Hematopoietic Stem Cell TransplantationEarly Phase 1NCT07244419
Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients
A Early Phase 1 study testing Emapalumab 3 mg/kg and Emapalumab 10 mg/kg in people with Hematopoietic Stem Cell Transplantation and Graft Failure, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hematopoietic Stem Cell Transplantation, Graft Failure
- What is being tested?
- Emapalumab 3 mg/kg and Emapalumab 10 mg/kg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Jan 2026to Aug 2029
- Phase
- Early Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Known hypersensitivity to any constituent of the study medication.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Jessica Anderson, BSN, RN, CCRC, 513-636-4200, Jessica.Anderson@cchmc.org
Manisha Pathak, MS, 513-636-4200, Manisha.Pathak@cchmc.org
About this study
Official title: Emapalumab for the 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.
Read the 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.
- Being tested
- Emapalumab 3 mg/kg, Emapalumab 10 mg/kg
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Emapalumab 3 mg/kgDrug
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/kgDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Emapalumab 10 mg/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Preliminary efficacy of Emapalumab
Measured by the incidence of graft rejection in the treatment cohort.
Measured: 100 days
Secondary outcomes (13)
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.
Measured: Until day 42 or time of rescue dose, whichever is sooner
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.
Measured: Until day 42 or time of rescue dose, whichever is sooner
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.
Measured: 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).
- 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.
Measured: 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).
- 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.
Measured: 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.
- 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.
Measured: 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.
- 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.
Measured: Until day 42 or 1 week after rescue dose, whichever is later
Emapalumab half-life after 10 mg/kg prophylactic dosing
- 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.
Measured: Until day 42 or time of rescue dose, whichever is sooner
Emapalumab half-life after 3 mg/kg prophylactic dosing
- 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.
Measured: Until day 42 or time of rescue dose, whichever is sooner
Emapalumab half-life after 10mg/kg rescue dosing
- 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.
Measured: Until day 42 or time of rescue dose, whichever is sooner
Overall survival
- Measured by overall survival of patients who receive prophylactic emapalumab.
Measured: 100 days after HSCT.
Number of patients who develop infections
Measured by the incidence of infection in patients who receive prophylactic emapalumab.
Measured: 100 days after HSCT.
Number of patients who develop mixed chimerism.
Measured by the incidence of mixed chimerism in patients who receive prophylactic emapalumab.
Measured: 100 days after HSCT.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2025
- Study start7 Jan 2026
- Primary completionJun 2029 (estimated)
- Study completionAug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Stem Cell TransplantationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Sobi, Inc.
- Responsible party
- Sponsor
- Investigators
- Anthony Sabulski, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07244419
- Sponsor's protocol ID
- 2024-0167
- Status last verified
- Jan 2026
- First submitted
- 30 Oct 2024
- Last updated
- 22 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No