Safety of Transplantation of CRISPR CCR5 Modified CD34+ Cells in HIV-infected Subjects With Hematological Malignances
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|