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

Phase 2

Completed

• Conditions: HIV-1
• Interventions: Genetic: OZ1 transduced cells

• Registration Number: NCT01177059
• Lead Sponsor: Janssen-Cilag Pty Ltd

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.

Study Timeline

• Study Start: 2004-12-06
• Study First Submitted: 2010-08-05
• Study First Submitted QC: 2010-08-05
• Study First Posted: 2010-08-06
• Primary Completion: 2017-11-30
• Study Completion: 2017-11-30
• Status Verified: 2018-11
• Results First Submitted: 2018-11-20
• Results First Submitted QC: 2018-11-20
• Last Update Submitted: 2018-11-20
• Results First Posted: 2018-12-12
• Last Update Posted: 2018-12-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 68

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Anti-HIV-1 Ribozyme (OZ1) transduced cells	OZ1 transduced cells	OZ1 transduced cells Long term follow up of previously infused OZ1 transduced cells

Primary Outcome Measures

Name	Time	Method
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.
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.