Gene Transfer for Patients With Sickle Cell Disease

Status Terminated

Clinical Trial 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

Clinical Trial Description

This study will assess the feasibility, safety and efficacy of gene transfer using ARU-1801 (CD34+ cells transduced with the gamma-globin lentiviral vector). Gene transfer will occur ex-vivo into CD34+ enriched human bone marrow or plerixafor-mobilized peripheral blood hematopoietic stem cells (HSC) collected from subjects with severe sickle cell disease (SCD). Subjects will undergo reduced intensity chemotherapy conditioning with single-dose melphalan to facilitate engraftment of ex-vivo ARU-1801 via IV infusion. Subjects will return to the study site at regular intervals for follow-up for 2 years after the ARU-1801 infusion. It is anticipated that a separate long-term follow-up (LTFU) clinical study will be initiated, in which all subjects completing the 2 year study visit will be asked to consent and enroll, and will followed for a further 13 years. ;

Study Design

Related Conditions & MeSH terms

Anemia, Sickle Cell

Clinical Trial Details

NCT number	NCT02186418
Study type	Interventional
Source	Children's Hospital Medical Center, Cincinnati
Contact
Status	Terminated
Phase	Phase 1/Phase 2
Start date	July 2014
Completion date	October 31, 2022

View Details

See also
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