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Stem Cell Gene Therapy for Sickle Cell Disease

NCT02247843 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2025-12-17

No results posted yet for this study

Summary

This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified peripheral blood for adults with severe sickle cell disease.

Conditions

Interventions

BIOLOGICAL

βAS3-FB vector transduced peripheral blood CD34+ cells

CD34+ from the peripheral blood of patients with sickle cell disease (SCD) are transduced ex-vivo with the Lenti/βAS3-FB lentiviral vector. The transduced cells are then infused into the patient.

Sponsors & Collaborators

  • California Institute for Regenerative Medicine (CIRM)

    collaborator OTHER

  • Donald B. Kohn, M.D.

    lead OTHER

Principal Investigators

  • Donald Kohn, MD · University of California, Los Angeles

  • Gary Schiller, MD · University of California, Los Angeles

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-12-31
Primary Completion
2025-09-24
Completion
2025-09-24
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02247843 on ClinicalTrials.gov