Long Term Follow-Up Study of Human Immunodeficiency Virus Type 1 (HIV-1) Positive Patients Who Have Received OZ1 Gene Therapy as Part of a Clinical Trial

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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A Long Term Follow-up Protocol to Evaluate the Safety and Survival of Autologous CD34+ Hematopoietic Progenitor Cells Transduced With an Anti-HIV-1 Ribozyme (OZ1) in Patients With HIV-1 Infection

Conditions
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HIV-1

Intervention / Treatment
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* Genetic: OZ1 transduced cells


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Anti-HIV-1 Ribozyme (OZ1) transduced cells<br>OZ1 transduced cells Long term follow up of previously infused OZ1 transduced cells | Genetic: OZ1 transduced cells<br>* Long term follow up of previously infused OZ1 transduced cells<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Gene Therapy, Anti-HIV-1 Ribozyme, OZ1, HIV-1 Infections