Active, not recruitingPhase 2NCT05353647

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

Studying Sickle cell disease

Last synced from ClinicalTrials.gov

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Key facts

Sponsor
David Williams
Principal Investigator
David Williams
Boston Children's Hospital
Intervention
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a(biological)
Enrollment
25 enrolled
Eligibility
13-40 years · All sexes
Timeline
20222027

Study locations (9)

Collaborators

National Heart, Lung, and Blood Institute (NHLBI) · California Institute for Regenerative Medicine (CIRM) · Genetix Biotherapeutics Inc. · Blood and Marrow Transplant Clinical Trials Network

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT05353647 on ClinicalTrials.gov

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