Trials / Active Not Recruiting
Active Not RecruitingNCT02500849
Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients
A Pilot Study to Evaluate the Feasibility, Safety and Engraftment of Zinc Finger Nuclease (ZFN) CCR5 Modified CD34+ Hematopoietic Stem/Progenitor Cells (SB-728mR-HSPC) in HIV-1 (R5) Infected Patients
- Status
- Active Not Recruiting
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- Sponsor
- City of Hope Medical Center · Academic / Other
- Sex
- All
- Age
- 18 Years – 75 Years
- Healthy volunteers
- Not accepted
Summary
The purpose of the study is to evaluate the safety and feasibility of administering SB-728mR-HSPC after conditioning with busulfan.
Detailed description
The objective of the study is to evaluate the safety and feasibility of giving autologous SB-728mR-HSPC to HIV-1 (R5) infected patients who are being treated with cART and have undetectable virus but suboptimal CD4+ cell levels. To strengthen the possibility that CCR5-disrupted HSPCs engraft, patients will receive either a two- or three-day (Cohort 1 or Cohort 2) course of busulfan (dose targeting AUC of 4000 µM/day) before being infused with the genetically modified cells. At 9-12 months after SB-728mR-HSPC infusion, subjects who are aviremic with CD4 cell counts ≥600 cells/µL and have ≥1% CCR5-modified CD4 cells within the peripheral blood detected by pentamer PCR will undergo an ATI.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | SB-728mR-HSPC Infusion 3 days following busulfan conditioning |
Timeline
- Start date
- 2016-03-10
- Primary completion
- 2026-08-24
- Completion
- 2026-08-24
- First posted
- 2015-07-17
- Last updated
- 2025-10-09
Locations
5 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT02500849. Inclusion in this directory is not an endorsement.