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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 Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Other
Condition HIV-1
Intervention Genetic: OZ1 transduced cells
Enrollment 68
Recruitment Details  
Pre-assignment Details Participants who completed original study (OTH/OZ1-INT-1 [NCT00074997]) were enrolled in this long term follow-up (LTFU) study. After unblinding of original study, participants in placebo group were withdrawn from LTFU study, except first participant who received Moloney murine leukemia virus based retroviral vector (LNL6) transduced CD34+ cells.
Arm/Group Title Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Hide Arm/Group Description Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study.
Period Title: Overall Study
Started 68
Completed 18
Not Completed 50
Reason Not Completed
Received placebo in OTH/OZ1-INT-1 study             30
Withdrew consent             2
Lost to Follow-up             18
Arm/Group Title Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Hide Arm/Group Description Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study.
Overall Number of Baseline Participants 68
Hide Baseline Analysis Population Description
The baseline (safety) population included all participants who were enrolled in the original OTH/OZ1-INT-1 study and continued participation in the long term follow-up OZ1-HV1-202/OZ1HIV2001 study.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 68 participants
40.2  (5.79)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 68 participants
Female
4
   5.9%
Male
64
  94.1%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 68 participants
African American
5
   7.4%
Asian
2
   2.9%
Caucasian
53
  77.9%
Hispanic
6
   8.8%
Maori/Polynesian
1
   1.5%
Other
1
   1.5%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 68 participants
Australia
32
  47.1%
United States
36
  52.9%
1.Primary Outcome
Title Percentage of Participants With Clonal Expansion of Cells With a Predominant OZ1 Insertion Site
Hide Description 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.
Time Frame Approximately up to 15 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Per protocol (PP) population set included all participants in the safety population who received OZ1 or Moloney murine leukemia virus based retroviral vector (LNL6) transduced final cell product in the original OTH/OZ1-INT-1 study.
Arm/Group Title Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Hide Arm/Group Description:
Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study.
Overall Number of Participants Analyzed 37
Measure Type: Number
Unit of Measure: Percentage of participants
0
2.Primary Outcome
Title Percentage of Participants With Insertional Oncogenesis
Hide Description Percentage of participants with insertional oncogenesis by clonal expansion of cells modified with OZ1/LNL6 were reported.
Time Frame Approximately up to 15 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Per protocol population set included all participants in the safety population who received OZ1 or LNL6 transduced final cell product in the original OTH/OZ1-INT-1 study.
Arm/Group Title Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Hide Arm/Group Description:
Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study.
Overall Number of Participants Analyzed 37
Measure Type: Number
Unit of Measure: Percentage of participants
0
3.Primary Outcome
Title Number of Participants With Quantitative Marking of Gene Transfer Product in Peripheral Blood Mononuclear Cells (PBMC) Over Time
Hide Description 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.
Time Frame Up to end of study (Approximately up to 15 years)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP population set included all participants in safety population who received OZ1/LNL6 transduced final cell product in original OTH/OZ1-INT-1 study. Here ‘n’ specifies participants analyzed for this endpoint at given time point.
Arm/Group Title Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Hide Arm/Group Description:
Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study.
Overall Number of Participants Analyzed 37
Measure Type: Number
Unit of Measure: Participants
2 years post-infusion: Not Detected Number Analyzed 37 participants
33
2 years post-infusion: 1/3 Detected Number Analyzed 37 participants
4
2 years post-infusion: 2/3 Detected Number Analyzed 37 participants
0
2 years post-infusion: 3/3 Detected Number Analyzed 37 participants
0
2 years post-infusion: Detected (Quantifiable) Number Analyzed 37 participants
0
2.5 years post-infusion: Not Detected Number Analyzed 34 participants
31
2.5 years post-infusion: 1/3 Detected Number Analyzed 34 participants
