Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Emapalumab 3 mg/kg, Emapalumab 10 mg/kg.
- Who it may be relevant to
- Registry conditions: Hematopoietic Stem Cell Transplantation, Graft Failure. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Emapalumab for the Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients
Overview
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.
Interventions
- 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.
Primary outcome measures
- Preliminary efficacy of Emapalumab [Time frame: 100 days]
Secondary outcome measures (12)
- 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]
- Maximum plasma concentration of emapalumab after 3 mg/kg prophylactic dosing [Time frame: Until day 42 or time of rescue dose, whichever is sooner]
- Maximum plasma concentration of emapalumab after rescue dosing [Time frame: 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). [Time frame: 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). [Time frame: 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 2.6x the upper limit of normal for the test. [Time frame: 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 2.6x the upper limit of normal for the test. [Time frame: Until day 42 or 1 week after rescue dose, whichever is later]
- Emapalumab half-life after 10 mg/kg prophylactic dosing [Time frame: Until day 42 or time of rescue dose, whichever is sooner]
- Emapalumab half-life after 3 mg/kg prophylactic dosing [Time frame: Until day 42 or time of rescue dose, whichever is sooner]
- Emapalumab half-life after 10mg/kg rescue dosing [Time frame: Until day 42 or time of rescue dose, whichever is sooner]
- Overall survival [Time frame: 100 days after HSCT.]
- Number of patients who develop infections [Time frame: 100 days after HSCT.]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Cincinnati Children's Hospital Medical Center — Cincinnati
Identifiers
NCT: NCT07244419 · 2024-0167