Gene Transfer for Patients With Sickle Cell Disease
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ClinicalTrials.gov Identifier: NCT02186418 |
Recruitment Status :
Active, not recruiting
First Posted : July 10, 2014
Last Update Posted : November 17, 2022
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Sponsor:
Children's Hospital Medical Center, Cincinnati
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
Tracking Information | |||||
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First Submitted Date ICMJE | June 30, 2014 | ||||
First Posted Date ICMJE | July 10, 2014 | ||||
Last Update Posted Date | November 17, 2022 | ||||
Actual Study Start Date ICMJE | July 2014 | ||||
Estimated Primary Completion Date | June 2023 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Not Provided | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Gene Transfer for Patients With Sickle Cell Disease | ||||
Official Title ICMJE | Gene Transfer for Patients With Sickle Cell Disease Using a Gamma Globin Lentivirus Vector: An Open Label Phase 1/2 Pilot Study | ||||
Brief 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 | ||||
Detailed 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 Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Intervention Model Description: Gamma Globin Lentivirus Vector Masking: None (Open Label)Primary Purpose: Treatment |
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Condition ICMJE | Anemia, Sickle Cell | ||||
Intervention ICMJE | Genetic: ARU-1801
Autologous CD34+ hematopoietic stem cells transduced ex-vivo with a gamma-globin lentiviral vector
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Study Arms ICMJE | Experimental: ARU-1801
Autologous CD34+ hematopoietic stem cells transduced ex-vivo with gamma-globin lentiviral vector. Administered via IV infusion.
Intervention: Genetic: ARU-1801
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Actual Enrollment ICMJE |
7 | ||||
Original Estimated Enrollment ICMJE |
10 | ||||
Estimated Study Completion Date ICMJE | June 2035 | ||||
Estimated Primary Completion Date | June 2023 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria
Exclusion Criteria
Other protocol-defined inclusion-exclusion criteria may apply. |
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years to 45 Years (Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | Canada, Jamaica, Switzerland | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT02186418 | ||||
Other Study ID Numbers ICMJE | ARU-1801_Ph1_01 | ||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Children's Hospital Medical Center, Cincinnati | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Children's Hospital Medical Center, Cincinnati | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Children's Hospital Medical Center, Cincinnati | ||||
Verification Date | July 2022 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |