NCT01177059

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
68

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Dec 2004

Longer than P75 for phase_2

Geographic Reach
2 countries

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 6, 2004

Completed
5.7 years until next milestone

First Submitted

Initial submission to the registry

August 5, 2010

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 6, 2010

Completed
7.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 30, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 30, 2017

Completed
1 year until next milestone

Results Posted

Study results publicly available

December 12, 2018

Completed
Last Updated

December 12, 2018

Status Verified

November 1, 2018

Enrollment Period

13 years

First QC Date

August 5, 2010

Results QC Date

November 20, 2018

Last Update Submit

November 20, 2018

Conditions

HIV-1

Keywords

Gene TherapyAnti-HIV-1 RibozymeOZ1HIV-1 Infections

Outcome Measures

Primary Outcomes (3)

  • Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site

    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.

    Approximately up to 15 years

  • Percentage of Participants With Insertional Oncogenesis

    Percentage of participants with insertional oncogenesis by clonal expansion of cells modified with OZ1/LNL6 were reported.

    Approximately up to 15 years

  • Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time

    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.

    Up to end of study (Approximately up to 15 years)

Study Arms (1)

Anti-HIV-1 Ribozyme (OZ1) transduced cells

OTHER

OZ1 transduced cells Long term follow up of previously infused OZ1 transduced cells

Genetic: OZ1 transduced cells

Interventions

OZ1 transduced cellsGENETIC

Long term follow up of previously infused OZ1 transduced cells

Anti-HIV-1 Ribozyme (OZ1) transduced cells

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Unknown Facility

Los Angeles, California, United States

Location

Unknown Facility

San Francisco, California, United States

Location

Unknown Facility

New York, New York, United States

Location

Unknown Facility

Darlinghurst, Australia

Location

Unknown Facility

Surry Hills, Australia

Location

Unknown Facility

Sydney, Australia

Location

Results Point of Contact

Title
Executive Director Medical & Scientific
Organization
Janssen-Cilag Pty Ltd, Australia
ClinicalTrialDisclosure@its.jnj.com
844-434-4210

Study Officials

  • Janssen-Cilag Pty Ltd Clinical Trial

    Janssen-Cilag Pty Ltd

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 5, 2010

First Posted

August 6, 2010

Study Start

December 6, 2004

Primary Completion

November 30, 2017

Study Completion

November 30, 2017

Last Updated

December 12, 2018

Results First Posted

December 12, 2018

Record last verified: 2018-11

Locations

US(3)AU(3)