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
CompletedPhase 2Results postedLast updated 12 December 2018
What this trial tests
Phase 2 trial testing OZ1 transduced cells in HIV-1 in 68 participants. Completed in 30 November 2017.
Eligibility, any sex, with HIV-1. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion SitePrimary· 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.
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
0
Percentage of Participants With Insertional OncogenesisPrimary· Approximately up to 15 years
Percentage of participants with insertional oncogenesis by clonal expansion of cells modified with OZ1/LNL6 were reported.
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
0
Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over TimePrimary· 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.
2 years post-infusion: Not Detected
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
33
2 years post-infusion: 1/3 Detected
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
4
2 years post-infusion: 2/3 Detected
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
0
2 years post-infusion: 3/3 Detected
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
0
2 years post-infusion: Detected (Quantifiable)
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
0
2.5 years post-infusion: Not Detected
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
31
2.5 years post-infusion: 1/3 Detected
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
2
2.5 years post-infusion: 2/3 Detected
Group
Value
95% CI
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
1
Adverse events — posted to ClinicalTrials.gov
Time frame: Approximately up to 15 years.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Serious: 7/68 (10%)
Deaths: 0/68
Serious adverse events (11 terms)
Reaction
System
Anti-HIV-1 Ribozyme (OZ1) …
Rib Fracture
Injury, poisoning and procedural complications
—
Basal Cell Carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
Hodgkin's Disease
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
Kaposi's Sarcoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
Papillary Thyroid Cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
Skin Cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
Testicle Adenoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT06517693 — Safety and Pharmacokinetics Study of PGT121.414.LS Alone and in Combination With VRC07-523LS in Infants Exposed to HIV-1
· Phase 1
· recruiting
NCT06252402 — CMV-specific HIV-CAR T Cells as Immunotherapy for HIV/AIDS
· EARLY_PHASE1
· recruiting
NCT05612178 — A Study to Evaluate the Safety and Effects of Repeated Doses of 3BNC117-LS and 10-1074-LS on Persistent Viral Reservoirs
· Phase 1
· active not recruiting
NCT00980538 — TMC125-TiDP35-C239 - Continued Access to Etravirine (ETR) in Treatment Experienced HIV-1 Infected Participants
· Phase 3
· active not recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Janssen-Cilag Pty Ltd
Last refreshed: 12 December 2018
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01177059.