Clinical trial NCT01177059: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: HIV-1
Keywords: Gene Therapy
Anti-HIV-1 Ribozyme
OZ1
HIV-1 Infections
Lead sponsor: Janssen-Cilag Pty Ltd
Sponsor class: INDUSTRY
First posted: 2010-08-06
Enrollment: 68
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.

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

Primary outcomes:
measure: Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site
timeFrame: Approximately up to 15 years
measure: Percentage of Participants With Insertional Oncogenesis
timeFrame: Approximately up to 15 years
measure: Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time
timeFrame: Up to end of study (Approximately up to 15 years)
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
state: California
city: San Francisco
country: United States
state: California
city: New York
country: United States
state: New York
city: Darlinghurst
country: Australia
city: Surry Hills
country: Australia
city: Sydney
country: Australia
Study officials:
affiliation: Janssen-Cilag Pty Ltd
name: Janssen-Cilag Pty Ltd Clinical Trial
role: STUDY_DIRECTOR
References: