Stem Cell Gene Therapy for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

Overseen byGary Schiller, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Donald B. Kohn, M.D.

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial tests a new treatment for adults with severe sickle cell disease using their own modified blood cells. The treatment aims to fix the genetic problem causing sickle cell disease and help patients produce healthy red blood cells. This approach could be better than current treatments and avoid complications from donor transplants. The new therapy involves modifying the patient's own blood cells to correct the genetic issue.

Eligibility Criteria

Adults with severe sickle cell disease who have had complications like pain crises, stroke, or acute chest syndrome despite treatment can join this trial. They must not be pregnant, have HIV/HCV/HTLV-1/hepatitis B/Cancer, and should not have a suitable sibling donor for transplant.

Inclusion Criteria

I do not have a perfect match donor for a stem cell transplant or I refuse to have one.

My condition did not improve after taking hydroxyurea for 3 months.

I have been hospitalized for sickle cell pain crises at least twice.

+17 more

Exclusion Criteria

I don't have any other cancer or active infections.

My blood pressure is high (>135/95) despite taking medication.

I do not have active infections like HIV, Hepatitis B/C, HTLV-1, CMV, or parvovirus B19.

+10 more

Participant Groups

The trial is testing a gene therapy where patients' own blood stem cells are modified with the βAS3-FB vector to potentially treat sickle cell disease. It's in Phase I to check safety and initial effectiveness.

1Treatment groups

Experimental Treatment

Group I: βAS3-FB vector transduced peripheral blood CD34+ cellsExperimental Treatment1 Intervention

This is a single arm study without randomization. All subjects will receive the intervention of BetaAS3 lentiviral vector-modified autologous peripheral blood stem cell transplant.