NCT00074997

Brief Summary

The purpose of this study is to determine the safety and efficacy of administration of a cell-delivered ribozyme gene transfer product to participants with chronic (lasting a long time) Human Immunodeficiency Virus Type 1 (HIV-1) infection (a life-threatening infection which you can get from an infected person's blood or from having sex with an infected person).

Trial Health

87
On Track

Trial Health Score

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

Enrollment
1

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2002

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

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 1, 2002

Completed
1.1 years until next milestone

First Submitted

Initial submission to the registry

December 28, 2003

Completed
3 days until next milestone

First Posted

Study publicly available on registry

December 31, 2003

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2008

Completed
Last Updated

June 20, 2016

Status Verified

June 1, 2016

Enrollment Period

5.1 years

First QC Date

December 28, 2003

Last Update Submit

June 17, 2016

Conditions

HIV-1

Keywords

Gene TherapyAnti-HIV-1 RibozymeOZ1HIV-1 Infections

Outcome Measures

Primary Outcomes (2)

  • Difference in viral load between the placebo and OZ1 groups.

    Week 47

  • Difference in viral load between the placebo and OZ1 groups.

    Week 48

Secondary Outcomes (4)

  • CD4+ cell count

    Weeks 41 - 48

  • HIV proviral DNA

    Weeks 41 - 48

  • Thymic function

    Weeks 41 - 48

  • Time to resumption of antiretroviral therapy

    Weeks 41 - 48

Study Arms (2)

001

EXPERIMENTAL

OZ1 Single intravenous infusion of 2-20 x 10 to the power of 7 OZ1 transduced autologous CD34+ cells per kilogram of body weight

Genetic: OZ1Genetic: CD34+ cells

002

PLACEBO COMPARATOR

Placebo Single intravenous infusion of placebo transduced autologous CD34+ cells per kilogram of body weight

Other: PlaceboGenetic: CD34+ cells

Interventions

PlaceboOTHER

Single intravenous infusion of placebo.

002
OZ1GENETIC

Single intravenous infusion of OZ1.

001
CD34+ cellsGENETIC

Autologous CD34+ cells.

001002

Eligibility Criteria

Age18 Years - 45 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • An Human Immunodeficiency Virus 1 (HIV-1) infection for at least 6 months documented by positive HIV serology including confirmation by Western Blot
  • Receiving either the first or second regimen of antiretroviral therapy (ART) defined as 3 or more antiretroviral drugs in combination
  • A Viral load less than 400 copies per milliliter (copies/ml), as measured by Roche Amplicor HIV-1 Monitor assay, on 2 consecutive occasions, at least 7 days apart and within 45 days prior to granulocyte colony-stimulating factor (G-CSF) administration and the second measurement has to be within 14 days prior to G-CSF
  • Cluster of Differentiation 4 (CD4+) cell count must be greater that 300 cells per cubic millimeter (cells/mm\^3)
  • Women and men (or their partners) had to agree to use a medically accepted form of contraception and safe sexual practices

You may not qualify if:

  • Any previous or current Acquired Immunodeficiency Syndrome (AIDS) defining illness by the Center for Disease Control (CDC) case definition, including AIDS-related dementia (mental decline), with the exception of Kaposi's sarcoma (purple or brown cancerous pimples on the skin, often associated with AIDS)
  • Clinically significant clinical laboratory results
  • Participants with veins unsuitable for study related procedures
  • Current ART that included antiretroviral agents which exhibited antagonism when used together (example, zidovudine and stavudine ), or current or previous ART that included hydroxyurea
  • Current pregnancy or breastfeeding

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Unknown Facility

Darlinghurst, Australia

Location

Related Publications (1)

  • Mitsuyasu RT, Merigan TC, Carr A, Zack JA, Winters MA, Workman C, Bloch M, Lalezari J, Becker S, Thornton L, Akil B, Khanlou H, Finlayson R, McFarlane R, Smith DE, Garsia R, Ma D, Law M, Murray JM, von Kalle C, Ely JA, Patino SM, Knop AE, Wong P, Todd AV, Haughton M, Fuery C, Macpherson JL, Symonds GP, Evans LA, Pond SM, Cooper DA. Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells. Nat Med. 2009 Mar;15(3):285-92. doi: 10.1038/nm.1932. Epub 2009 Feb 15.

    PMID: 19219022DERIVED

MeSH Terms

Interventions

betibeglogene autotemcel

Study Officials

  • Janssen-Cilag Pty Ltd Clinical Trial

    Janssen-Cilag Pty Ltd

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 28, 2003

First Posted

December 31, 2003

Study Start

December 1, 2002

Primary Completion

January 1, 2008

Study Completion

January 1, 2008

Last Updated

June 20, 2016

Record last verified: 2016-06

Locations

AU(1)