is provided by the study sponsor or research team.

Jessica Anderson, BSN, RN, CCRC

CONTACT

[email protected]

513-636-4200

Manisha Pathak, MS

CONTACT

[email protected]

513-636-4200

Sponsors and collaborators

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Other

Collaborators

  • Sobi, Inc.

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2029
Study completion
2029
First posted
Nov 24, 2025
Registry last updated
Jan 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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