Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorChildren's Hospital Medical Center, Cincinnati
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
Known hypersensitivity to any constituent of the study medication.
Trial design
Design model
Parallel
Treatments tested in this trial
Emapalumab 3 mg/kg
DrugSubjects 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
DrugSubjects 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.
Treatment groups
20 Participants
are divided into 2 treatment groupsGroup A: Emapalumab 3 mg/kgActive comparator 1 intervention
Group B: Emapalumab 10 mg/kgActive comparator 1 intervention
Trial outcomes
Primary outcomes
1
Preliminary efficacy of Emapalumab
Measured by the incidence of graft rejection in the treatment cohort.
Time frame
100 daysSecondary outcomes
1
Maximum plasma concentration (Cmax) of emapalumab after 10 mg/kg prophylactic dosing
* 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.
Time frame
Until day 42 or time of rescue dose, whichever is sooner2
Maximum plasma concentration of emapalumab after 3 mg/kg prophylactic dosing
* 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.
Time frame
Until day 42 or time of rescue dose, whichever is sooner3
Maximum plasma concentration of emapalumab after rescue dosing
* 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.
Time frame
Until day 42 or 1 week after rescue dose, whichever is later4
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).
* 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.
Time frame
Until day 42 or 1 week after rescue dose, whichever is laterSponsors and contacts
Click on the lead sponsor to view all of their trials.
Children's Hospital Medical Center, Cincinnati
Lead sponsor
Sobi, Inc.
Collaborator
Source ClinicalTrials.gov