Summary

Eligibility
for people ages 3-40 (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
completion around

Description

Summary

Official Title

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease

Details

Keywords

Sickle Cell Disease, Gene therapy, lentivirus vector, BCL11A, Fetal Hemoglobin, Sickle Cell Anemia, single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a

Eligibility

Locations

  • UCLA - Mattel Children's Hospital
    Los Angeles California 90095 United States
  • Boston Children's Hospital
    Boston Massachusetts 02115 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
David Williams
ID
NCT03282656
Phase
Phase 1 research study
Study Type
Interventional
Participants
About 10 people participating
Last Updated