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Trial record 1 of 1 for:    OZ1-HV1-202
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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01177059
Recruitment Status : Completed
First Posted : August 6, 2010
Results First Posted : December 12, 2018
Last Update Posted : December 12, 2018
Sponsor:
Information provided by (Responsible Party):
Janssen-Cilag Pty Ltd

Tracking Information
First Submitted Date  ICMJE August 5, 2010
First Posted Date  ICMJE August 6, 2010
Results First Submitted Date  ICMJE November 20, 2018
Results First Posted Date  ICMJE December 12, 2018
Last Update Posted Date December 12, 2018
Actual Study Start Date  ICMJE December 6, 2004
Actual Primary Completion Date November 30, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 20, 2018)
  • Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site [ Time Frame: 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 [ Time Frame: Approximately up to 15 years ]
    Percentage of participants with insertional oncogenesis by clonal expansion of cells modified with OZ1/LNL6 were reported.
  • Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time [ Time Frame: 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.
Original Primary Outcome Measures  ICMJE
 (submitted: August 5, 2010)
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. ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: August 5, 2010)
  • Clonal expansion of cells with a predominant OZ1 insertion site [ Time Frame: Lifelong follow-up ]
  • Assess quantitative marking of the gene transfer product in peripheral blood mononuclear cells over time [ Time Frame: For a minimum of 15 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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  ICMJE 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
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE HIV-1
Intervention  ICMJE Genetic: OZ1 transduced cells
Long term follow up of previously infused OZ1 transduced cells
Study Arms  ICMJE Anti-HIV-1 Ribozyme (OZ1) transduced cells
OZ1 transduced cells Long term follow up of previously infused OZ1 transduced cells
Intervention: Genetic: OZ1 transduced cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 28, 2018)
68
Original Actual Enrollment  ICMJE
 (submitted: August 5, 2010)
37
Actual Study Completion Date  ICMJE November 30, 2017
Actual Primary Completion Date November 30, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01177059
Other Study ID Numbers  ICMJE CR016027
OZ1-HV1-202 ( Other Identifier: Janssen-Cilag Pty Ltd, Australia )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Janssen-Cilag Pty Ltd
Study Sponsor  ICMJE Janssen-Cilag Pty Ltd
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Janssen-Cilag Pty Ltd Clinical Trial Janssen-Cilag Pty Ltd
PRS Account Janssen-Cilag Pty Ltd
Verification Date November 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP