Adjusting Alemtuzumab Dosing for Bone Marrow Transplantation
Official title: Precision Alemtuzumab Dosing for Allogeneic Hematopoietic Cell Transplantation in Non-Malignant Diseases
Run by Children's Hospital Medical Center, Cincinnati · NCT05501756 · Data updated 2026-11-12
This study is testing if adjusting the dose of alemtuzumab can help kids and young adults getting a bone marrow transplant have fewer complications and recover better.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Sex | All |
| Enrollment | 60 (estimated) |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Drugs / interventions | alemtuzumab |
| Locations | 1 site (Cincinnati, Ohio) |
| Status | Recruiting |
| Started | January 11, 2023 |
| Main results expected | August 31, 2028 |
| Study ends | August 31, 2028 |
| Registry record updated | June 12, 2026 |
| Trial ID | NCT05501756 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are patients aged 6 weeks to 30 years undergoing allogeneic HCT for non-malignant diseases with an alemtuzumab-containing regimen.
✕Probably not a fit
Patients with a history of anaphylaxis to alemtuzumab or those who have not cleared alemtuzumab prior to the preparative regimen may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study investigates the optimal dosing of alemtuzumab, an antibody used to suppress the immune system, in patients undergoing allogeneic hematopoietic cell transplantation (HCT). The goal is to adjust the alemtuzumab dose to achieve a target concentration of 0.15-0.9 ug/mL on the day of graft infusion, which may reduce the risk of complications such as acute graft versus host disease (GVHD) and mixed chimerism. By monitoring and adjusting the dosage, the study aims to improve immune recovery and overall clinical outcomes for patients. The research will evaluate the effectiveness of this tailored dosing approach in a pediatric and young adult population.
Why it matters
Potential benefit: If successful, this approach could lead to improved outcomes and reduced complications for patients undergoing bone marrow transplantation.
How similar studies have gone: While the specific dosing adjustment approach may be novel, similar studies have shown that tailored immunosuppressive regimens can improve outcomes in HCT.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients who are undergoing allogeneic HCT at CCHMC with an alemtuzumab-containing preparative regimen for treatment of a non-malignant disease are eligible. * For the first 7 patients, patients must have a 10/10 HLA matched related or unrelated stem cell donor, or be receiving a CD34+ selected stem cell product. After the first 7 patients, any donor match may be allowed after data review by the BMT clinicians and the PI. Exclusion Criteria: * Patients with a history of anaphylaxis to alemtuzumab. * Patients who have previously received alemtuzumab and have not cleared alemtuzumab prior to the start of the preparative regimen. * Life expectancy less than 4 weeks. * Patients receiving dialysis or plasmapheresis at the time of the start of the conditioning regimen. * Failure to sign informed consent and/or assent, or inability to undergo informed consent process. * It is not medically advisable to obtain the specimens necessary for this study. * Not able to tolerate subcutaneous dosing (patients with severe skin conditions). * Patients with cancer. * Patients whose clinical condition suggest there may be inability to successfully perform the PK modeling such as, but not limited to, active flaring of hemophagocytic lymphohistiocytosis in which excessive lymphoproliferation may significantly alter the target-mediated clearance of alemtuzumab and prevent observation of non-target mediated clearance which is needed for robust modeling. * Patients whose pre-alemtuzumab level reveals an interfering substance which prevents accurate measurement of alemtuzumab.
Where it's running
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States Recruiting
Study contacts
- Principal investigator: Parinda Mehta, MD — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Caitlin Cottrell
- Email: Caitlin.Cottrell@cchmc.org
- Phone: 513-803-7039
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Partial stem cell transplant for sickle cell disease and beta-thalassemia using matched donors
This study tests a partial stem cell transplant from matched donors to help people with severe sickle cell disease or beta-thalassemia see if more donor cells can persist long-term.
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Transplantation for Chronic Granulomatous Disease using a new drug regimen
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.