Preventing graft rejection after allogeneic stem cell transplant
Emapalumab for the Prevention of Graft Rejection in Hematopoietic Stem Cell Transplant (HSCT) Recipients
This trial will try the drug emapalumab to see if it can prevent graft rejection in people judged high risk after allogeneic hematopoietic stem cell transplant.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Drugs / interventions | emapalumab |
| Locations | 1 site (Cincinnati, Ohio) |
| Trial ID | NCT07244419 on ClinicalTrials.gov |
What this trial studies
This early-phase, single-center trial gives emapalumab, an anti–interferon-gamma antibody, as a preventive treatment to patients at high risk for graft rejection after allogeneic HSCT. Eligible patients are identified by having two or more risk factors (mismatched or haploidentical donor, ex vivo T-cell depleted graft, or prior graft rejection) and will be assigned to receive a prophylactic dose of emapalumab (3 mg/kg or 10 mg/kg). The protocol collects safety, pharmacokinetic/pharmacodynamic (PK/PD), and in vitro mechanistic data to inform dosing and to look for early signs that interferon-gamma blockade reduces graft failure. All treatments and follow-up occur at Cincinnati Children's Hospital Medical Center in partnership with Sobi.
Who should consider this trial
Good fit: People undergoing allogeneic HSCT who are judged high risk for graft rejection by having two or more predefined risk factors (mismatched or haploidentical donor, ex vivo T-cell depleted graft, or prior graft rejection).
Not a fit: Patients who are not high risk for graft rejection, those with known hypersensitivity to the drug, or those with contraindications to additional immunomodulation are unlikely to benefit from this prophylactic approach.
Why it matters
Potential benefit: If successful, prophylactic emapalumab could reduce or prevent graft rejection and spare patients the risks of re-transplantation.
How similar studies have performed: Emapalumab is approved for interferon-gamma–driven hemophagocytic lymphohistiocytosis and shows target engagement, but using it prophylactically to prevent HSCT graft rejection is largely novel with limited prior clinical data.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Cincinnati, Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
Study contacts
- Principal investigator: Anthony Sabulski, MD — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Jessica Anderson, BSN, RN, CCRC
- Email: Jessica.Anderson@cchmc.org
- Phone: 513-636-4200
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.