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| ID | Type | Description | Link |
|---|---|---|---|
| OZ1-HV1-202 | Other Identifier | Janssen-Cilag Pty Ltd, Australia |
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The purpose of this Observational Study is long term follow-up of the Human Immunodeficiency Virus -1 (HIV-1) infected patients who have received a gene therapy product (anti-HIV-1 Ribozyme [OZ1]) as part of an earlier phase 2 trial. Patients are seen twice yearly until 5 years from initial infusion of study drug has elapsed and then yearly until withdrawal or study completion. The study will monitor for and record any ill effects from the gene therapy product to provide long term safety information.
During an earlier phase 2 base trial (study # NCT00074997), HIV-1 infected patients received a gene therapy product (anti-HIV-1 Ribozyme [OZ1]). Gene therapy is an investigational treatment where genes are inserted into an individual's cells and tissues to treat a disease. The gene therapy OZ1 is thought to have anti-HIV-1 actions. This is an Observational Study to provide long term follow-up of those HIV-1 infected patients who received the gene therapy product (anti-HIV-1 Ribozyme [OZ1]) as part of the earlier study. Patients are seen twice yearly until 5 years from initial infusion of study drug has elapsed and then yearly until withdrawal or study completion. The study will monitor for and record any ill effects from the gene therapy product to provide long term safety information. Observational study - no investigational drug administered.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Anti-HIV-1 Ribozyme (OZ1) transduced cells | Other | OZ1 transduced cells Long term follow up of previously infused OZ1 transduced cells |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| OZ1 transduced cells | Genetic | Long term follow up of previously infused OZ1 transduced cells |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site | Percentage of participants with clonal expansion of cells with a predominant OZ1 insertion site was reported. A predominant integration site was defined as an integration site which has a density of at least 50 percent (%) of the total signal detected by polymerase chain reaction (PCR), when the percentage of cells marked by vector was greater than (>)1% of the test cell population. | Approximately up to 15 years |
| Percentage of Participants With Insertional Oncogenesis | Percentage of participants with insertional oncogenesis by clonal expansion of cells modified with OZ1/LNL6 were reported. | Approximately up to 15 years |
| Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | OZ1 and LNL6 marking analysis were performed by quantitative deoxyribonucleic acid-polymerase chain reaction (DNA-PCR). Number of participants in each of 3 categories for gene detection: Not Detected, Detected (1, 2 and 3 of the 3 triplicates of the sample were detected respectively [1/3 Detected, 2/3 Detected, 3/3 Detected]) and Detected (Quantifiable) were reported for marking of gene transfer product in PBMC. | Up to end of study (Approximately up to 15 years) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Janssen-Cilag Pty Ltd Clinical Trial | Janssen-Cilag Pty Ltd | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Los Angeles | California | United States | ||||
Participants who completed original study (OTH/OZ1-INT-1 [NCT00074997]) were enrolled in this long term follow-up (LTFU) study. After unblinding of original study, participants in placebo group were withdrawn from LTFU study, except first participant who received Moloney murine leukemia virus based retroviral vector (LNL6) transduced CD34+ cells.
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| ID | Title | Description |
|---|---|---|
| FG000 | Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 7, 2017 | Nov 20, 2018 |
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| San Francisco |
| California |
| United States |
| New York | New York | United States |
| Darlinghurst | Australia |
| Surry Hills | Australia |
| Sydney | Australia |
| COMPLETED |
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| NOT COMPLETED |
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The baseline (safety) population included all participants who were enrolled in the original OTH/OZ1-INT-1 study and continued participation in the long term follow-up OZ1-HV1-202/OZ1HIV2001 study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Region of Enrollment | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site | Percentage of participants with clonal expansion of cells with a predominant OZ1 insertion site was reported. A predominant integration site was defined as an integration site which has a density of at least 50 percent (%) of the total signal detected by polymerase chain reaction (PCR), when the percentage of cells marked by vector was greater than (>)1% of the test cell population. | Per protocol (PP) population set included all participants in the safety population who received OZ1 or Moloney murine leukemia virus based retroviral vector (LNL6) transduced final cell product in the original OTH/OZ1-INT-1 study. | Posted | Number | Percentage of participants | Approximately up to 15 years |
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| Primary | Percentage of Participants With Insertional Oncogenesis | Percentage of participants with insertional oncogenesis by clonal expansion of cells modified with OZ1/LNL6 were reported. | Per protocol population set included all participants in the safety population who received OZ1 or LNL6 transduced final cell product in the original OTH/OZ1-INT-1 study. | Posted | Number | Percentage of participants | Approximately up to 15 years |
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| Primary | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | OZ1 and LNL6 marking analysis were performed by quantitative deoxyribonucleic acid-polymerase chain reaction (DNA-PCR). Number of participants in each of 3 categories for gene detection: Not Detected, Detected (1, 2 and 3 of the 3 triplicates of the sample were detected respectively [1/3 Detected, 2/3 Detected, 3/3 Detected]) and Detected (Quantifiable) were reported for marking of gene transfer product in PBMC. | PP population set included all participants in safety population who received OZ1/LNL6 transduced final cell product in original OTH/OZ1-INT-1 study. Here 'n' specifies participants analyzed for this endpoint at given time point. | Posted | Number | Participants | Up to end of study (Approximately up to 15 years) |
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Approximately up to 15 years
The safety population included all participants who were enrolled in the original OTH/OZ1-INT-1 study and continued participation in the long term follow-up OZ1-HV1-202/OZ1HIV2001 study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study. | 0 | 68 | 7 | 68 | 1 | 68 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Rib Fracture | Injury, poisoning and procedural complications | MedDRA Version 20.0 | Non-systematic Assessment |
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| Basal Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA Version 20.0 | Non-systematic Assessment |
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| Hodgkin's Disease | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA Version 20.0 | Non-systematic Assessment |
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| Kaposi's Sarcoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA Version 20.0 | Non-systematic Assessment |
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| Papillary Thyroid Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA Version 20.0 | Non-systematic Assessment |
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| Skin Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA Version 20.0 | Non-systematic Assessment |
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| Testicle Adenoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA Version 20.0 | Non-systematic Assessment |
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| Premature Baby | Pregnancy, puerperium and perinatal conditions | MedDRA Version 20.0 | Non-systematic Assessment |
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| Suicidal Ideation | Psychiatric disorders | MedDRA Version 20.0 | Non-systematic Assessment |
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| Pulmonary Embolism | Respiratory, thoracic and mediastinal disorders | MedDRA Version 20.0 | Non-systematic Assessment |
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| Deep Vein Thrombosis | Vascular disorders | MedDRA Version 20.0 | Non-systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypertension | Vascular disorders | MedDRA Version 20.0 | Non-systematic Assessment |
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A copy of the manuscript must be provided to the sponsor for review at least 60 days before submission for publication or presentation. If requested in writing, such publication will be withheld for up to an additional 60 days.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Executive Director Medical & Scientific | Janssen-Cilag Pty Ltd, Australia | 844-434-4210 | ClinicalTrialDisclosure@its.jnj.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 7, 2017 | Nov 20, 2018 | SAP_001.pdf |
| Caucasian |
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| Hispanic |
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| Maori/Polynesian |
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| Other |
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