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Status unknownNCT03517631Updated Feb 19, 2020

An Efficacy and Safety Study of shRNA-modified CD34+ Cells in HIV-infected Patients.

A Phase 1 interventional study of shRNA-modified CD34+ cells and Low dose busulfan preconditioning in HIV Infections and AIDS, sponsored by Shanghai Public Health Clinical Center. Status unknown at 1 site in China. Open to participants aged 18 Years to 50 Years. Per ClinicalTrials.gov, last updated 2020-02-19.

Sponsored by Shanghai Public Health Clinical Center · Phase 1, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Feb 2020), so the status shown — last known as Recruiting — may be out of date.
Phase
Phase 1
Study type
Interventional
Enrollment
6
Allocation
Non-randomized
Ages
18 Years to 50 Years
Sex
All
01

Study summary

The purpose of the study is to evaluate the efficacy and safety of autologous CD34+ cells that stably express multiplexed shRNA to treat HIV infection.

Read the detailed description

CD34+ cells will be isolated from mobilized PBMC of HIV patients. The cells will be lentivirally transduced with multiplexed shRNAs in the same vector that target CCR5 and HIV genome. Such modified cells will be infused back to the patients who have received bulsufan preconditioning before infusion. The patients will then be evaluated for efficacy and safety.

02

Conditions studied

  • HIV Infections
  • AIDS

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Keywords

  • HIV
  • AIDS
  • Lentivirus
  • shRNA
  • Multiplex
  • Gene therapy
03

In context

HIV Infections

4,258 studies on the registry are indexed under HIV Infections; 240 are open to participants now.

This study's planned enrollment of 6 is below the median of 83 across 3,251 interventional studies indexed under HIV Infections.

Browse HIV Infections studies →

Lead sponsor

Shanghai Public Health Clinical Center is the lead sponsor of 37 studies on the registry; 11 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 50 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Body mass index (BMI) from 18 - 25; body weight ≥50kg.
  • Diagnosis of HIV infections/AIDS based on "Diagnostic Criteria for HIV/AIDS" WS 293-2008.
  • No antiretroviral therapy (ART) in the past 6 weeks and refuse to receive ART.
  • CD4 T cell count ≥350/μl.
  • No plan for pregnancy in the near future and agree to practice non-drug based contraception.
  • Voluntary to participate in the study, comply with the study design to complete all monitory measurements, and agree to sign the study agreement.

Exclusion criteria

Exclusion Criteria:

  • Existence of infections/opportunistic tumors.
  • Mutations in the shRNA target sequences.
  • White blood cell count \<3x10\^9/L, neutrophil count \<1.5x10\^9/L, hemoglobin \<110g/L, platelet count \<100x10\^9/L.
  • Liver diseases (HBV, chronic HCV infection, congenital liver metabolic disease), abnormal liver functions (test value 2x above normal).
  • Kidney deficiency (Creatinine level above the upper limit of normal levels).
  • Severe chronic disease, metabolic disease (e.g., diabetes), neurological and psychiatric diseases.
  • History of pancreatitis.
  • Women in pregnancy, lactating or at reproductive age who do not practice contraception.
  • Allergy to agents or drugs used in the study.
  • Verified or suspected abuse of alcohol and drugs.
  • Participated in other clinical trials within 3 months.
  • Based on investigator's assessment, those who are unfit for the study (e.g., general weakness, poor compliance with study design).
  • Personal or family history of tumors.
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
6 participants (estimated)

Study arms

  • Experimental
    No busulfan preconditioning

    shRNA-modified CD34+ cells without busulfan preconditioning.

    Biological: shRNA-modified CD34+ cells

  • Experimental
    Low dose busulfan preconditioning

    shRNA-modified CD34+ cells, with a single dose of 1 mg/kg busulfan administered every 6 hours for 4 times as preconditioning for transplantation.

    Biological: shRNA-modified CD34+ cells · Drug: Low dose busulfan preconditioning

  • Experimental
    High dose busulfan preconditioning

    shRNA-modified CD34+ cells, with a single dose of 1 mg/kg busulfan administered every 6 hours for 8 times as preconditioning for transplantation.

    Biological: shRNA-modified CD34+ cells · Drug: Busulfan preconditioning

Interventions

  • BiologicalshRNA-modified CD34+ cells

    Infusion of CD34+ cells transduced with shRNAs.

  • DrugLow dose busulfan preconditioning

    A single dose of 1 mg/kg busulfan administered every 6 hours for 4 times as preconditioning for transplantation.

    Also known as: Busulfex

  • DrugBusulfan preconditioning

    A single dose of 1 mg/kg busulfan administered every 6 hours for 8 times as preconditioning for transplantation.

    Also known as: Busulfex

06

What researchers measure

Primary outcomes

  1. Adverse side effects

    Patients will be monitored for any signs of adverse effects.

    Time frame: 18 months

Secondary outcomes

  1. Efficacy of treatment

    Evaluate the efficacy of modified autologous CD34+ cells by monitoring CD4 counts and HIV-1 RNA level.

    Time frame: 18 months

07

Study locations

1 of 1 sites recruiting
  • Shanghai Public Health Clinical Center
    Shanghai, Shanghai 201508, China
    Recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 19, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03517631
Lead sponsor
Shanghai Public Health Clinical Center
Collaborators
R&D Kanglin Biotech
Responsible party
Hongzhou Lu (professor, Shanghai Public Health Clinical Center) — Principal investigator
First posted
May 7, 2018
Start date
Feb 27, 2018
Primary completion
Dec 15, 2020 (estimated)
Completion
Dec 31, 2020 (estimated)
Last update
Feb 19, 2020

Study contacts

Hongzhou Lu, M.D., Ph.D
Contact
luhongzhou@fudan.edu.cn
+86-021-37990333 ext. 3222
Li Liu, M.D., Ph.D
Contact
liulishaphc@163.com
86-021-37990333 ext. 3222
Hongzhou Lu, Ph.D
study director · Caolang Road NO. 2901, Jinshan District, Shanghai

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Feb 2020. You cannot join it, but the record below documents what was studied.

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