HIV-1
Conditions
Keywords
Gene Therapy, Anti-HIV-1 Ribozyme, OZ1, HIV-1 Infections
Brief summary
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.
Detailed description
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.
Interventions
GENETICOZ1 transduced cells
Long term follow up of previously infused OZ1 transduced cells
Sponsors
Janssen-Cilag Pty Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* All patients who were enrolled (between 18 and 45 years of age) in the base study OTH/OZ1-INT-1 (NCT00074997) will be invited to participate in this long term follow-up study * Patients must have received the Final Cell Product infusion in base study OTH/OZ1-INT-1 * Have signed Informed Consent Form
Exclusion criteria
* All patients who were enrolled in the OTH/OZ1-INT-1 study and received the final cell product will be invited to participate in this long term follow-up study. After unblinding of the OTH/OZ1-INT-1 study, any patients in the placebo arm will be withdrawn from this protocol
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site | Approximately up to 15 years | 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. |
| Percentage of Participants With Insertional Oncogenesis | Approximately up to 15 years | Percentage of participants with insertional oncogenesis by clonal expansion of cells modified with OZ1/LNL6 were reported. |
| Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | Up to end of study (Approximately up to 15 years) | 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. |
Countries
Australia, United States
Participant flow
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 68 |
| Total | 68 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Lost to Follow-up | 18 |
| Overall Study | Received placebo in OTH/OZ1-INT-1 study | 30 |
| Overall Study | Withdrew consent | 2 |
Baseline characteristics
| Characteristic | Anti-HIV-1 Ribozyme (OZ1) Transduced Cells |
|---|---|
| Age, Continuous | 40.2 Years STANDARD_DEVIATION 5.79 |
| Race/Ethnicity, Customized African American | 5 Participants |
| Race/Ethnicity, Customized Asian | 2 Participants |
| Race/Ethnicity, Customized Caucasian | 53 Participants |
| Race/Ethnicity, Customized Hispanic | 6 Participants |
| Race/Ethnicity, Customized Maori/Polynesian | 1 Participants |
| Race/Ethnicity, Customized Other | 1 Participants |
| Region of Enrollment Australia | 32 Participants |
| Region of Enrollment United States | 36 Participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 64 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 68 |
| other Total, other adverse events | 1 / 68 |
| serious Total, serious adverse events | 7 / 68 |
Outcome results
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.
Time frame: Up to end of study (Approximately up to 15 years)
Population: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 13 years post-infusion: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 14 years post-infusion: Not Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 14 years post-infusion: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 14 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 14 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 14 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | End of study: Not Detected | 6 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | End of study: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | End of study: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | End of study: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | End of study: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2 years post-infusion: Not Detected | 33 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2 years post-infusion: 1/3 Detected | 4 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2.5 years post-infusion: Not Detected | 31 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2.5 years post-infusion: 1/3 Detected | 2 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2.5 years post-infusion: 2/3 Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2.5 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 2.5 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3 years post-infusion: Not Detected | 31 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3 years post-infusion: 1/3 Detected | 3 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3 years post-infusion: 3/3 Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3.5 years post-infusion: Not Detected | 28 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3.5 years post-infusion: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3.5 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3.5 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 3.5 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4 years post-infusion: Not Detected | 28 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4 years post-infusion: 1/3 Detected | 4 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4.5 years post-infusion: Not Detected | 19 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4.5 years post-infusion: 1/3 Detected | 5 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4.5 years post-infusion: 2/3 Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4.5 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 4.5 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 5 years post-infusion: Not Detected | 27 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 5 years post-infusion: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 5 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 5 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 5 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 6 years post-infusion: Not Detected | 18 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 6 years post-infusion: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 6 years post-infusion: 2/3 Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 6 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 6 years post-infusion: Detected (Quantifiable) | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 7 years post-infusion: Not Detected | 9 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 7 years post-infusion: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 7 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 7 years post-infusion: 3/3 Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 7 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 8 years post-infusion: Not Detected | 9 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 8 years post-infusion: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 8 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 8 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 8 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 9 years post-infusion: Not Detected | 6 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 9 years post-infusion: 1/3 Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 9 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 9 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 9 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 10 years post-infusion: Not Detected | 9 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 10 years post-infusion: 1/3 Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 10 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 10 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 10 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 11 years post-infusion: Not Detected | 7 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 11 years post-infusion: 1/3 Detected | 1 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 11 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 11 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 11 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 12 years post-infusion: Not Detected | 7 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 12 years post-infusion: 1/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 12 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 12 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 12 years post-infusion: Detected (Quantifiable) | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 13 years post-infusion: Not Detected | 3 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 13 years post-infusion: 2/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 13 years post-infusion: 3/3 Detected | 0 Participants |
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time | 13 years post-infusion: Detected (Quantifiable) | 0 Participants |
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.
Time frame: Approximately up to 15 years
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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site | 0 Percentage of participants |
Primary
Percentage of Participants With Insertional Oncogenesis
Percentage of participants with insertional oncogenesis by clonal expansion of cells modified with OZ1/LNL6 were reported.
Time frame: Approximately up to 15 years
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anti-HIV-1 Ribozyme (OZ1) Transduced Cells | Percentage of Participants With Insertional Oncogenesis | 0 Percentage of participants |