CD34+ Stem Cell Selection for Bone Marrow Failure Syndromes

Palo Alto (17 mi)

Overseen byDiane George, MD

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Diane George

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial uses a special machine to filter out harmful cells from donor blood to make stem cell transplants safer for young patients with non-cancerous diseases. By removing specific cells, it aims to prevent a serious immune reaction.

Eligibility Criteria

This trial is for children, adolescents, and young adults with various non-malignant diseases like sickle cell anemia or immune deficiencies. Participants must be under 40 years old, have a matched family or unrelated adult donor for stem cells, and good organ function. Pregnant women and those with uncontrolled infections or certain complications from sickle cell anemia are excluded.

Inclusion Criteria

I am 40 years old or younger. If I have sickle cell anemia, I am at least 2 years old.

I need a second stem cell transplant from a donor.

I have a matched unrelated donor for my treatment.

My family donor matches me at least 50% for the transplant.

Exclusion Criteria

I have sickle cell anemia but no recent severe infections, HIV, or recent HCT.

I do not have any untreated infections.

Treatment Details

The study tests CD34 Stem Cell Selection Therapy in patients receiving allogeneic peripheral blood stem cell transplants. It aims to assess the treatment's impact on graft versus host disease, graft rejection, survival rates, immune system recovery timeframes, and infection incidences post-transplant.

1Treatment groups

Experimental Treatment

Group I: CliniMACS PLUS followed by chemotherapyExperimental Treatment1 Intervention

Patients will receive a pre-transplant conditioning regimen of Busulfan Fludarabine and Alemtuzumab. For patients with pre-transplant hepatic dysfunction, Melphalan will be substituted for the Busulfan. For patients receiving a second transplant or a "boost", pre-transplant conditioning based on the clinical condition of the patient will be determined by the Principal Investigator and the patient's bone marrow transplantation (BMT) physician. The donor peripheral blood stem cells will undergo CD34+ selection (Biological/Vaccine: CD34 Stem Cell Selection Therapy). The CliniMACS (PLUS) Reagent System will be used to remove T-cells from the peripheral blood stem cell transplant in order to decrease the risk of acute and chronic graft versus host disease (GVHD).