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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

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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

Study Description
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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.

Condition or disease Intervention/treatment Phase
HIV-1 Genetic: OZ1 transduced cells Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 68 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
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
Actual Study Start Date : December 6, 2004
Actual Primary Completion Date : November 30, 2017
Actual Study Completion Date : November 30, 2017

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Anti-HIV-1 Ribozyme (OZ1) transduced cells
OZ1 transduced cells Long term follow up of previously infused OZ1 transduced cells
 Genetic: OZ1 transduced cells
Long term follow up of previously infused OZ1 transduced cells


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01177059


Locations
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United States, California
Los Angeles, California, United States
San Francisco, California, United States
United States, New York
New York, New York, United States
Australia
Darlinghurst, Australia
Surry Hills, Australia
Sydney, Australia
Sponsors and Collaborators
Janssen-Cilag Pty Ltd
Investigators
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Study Director: Janssen-Cilag Pty Ltd Clinical Trial Janssen-Cilag Pty Ltd
  Study Documents (Full-Text)

Documents provided by Janssen-Cilag Pty Ltd:
Study Protocol  [PDF] June 7, 2017
Statistical Analysis Plan  [PDF] November 7, 2017

More Information
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Responsible Party: Janssen-Cilag Pty Ltd
ClinicalTrials.gov Identifier: NCT01177059    
Other Study ID Numbers: CR016027
OZ1-HV1-202 ( Other Identifier: Janssen-Cilag Pty Ltd, Australia )
First Posted: August 6, 2010    Key Record Dates
Results First Posted: December 12, 2018
Last Update Posted: December 12, 2018
Last Verified: November 2018

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Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Janssen-Cilag Pty Ltd:
Gene Therapy
Anti-HIV-1 Ribozyme
OZ1
HIV-1 Infections