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Gene Therapy

Gene-Edited Stem Cell Therapy for HIV/AIDS

Phase 1
Waitlist Available
Led By Amrita Y. Krishnan, MD
Research Sponsored by City of Hope Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Maximum age 75 years for cohort 1 and 65 years for cohort 2
HIV-1 R5 seropositive with no evidence of CXCR4-tropic virus
Must not have
Any AIDS-related opportunistic infection occurring within the past year such as tuberculosis, cryptococcosis and for which treatment has been unsuccessful as determined by the Principal Investigator
Participants may not be receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy. Participation in prior investigational drug or medical device study within the previous 45 days
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial will test whether it is safe to give a person with leukemia a new treatment that uses their own stem cells.

Who is the study for?
This trial is for HIV-1 infected patients under 75 years old, with controlled virus levels due to consistent antiretroviral therapy (cART) for at least a year, and specific CD4+ T-cell counts. They must not have significant heart issues or psychosocial conditions affecting study participation. Excluded are those with active hepatitis, pregnant or nursing women, recent investigational drug use, substance abuse history, certain hematologic diseases, recent opportunistic infections or malignancies.
What is being tested?
The trial tests the safety of SB-728mR-HSPC infusion in HIV/AIDS patients after busulfan conditioning. SB-728mR-HSPC is a genetically modified stem/progenitor cell product aimed at altering the immune system to resist HIV more effectively.
What are the potential side effects?
Potential side effects may include reactions related to the infusion process itself and complications from busulfan conditioning like lowered blood counts leading to increased infection risk. Genetic modification might also pose risks that will be closely monitored.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 75 or younger for cohort 1, and 65 or younger for cohort 2.
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I am HIV positive with only the R5 virus strain.
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I have been on HIV treatment with undetectable levels for at least a year.
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I do not have serious heart disease or heart failure.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had an AIDS-related infection in the last year that hasn't improved with treatment.
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I am not currently on any experimental treatments or have been in a drug/device study in the last 45 days.
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I am currently using or have used steroids or drugs that affect the immune system.
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I have previously received an HIV vaccine or gene therapy.
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I have an active CMV infection affecting my eyes or other organs.
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I am taking AZT or maraviroc in my HIV treatment.
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I have a history of serious blood disorders.
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My virus is CXCR4-tropic.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort 2:Experimental Treatment1 Intervention
busulfan AUC levels: 12,000 µM\*min (+/- 1,000)
Group II: Cohort 1:Experimental Treatment1 Intervention
target busulfan AUC levels: 8,000 µM\*min (+/- 1,000)

Find a Location

Who is running the clinical trial?

City of Hope Medical CenterLead Sponsor
605 Previous Clinical Trials
1,923,593 Total Patients Enrolled
Sangamo TherapeuticsIndustry Sponsor
28 Previous Clinical Trials
937 Total Patients Enrolled
California Institute for Regenerative Medicine (CIRM)OTHER
69 Previous Clinical Trials
3,350 Total Patients Enrolled
Amrita Y. Krishnan, MDPrincipal InvestigatorCity of Hope Medical Center
3 Previous Clinical Trials
152 Total Patients Enrolled

Media Library

SB-728mR-HSPC (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02500849 — Phase 1
HIV/AIDS Research Study Groups: Cohort 1:, Cohort 2:
HIV/AIDS Clinical Trial 2023: SB-728mR-HSPC Highlights & Side Effects. Trial Name: NCT02500849 — Phase 1
SB-728mR-HSPC (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02500849 — Phase 1
~1 spots leftby Dec 2025