Phase 2
Completed N=68
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
Source: ClinicalTrials.gov NCT01177059 ↗Enrolled (actual)
68
Serious AEs
10.3%
Results posted
Dec 2018
Primary outcomePrimary: Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site — 0 Percentage of participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site |
— | — |
| PRIMARY Percentage of Participants With Insertional Oncogenesis |
— | — |
| PRIMARY Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time |
33; 4; 0; 0; 0; 31 | — |
Eligibility Criteria
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 After unblinding of the OTH/OZ1-INT-1 study, any patients in the placebo arm will be withdrawn from this protocol
Data sourced from ClinicalTrials.gov (NCT01177059). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.