HIV InfectionsPhase 1NCT03517631
An Efficacy and Safety Study of shRNA-modified CD34+ Cells in HIV-infected Patients.
A Phase 1 study testing shRNA-modified CD34+ cells, Low dose busulfan preconditioning and Busulfan preconditioning in people with HIV Infections and AIDS, run by Shanghai Public Health Clinical Center at 1 site in China.
Trial 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 more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- HIV Infections, AIDS
- What is being tested?
- shRNA-modified CD34+ cells, Low dose busulfan preconditioning and Busulfan preconditioning
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 27 Feb 2018to 31 Dec 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Shanghai Public Health Clinical Center
Shanghai, Shanghai Municipality, 201508
Li Liu, Ph.D, 86-021-37990333 ext. 3222, liulishaphc@163.com
About this study
Official title: A Pilot Study to Evaluate Efficacy and Safety of Multiplexed shRNA-modified CD34+ Cells in HIV-infected Patients.
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.
- Conditions
- HIV Infections, AIDS
- Being tested
- shRNA-modified CD34+ cells, Low dose busulfan preconditioning, Busulfan preconditioning
- Keywords
- HIV, AIDS, Lentivirus, shRNA, Multiplex, Gene therapy
- Medical subject headings
- HIV Infections, Acquired Immunodeficiency Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
shRNA-modified CD34+ cellsBiological
Infusion of CD34+ cells transduced with shRNAs.
Low dose busulfan preconditioningDrug
Also called Busulfex
A single dose of 1 mg/kg busulfan administered every 6 hours for 4 times as preconditioning for transplantation.
Busulfan preconditioningDrug
Also called Busulfex
A single dose of 1 mg/kg busulfan administered every 6 hours for 8 times as preconditioning for transplantation.
| Study group | Role | What they receive |
|---|---|---|
| No busulfan preconditioning | Experimental | shRNA-modified CD34+ cells without busulfan preconditioning. Receives: Biological: shRNA-modified CD34+ cells |
| Low dose busulfan preconditioning | Experimental | shRNA-modified CD34+ cells, with a single dose of 1 mg/kg busulfan administered every 6 hours for 4 times as preconditioning for transplantation. Receives: Biological: shRNA-modified CD34+ cells; Drug: Low dose busulfan preconditioning |
| High dose busulfan preconditioning | Experimental | shRNA-modified CD34+ cells, with a single dose of 1 mg/kg busulfan administered every 6 hours for 8 times as preconditioning for transplantation. Receives: Biological: shRNA-modified CD34+ cells; Drug: Busulfan preconditioning |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse side effects
Patients will be monitored for any signs of adverse effects.
Measured: 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.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2018
- Study start27 Feb 2018
- Primary completion15 Dec 2020 (estimated)
- Study completion31 Dec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HIV InfectionsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Infusion of autologous CD34+ cells transduced with shRNAs targeting CCR5 and HIV genome
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Public Health Clinical Center
- Sponsor type
- Other government
- Collaborators
- R&D Kanglin Biotech
- Responsible party
- Principal investigator, Hongzhou Lu, Shanghai Public Health Clinical Center
- Investigators
- Hongzhou Lu, Ph.D, Study director, Caolang Road NO. 2901, Jinshan District, Shanghai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03517631
- Sponsor's protocol ID
- KL1702
- Status last verified
- Feb 2020
- First submitted
- 20 Mar 2018
- Last updated
- 19 Feb 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Evaluate Efficacy and Safety of Multiplexed shRNA-modified CD34+ Cells in HIV-infected Patients.
- ClinicalTrials.gov ID
- NCT03517631
- Acronym / sponsor ID
- KL1702
- Conditions
- HIV Infections, AIDS
- Keywords
- HIV, AIDS, Lentivirus, shRNA, Multiplex, Gene therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Shanghai Public Health Clinical CenterOther government
- Collaborators
- R&D Kanglin Biotech
- Study start
- 27 Feb 2018 (actual)
- Primary completion
- 15 Dec 2020 (estimated)
- Study completion
- 31 Dec 2020 (estimated)
- First posted
- 7 May 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Infusion of autologous CD34+ cells transduced with shRNAs targeting CCR5 and HIV genome
| Arm | Type | Receives |
|---|---|---|
| No busulfan preconditioning | Experimental | Biological: shRNA-modified CD34+ cellsshRNA-modified CD34+ cells without busulfan preconditioning. |
| Low dose busulfan preconditioning | Experimental | Biological: shRNA-modified CD34+ cells, Drug: Low dose busulfan preconditioningshRNA-modified CD34+ cells, with a single dose of 1 mg/kg busulfan administered every 6 hours for 4 times as preconditioning for transplantation. |
| High dose busulfan preconditioning | Experimental | Biological: shRNA-modified CD34+ cells, Drug: Busulfan preconditioningshRNA-modified CD34+ cells, with a single dose of 1 mg/kg busulfan administered every 6 hours for 8 times as preconditioning for transplantation. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (13 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| shRNA-modified CD34+ cellsInfusion of CD34+ cells transduced with shRNAs. | Biological | High dose busulfan preconditioning, Low dose busulfan preconditioning, No busulfan preconditioning |
| Low dose busulfan preconditioningAlso: BusulfexA single dose of 1 mg/kg busulfan administered every 6 hours for 4 times as preconditioning for transplantation. | Drug | Low dose busulfan preconditioning |
| Busulfan preconditioningAlso: BusulfexA single dose of 1 mg/kg busulfan administered every 6 hours for 8 times as preconditioning for transplantation. | Drug | High dose busulfan preconditioning |
Primary outcome
Adverse side effects
Time frame: 18 months
Patients will be monitored for any signs of adverse effects.
1 secondary outcome
Efficacy of treatment
Time frame: 18 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hongzhou Lu, Ph.D study director
Caolang Road NO. 2901, Jinshan District, Shanghai
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing shRNA-modified CD34+ cells, Low dose busulfan preconditioning and Busulfan preconditioning in people with HIV Infections and AIDS, run by Shanghai Public Health Clinical Center at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Hongzhou Lu, M.D., Ph.D (+86-021-37990333 ext. 3222, luhongzhou@fudan.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse side effects (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Feb 2020.