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 : Unknown
Verified May 2017 by Chen Hu, Affiliated Hospital to Academy of Military Medical Sciences.
Recruitment status was: Recruiting
First Posted : May 23, 2017
Last Update Posted : May 23, 2017
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|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
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|Ages Eligible for Study:||18 Years to 60 Years (Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Age between 18 to 60, male of female;
- Hematological neoplasms;
- HIV-1 R5 tropic virus with no CXCR4-tropic or R5/X4 dual-tropic HIV;
- On ART with undetectable HIV-1 level (<40gc/ml, HIV-1 RNA);
- Availability of a consenting HLA-matched donor;
- No cardiomyopathy or congestive heart failure;
- CD4+ T-cell counts ≥200 cells/µL and ≤750 cells/µL;
- Absence of psychosocial conditions and be willing to comply with study-mandated evaluations for 2 years;
- Life expectancy of at least 1 year.
- Acute or chronic hepatitis B or hepatitis C infection;
- Any cancer or malignancy other than hematological neoplasms;
- Subject with CMV retinitis or other active CMV infection related diseases;
- Subject with organ dysfunction;
- Non-pregnant and non-nursing;
- Drug or alcohol abuse or dependence;
- Currently enrolled in another clinical trial or underwent cell therapy;
- Donor incapable for HSPC mobilization;
- in the opinion of the site investigator, would interfere with adherence to study requirements.
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, PhDfirstname.lastname@example.org|
|Contact: Hu Chen, MD, PhDemail@example.com|
|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 firstname.lastname@example.org|
|Contact: Lei Xu, MD, PhD email@example.com|
|Principal Investigator: Hu Chen, MD, PhD|
|Principal Investigator: Hongkui Deng, PhD|
|Principal Investigator: Hao Wu, MD, PhD|
|Responsible Party:||Chen Hu, Study Director, Affiliated Hospital to Academy of Military Medical Sciences|
|Other Study ID Numbers:||
|First Posted:||May 23, 2017 Key Record Dates|
|Last Update Posted:||May 23, 2017|
|Last Verified:||May 2017|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|