Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

Masonic Cancer Center, University of Minnesota

Summary

A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.

Eligibility

Age range: Up to 55 years
Sex: All
Healthy volunteers: Yes

Detailed criteria

Inclusion Criteria: * Sickle Cell Disease (SCD) * SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent * Transfusion Dependent Alpha- or Beta- Thalassemia * Diamond Blackfan Anemia * Other Non-Malignant Hematologic Disorders * Karnofsky ≥ 60%, Lansky play score ≥ 60. Patients with lower performance score can be considered based on study team's evaluation. * Sexually active persons of childbearing po…

Interventions

Drug
Alemtuzumab
Alemtuzumab (Campath) will be administered IV over 2 hours on day -8 to day -4.
Radiation
Total Body Irradiation
400 cGy in 2 split fractions will be administered per Department of RadiationOncology SOPs.
Biological
Cell Infusion
On day 0 the cells will be infused per cell source specific institutional guidelines
Drug
Thymoglobulin
ATG will be administered IV every 24 hours beginning on day -8 for all patients. Dosing will be model-based using Bayesian methodology13,14,15. Total doses and total number of doses (1-4 doses) will be determined based on absolute lymphocyte count and weight.
Drug
Fludarabine
Fludarabine will be administered IV over 1 hour every 24 hours on day -5 to day - 2. The daily dose of fludarabine will be determined by model-based dosing utilizing Bayesian methodology with a cumulative area under the curve (cAUC) of 20 mg\*hr/L (range 18-22 mg\*hr/L).
Drug

Location

Masonic Cancer Center
Minneapolis, Minnesota