Precision Dosing of Alemtuzumab

• Status: Completed
• Trial end date: 2019-03-10
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to conduct a pilot trial of a Precision Dosing approach to alemtuzumab dosing for allogeneic hematopoietic cell transplantation of patients with non-malignant diseases. The investigators will measure the ability to use a population PK model of alemtuzumab to target patient Day 0 alemtuzumab levels to 0.15-0.6ug/mL in a pilot study of 20 patients.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Treatments: Alemtuzumab

Criteria

Inclusion Criteria:

1. Patients who are undergoing RIC HCT with alemtuzumab, fludarabine, and melphalan at CCHMC for treatment of a non-malignant disease.

2. Age ≥ 6 weeks to ≤ 30 years (at time of enrollment).

3. 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 PI and medical monitor.

Exclusion Criteria:

1. Patients with a history of anaphylaxis to alemtuzumab.

2. Patients who have previously received alemtuzumab and have not cleared alemtuzumab prior to the start of the preparative regimen.

3. Life expectancy less than 2 weeks.

4. Patients receiving dialysis.

5. Failure to sign informed consent and/or assent, or inability to undergo informed consent process.

6. It is not medically advisable to obtain the specimens necessary for this study.

7. Not able to tolerate subcutaneous dosing (patients with severe skin conditions such as epidermolysis bullosa).