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

Official Title: “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”

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 for the rest of their lives. The study will monitor for and record any ill effects from the gene therapy product to provide long term safety information.

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 for the rest of their lives. 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.

Intervention(s) in this Clinical Trial

• Genetic: OZ1 transduced cells
  • Long term follow up of previously infused OZ1 transduced cells

Arms, Groups and Cohorts in this Clinical Trial

• : 001
  • OZ1 transduced cells Long term follow up of previously infused OZ1 transduced cells

Primary Measures

• Adverse events, reportable events such as cancers and deaths
  • Time Frame: Lifetime of the patient. Each patient will have visits every 6 months until 5 years post infusion, then annual visits for safety assessments, until their death.
    Safety Issue?: Yes

Secondary Measures

• Clonal expansion of cells with a predominant OZ1 insertion site
  • Time Frame: Lifelong follow-up
    Safety Issue?: Yes
• Assess quantitative marking of the gene transfer product in peripheral blood mononuclear cells (PBMC) over time
  • Time Frame: For a minimum of 15 years
    Safety Issue?: Yes

Inclusion Criteria:

• Patients must have received the Final Cell Product infusion in base study
• Have signed Informed Consent Form

Exclusion Criteria:

• Any patient from the base study that was assigned to the placebo arm

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 18 Years

Maximum Age for this Clinical Trial: 45 Years

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Investigator: Janssen-Cilag Pty Ltd Industry

Overall Clinical Trial Officials and Contacts

Janssen-Cilag Pty Ltd Clinical Trial Study Director Janssen-Cilag Pty Ltd  

Information obtained from ClinicalTrials.gov on February 02, 2012

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT01177059

Study ID Number: CR016027

ClinicalTrials.gov Identifier: NCT01177059

Health Authority: United States: Food and Drug Administration