Summary

Eligibility
for people ages 13-40 (full criteria)
Location
at UC Davis UCLA UCSF
Dates
study started
study ends around

Description

Summary

Official Title

A Multi-Center, Phase 2 Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell (GRASP, BMT CTN 2001)

Details

Keywords

Sickle Cell Disease, gene therapy, lentivirus vector, BCL11A, fetal hemoglobin, Sickle Cell Anemia, Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a

Eligibility

Locations

  • UCLA Medical Center
    Los Angeles California 90095 United States
  • UCSF Benioff Children's Hospital Oakland
    Oakland California 94609 United States
  • UC Davis Medical Center
    Sacramento California 95817 United States
  • Children's Hospital of Los Angeles
    Los Angeles California 90027 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
David Williams
ID
NCT05353647
Phase
Phase 2 research study
Study Type
Interventional
Participants
About 25 people participating
Last Updated