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This study examines the safety of a dual anti-HIV gene transfer treatment called Cal-1 in people with HIV-1 infection, in which a patient's own blood stem cells and CD4+ T lymphocytes are genetically modified to resist HIV and are given together with Busulfan. It enrolled people aged 18-65 who had been diagnosed with HIV-1 for at least 6 months. The study was carried out in Phase 1/Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Prior to any study-related procedures, signed informed consent indicating that they understand the purpose, risks and procedures required for the study and are willing to participate in the study * Individuals aged 18 to 65 years of age (inclusive) at time of consent * Documented HIV-1 infection ≥ 6 months prior to Screening 1 * Previous treatment with antiretroviral agents that had a demonstrated suppressive effect (defined as plasma HIV RNA ≤ 50 copies/ml) * A documented viable ART regimen option, as determined by the Investigator, taking into account prior ART experience and HIV geno/phenotyping analyses * Not taking antiretroviral therapy for ≥ 6 weeks prior to Screening 1, for one or more of the following reasons: i) Concerns over short-term or long-term toxicities associated with antiretroviral agents, or ii) Treatment fatigue from the daily regimen of life-long therapy * Plasma HIV-1 viral RNA ≥ 5,000 copies/mL and ≤ 100,000 copies/ml at Screening 1 and Screening 2 * CD4+ T lymphocyte count ≥ 500 cells/µl at Screening 1 and Screening 2 Exclusion Criteria: * Abnormal hematology at Screening 1: Absolute neutrophil count (ANC) \< 1.5 x 109/L, Platelet count \< 100 x 109/L, Hemoglobin \< 10 g/dL * Abnormal biochemistry at Screening 1: Alanine aminotransferase (ALT) \> 2.5 x Upper Limit of Normal (ULN), Total bilirubin \> 1.5 x ULN, Serum creatinine \> 1.5 x ULN * Detection of any CXCR4-tropic HIV-1 at Screening 1 * Evidence of co-infection with hepatitis B virus, hepatitis C virus, West Nile Virus, or HTLV-1 as detected at Screening 2 * Evidence of active TB infection determined by positive QuantiFERON®-TB Gold/IGRA test result and clinical confirmation at Screening 2 * ART or other antiretroviral therapy within 6 weeks of Screening 1 or any time during the pre-infusion period * Documented history of CD4+ T lymphocyte count \< 250 cells/µl * Any previous or current AIDS-defining illnesses (CDC Category C), including AIDS-related dementia, with the exception of Kaposi's sarcoma confined to the skin * History of malignancy or systemic chemotherapy within the last 5 years (i.e., subjects with prior malignancy must be disease-free for 5 years), except curatively-treated basal cell carcinoma, cutaneous squamous cell carcinoma, or cervical or anal intra-epithelial neoplasia * History of steroid-dependent asthma in the past 5 years * History of seizure * Any clinical history of hematologic diseases including leukemia, myelodysplasia, myeloproliferative disease, thromboembolic disease, sickle cell disorder, thrombocytopenia or leukopenia * Class II-IV heart failure, according to the New York Heart Association classification * Inadequate venous access for apheresis, as assessed at Screening 1 * Current or planned systemic immunosuppressive or immunomodulatory medication * Taking warfarin, aspirin or any medication that is likely to affect platelet function or other aspects of blood coagulation, and unable to safely cease this medication for a period of 1 week prior, during, and 1 week after administration of G-CSF (a total period of 19 days) * Participation in any study involving any investigational drug or medical device within 30 days prior to Screening 1 * Receipt of a vaccine for HIV-1 or any gene transfer product at any time * Prior treatment with recombinant G-CSF or busulfan or other stem-cell mobilizing or modulating agent within the previous 12 months * Known hypersensitivity to busulfan, G-CSF (Neupogen™) or E. coli-derived proteins * Subjects who will not accept transfusions of blood products * Pregnant or breast-feeding at any time between Screening 1 and Baseline (infusion) * History of alcohol or drug abuse within the 12 months prior to Screening 1 * Inability to understand and provide informed consent