Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection
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ClinicalTrials.gov Identifier: NCT01734850 |
Recruitment Status :
Completed
First Posted : November 28, 2012
Results First Posted : August 6, 2020
Last Update Posted : August 6, 2020
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Tracking Information | ||||
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First Submitted Date ICMJE | November 19, 2012 | |||
First Posted Date ICMJE | November 28, 2012 | |||
Results First Submitted Date ICMJE | June 25, 2020 | |||
Results First Posted Date ICMJE | August 6, 2020 | |||
Last Update Posted Date | August 6, 2020 | |||
Actual Study Start Date ICMJE | April 2013 | |||
Actual Primary Completion Date | September 2017 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
Feasibility and safety of the introduction of Cal-1 into HSPC and CD4+ T lymphocyte cell populations, and the associated delivery procedures. [ Time Frame: 48 weeks ] Baseline demographic and clinical information, treatment, and duration of follow-up will be summarized by group and overall. Severity of clinical adverse events (AEs) and laboratory safety parameters will be graded according to DAIDS criteria. Numbers of individual AEs, in numbers of subjects, will be summarized by severity and treatment. AEs will also be summarized and aggregated by body system for clinical events. The worst severity of each clinical and laboratory AE, for each subject, will be summarized by treatment.
All grade 3 and 4 AEs, and any other events that lead to a subject ceasing trial follow-up, will be listed by treatment, with duration and resolution.
Feasibility measures include the number of manufacturing procedures successfully completed (i.e. complying with all release criteria); number of gene modified cells, as affected by CD4+ and CD34+ purity, transduction efficiency, and viability; and the number of target cells harvested.
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Change History | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
The benefit of busulfan conditioning in impacting efficacy of Cal-1 in controlling HIV-1 infection [ Time Frame: 48 weeks ] Log10 HIV-1 RNA, CD4+ T lymphocyte count, CD4+ %, CD4:CD8 ratio, measures of Cal-1 marking/expression and thymopoiesis will be plotted over time for each individual subject, and will be summarized for each nominal study week.
Changes in Log10 HIV-1 RNA and CD4+ T lymphocyte count from baseline will be formally compared in all subjects.
Associations between feasibility and safety measures and secondary outcomes include:
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Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection | |||
Official Title ICMJE | An Adaptive Phase I/II Study of the Safety of CD4+ T Lymphocytes and CD34+ Hematopoietic Stem/Progenitor Cells Transduced With LVsh5/C46, a Dual Anti-HIV Gene Transfer Construct, With and Without Conditioning With Busulfan in HIV-1 Infected Adults Previously Exposed to ART | |||
Brief Summary | This is an early phase research study looking at whether an experimental gene transfer, LVsh5/C46 (also known as Cal-1), is safe and if it can protect the immune system from the effects of HIV without the use of antiretroviral drugs. Cal-1 is an experimental gene transfer agent designed to inhibit HIV infection through 2 active parts:
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Detailed Description | It is estimated that 33 million individuals are currently infected with HIV. HIV/AIDS is a disease that impairs immune function, primarily by decreasing CD4+ T lymphocytes. The progression can be contained by daily dosing with antiretroviral therapy (ART) but there are side effects that can be treatment limiting, and the development of HIV drug resistance can force the physician to modify the ART regimen. There are no effective vaccines currently available for HIV. LVsh5/C46 (also known as Cal-1) is a dual therapeutic, self-inactivating lentiviral vector that encodes for both a short hairpin RNA against the HIV-1 co-receptor CCR5 (sh5) and a HIV-1 fusion inhibitor, C46 and inhibits two processes required for HIV-1 infection:
The rationale is that Cal-1 introduced into hematopoietic progenitor/stem cells (HSPC) and mature CD4+ T lymphocytes will protect these cells and their progeny cells from HIV-1 infection and its pathogenic sequelae. This may provide a continuous means of controlling HIV-1 after a single or infrequent dose(s), thereby decreasing or delaying (partially or completely) the need for antiretroviral drug therapy. |
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Study Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Human Immunodeficiency Virus | |||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
13 | |||
Original Estimated Enrollment ICMJE |
12 | |||
Actual Study Completion Date ICMJE | November 2017 | |||
Actual Primary Completion Date | September 2017 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years to 65 Years (Adult, Older Adult) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT01734850 | |||
Other Study ID Numbers ICMJE | CAL-USA-11 | |||
Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE | Not Provided | |||
Responsible Party | Calimmune, Inc. | |||
Study Sponsor ICMJE | Calimmune, Inc. | |||
Collaborators ICMJE | Not Provided | |||
Investigators ICMJE |
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PRS Account | Calimmune, Inc. | |||
Verification Date | July 2020 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |