Safety of Transplantation of CRISPR CCR5 Modified CD34+ Cells in HIV-infected Subjects With Hematological Malignances
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|ClinicalTrials.gov Identifier: NCT03164135|
Recruitment Status : Recruiting
First Posted : May 23, 2017
Last Update Posted : May 23, 2017
|Condition or disease||Intervention/treatment||Phase|
|HIV-1-infection||Genetic: CCR5 gene modification||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||CD34+ hematopoietic stem/progenitor cells from donor are treated with CRISPR/Cas9 before transplantation into the patient.|
|Masking:||None (Open Label)|
|Official Title:||Safety and Feasibility Study of Allotransplantation of CRISPR/Cas9 CCR5 Gene Modified CD34+ Hematopoietic Stem/Progenitor Cells in HIV-infected Subjects With Hematological Malignances|
|Estimated Study Start Date :||May 30, 2017|
|Estimated Primary Completion Date :||May 20, 2019|
|Estimated Study Completion Date :||May 20, 2021|
Experimental: CCR5 gene modification
CD34+ hematopoietic stem/progenitor cells from donor are treated with CRISPR/Cas9 before transplantation into the patient.
Genetic: CCR5 gene modification
CD34+ hematopoietic stem/progenitor cells from donor are treated with CRISPR/Cas9 targeting CCR5 gene.
- Persistence of CCR5 gene disruption in engrafted cells [ Time Frame: 12 months ]Participants will be transplanted with CD34+ cells which are treated using the CRISPR/Cas9 system to disrupt CCR5 gene. The persistence of CCR5 gene disruption in engrafted cells will be evaluated by sequencing.
- CD34+ cell number [ Time Frame: the first month ]The CD34+ cell number pre-infusion
- Gene disruption efficiency of bone marrow cells [ Time Frame: Up to Month 12 ]The percentage of disrupted CCR5 gene alleles in genome from bone marrow cells detected by sequencing.
- CCR5 gene disruption efficiency of peripheral blood cells [ Time Frame: Up to Month 12 ]The percentage of disrupted CCR5 gene alleles in genome of peripheral blood cells by sequencing.
- Hematopoietic cell engraftment [ Time Frame: Up to Year 3 ]Measurement of multi-lineage hematopoietic cell engraftment time after transplantation to evaluate the hematological recovery
- HIV-1 RNA level [ Time Frame: Up to Year 3 ]Level change of HIV-1 RNA in plasma after transplantation
- CD4+ T cell number [ Time Frame: Up to Year 3 ]Level change of the CD4+ T cell number after transplantation
- The ratio change of CD4/CD8 [ Time Frame: Up to Year 3 ]The ratio change of CD4/CD8 in peripheral blood after transplantation
- HIV-1 RNA levels during ATI [ Time Frame: Every two weeks, until the end of ATI or up to 3 months ]HIV-1 RNA levels in plasma during ATI.
- HIV-1 DNA level [ Time Frame: Up to Month 12 ]Changes of proviral DNA in PBMC pre- transplantation and 12 month post-transplantation
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03164135
|Contact: Bin Zhang, MD, PhDemail@example.com|
|Contact: Hu Chen, MD, PhDfirstname.lastname@example.org|
|307 Hospital of PLA (Affiliated Hospital of Academy to Military Medical Sciences)||Recruiting|
|Beijing, Beijing, China, 100071|
|Contact: Bin Zhang, MD, PhD +86-10-66947625 email@example.com|
|Contact: Lei Xu, MD, PhD firstname.lastname@example.org|
|Principal Investigator: Hu Chen, MD, PhD|
|Principal Investigator: Hongkui Deng, PhD|
|Principal Investigator: Hao Wu, MD, PhD|