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Gene Transfer for Patients With Sickle Cell Disease

NCT02186418 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2024-04-12

No results posted yet for this study

Summary

The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease

Conditions

  • Anemia, Sickle Cell

Interventions

GENETIC

ARU-1801

Autologous CD34+ hematopoietic stem cells transduced ex-vivo with a gamma-globin lentiviral vector * Subjects with sickle cell anemia will undergo hematopoietic stem cell procurement by bone marrow harvest or apheresis after mobilization with plerixafor * Reduced intensity chemotherapy conditioning with single dose melphalan will be used to facilitate engraftment of ex-vivo transduced cells.

Sponsors & Collaborators

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Stella M. Davies, MB BS, PhD, MRCP · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation
NA
Purpose
SUPPORTIVE_CARE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-07-31
Primary Completion
2022-10-31
Completion
2022-10-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

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 NCT02186418 on ClinicalTrials.gov