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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00074997




Registration number
NCT00074997
Ethics application status
Date submitted
28/12/2003
Date registered
31/12/2003
Date last updated
20/06/2016

Titles & IDs
Public title
An Efficacy and Safety Study of Autologous Cluster of Differentiation 34 (CD34+) Hematopoietic Progenitor Cells Transduced With Placebo or an Anti- Human Immunodeficiency Virus Type 1 (HIV-1) Ribozyme (OZ1) in Participants With HIV-1 Infection
Scientific title
A Randomized Phase II, Double-Blind, Controlled Trial to Evaluate the Safety and Efficacy of Autologous CD34+ Hematopoietic Progenitor Cells Transduced With Placebo or an Anti-HIV-1 Ribozyme (OZ1) in Patients With HIV-1 Infection
Secondary ID [1] 0 0
OZ1-HV1-201
Secondary ID [2] 0 0
CR010783
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
HIV-1 0 0
Condition category
Condition code
Infection 0 0 0 0
Studies of infection and infectious agents
Infection 0 0 0 0
Other infectious diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Placebo
Treatment: Other - OZ1
Treatment: Other - CD34+ cells

Experimental: 001 - OZ1 Single intravenous infusion of 2-20 x 10 to the power of 7 OZ1 transduced autologous CD34+ cells per kilogram of body weight

Placebo comparator: 002 - Placebo Single intravenous infusion of placebo transduced autologous CD34+ cells per kilogram of body weight


Other interventions: Placebo
Single intravenous infusion of placebo.

Treatment: Other: OZ1
Single intravenous infusion of OZ1.

Treatment: Other: CD34+ cells
Autologous CD34+ cells.
Intervention code [1] 0 0
Other interventions
Intervention code [2] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Difference in viral load between the placebo and OZ1 groups.
Timepoint [1] 0 0
Week 47
Primary outcome [2] 0 0
Difference in viral load between the placebo and OZ1 groups.
Timepoint [2] 0 0
Week 48
Secondary outcome [1] 0 0
CD4+ cell count
Timepoint [1] 0 0
Weeks 41 - 48
Secondary outcome [2] 0 0
HIV proviral DNA
Timepoint [2] 0 0
Weeks 41 - 48
Secondary outcome [3] 0 0
Thymic function
Timepoint [3] 0 0
Weeks 41 - 48
Secondary outcome [4] 0 0
Time to resumption of antiretroviral therapy
Timepoint [4] 0 0
Weeks 41 - 48

Eligibility
Key inclusion criteria
* 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
Minimum age
18 Years
Maximum age
45 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2002
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/01/2008
Sample size
Target
Accrual to date
Final
1
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Darlinghurst
Recruitment postcode(s) [1] 0 0
- Darlinghurst

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Janssen-Cilag Pty Ltd
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/study/NCT00074997
Trial related presentations / publications
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.
Public notes

Contacts
Principal investigator
Name 0 0
Janssen-Cilag Pty Ltd Clinical Trial
Address 0 0
Janssen-Cilag Pty Ltd
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00074997