T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age5-40
SponsorPaul Szabolcs
About this trial
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.
Eligibility criteria
Qualifiers
Patient, parent, or legal guardian must have given written informed consent and/or assent according to FDA guidelines.
Ages 5 years to 40 years, at time of consent.
Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring hospitalization for parenteral pain management in the previous 2 years.
Recurrent acute chest syndrome (ACS) despite supportive care, minimum of 2 episodes in preceding 2-year period.
Disqualifiers
Patients with alternate, superior donor options (matched sibling donor or matched unrelated donor).
Patients who have undergone stem cell transplantation in the 6 months prior to anticipated conditioning.
Patients with history of a central nervous system (CNS) event within six months prior to start of conditioning (patient will be delayed until eligible).
Patients who are pregnant or lactating
Trial design
Treatments tested in this trial
- CD3/CD19 depleted leukocytes
- CD45RA depleted leukocytes
- Hydroxyurea
- Rituximab
- Alemtuzumab
- Fludarabine
- Thiotepa
Treatment groups
5 Participants
are divided into 1 treatment groupSponsors and collaborators
Paul Szabolcs
Lead sponsor
University of Pittsburgh
Sponsor institution
Source ClinicalTrials.gov