2
2.5 years post-infusion: 2/3 Detected Number Analyzed 34 participants
1
2.5 years post-infusion: 3/3 Detected Number Analyzed 34 participants
0
2.5 years post-infusion: Detected (Quantifiable) Number Analyzed 34 participants
0
3 years post-infusion: Not Detected Number Analyzed 35 participants
31
3 years post-infusion: 1/3 Detected Number Analyzed 35 participants
3
3 years post-infusion: 2/3 Detected Number Analyzed 35 participants
0
3 years post-infusion: 3/3 Detected Number Analyzed 35 participants
1
3 years post-infusion: Detected (Quantifiable) Number Analyzed 35 participants
0
3.5 years post-infusion: Not Detected Number Analyzed 28 participants
28
3.5 years post-infusion: 1/3 Detected Number Analyzed 28 participants
0
3.5 years post-infusion: 2/3 Detected Number Analyzed 28 participants
0
3.5 years post-infusion: 3/3 Detected Number Analyzed 28 participants
0
3.5 years post-infusion: Detected (Quantifiable) Number Analyzed 28 participants
0
4 years post-infusion: Not Detected Number Analyzed 32 participants
28
4 years post-infusion: 1/3 Detected Number Analyzed 32 participants
4
4 years post-infusion: 2/3 Detected Number Analyzed 32 participants
0
4 years post-infusion: 3/3 Detected Number Analyzed 32 participants
0
4 years post-infusion: Detected (Quantifiable) Number Analyzed 32 participants
0
4.5 years post-infusion: Not Detected Number Analyzed 25 participants
19
4.5 years post-infusion: 1/3 Detected Number Analyzed 25 participants
5
4.5 years post-infusion: 2/3 Detected Number Analyzed 25 participants
1
4.5 years post-infusion: 3/3 Detected Number Analyzed 25 participants
0
4.5 years post-infusion: Detected (Quantifiable) Number Analyzed 25 participants
0
5 years post-infusion: Not Detected Number Analyzed 27 participants
27
5 years post-infusion: 1/3 Detected Number Analyzed 27 participants
0
5 years post-infusion: 2/3 Detected Number Analyzed 27 participants
0
5 years post-infusion: 3/3 Detected Number Analyzed 27 participants
0
5 years post-infusion: Detected (Quantifiable) Number Analyzed 27 participants
0
6 years post-infusion: Not Detected Number Analyzed 20 participants
18
6 years post-infusion: 1/3 Detected Number Analyzed 20 participants
0
6 years post-infusion: 2/3 Detected Number Analyzed 20 participants
1
6 years post-infusion: 3/3 Detected Number Analyzed 20 participants
0
6 years post-infusion: Detected (Quantifiable) Number Analyzed 20 participants
1
7 years post-infusion: Not Detected Number Analyzed 10 participants
9
7 years post-infusion: 1/3 Detected Number Analyzed 10 participants
0
7 years post-infusion: 2/3 Detected Number Analyzed 10 participants
0
7 years post-infusion: 3/3 Detected Number Analyzed 10 participants
1
7 years post-infusion: Detected (Quantifiable) Number Analyzed 10 participants
0
8 years post-infusion: Not Detected Number Analyzed 9 participants
9
8 years post-infusion: 1/3 Detected Number Analyzed 9 participants
0
8 years post-infusion: 2/3 Detected Number Analyzed 9 participants
0
8 years post-infusion: 3/3 Detected Number Analyzed 9 participants
0
8 years post-infusion: Detected (Quantifiable) Number Analyzed 9 participants
0
9 years post-infusion: Not Detected Number Analyzed 7 participants
6
9 years post-infusion: 1/3 Detected Number Analyzed 7 participants
1
9 years post-infusion: 2/3 Detected Number Analyzed 7 participants
0
9 years post-infusion: 3/3 Detected Number Analyzed 7 participants
0
9 years post-infusion: Detected (Quantifiable) Number Analyzed 7 participants
0
10 years post-infusion: Not Detected Number Analyzed 10 participants
9
10 years post-infusion: 1/3 Detected Number Analyzed 10 participants
1
10 years post-infusion: 2/3 Detected Number Analyzed 10 participants
0
10 years post-infusion: 3/3 Detected Number Analyzed 10 participants
0
10 years post-infusion: Detected (Quantifiable) Number Analyzed 10 participants
0
11 years post-infusion: Not Detected Number Analyzed 8 participants
7
11 years post-infusion: 1/3 Detected Number Analyzed 8 participants
1
11 years post-infusion: 2/3 Detected Number Analyzed 8 participants
0
11 years post-infusion: 3/3 Detected Number Analyzed 8 participants
0
11 years post-infusion: Detected (Quantifiable) Number Analyzed 8 participants
0
12 years post-infusion: Not Detected Number Analyzed 7 participants
7
12 years post-infusion: 1/3 Detected Number Analyzed 7 participants
0
12 years post-infusion: 2/3 Detected Number Analyzed 7 participants
0
12 years post-infusion: 3/3 Detected Number Analyzed 7 participants
0
12 years post-infusion: Detected (Quantifiable) Number Analyzed 7 participants
0
13 years post-infusion: Not Detected Number Analyzed 3 participants
3
13 years post-infusion: 1/3 Detected Number Analyzed 3 participants
0
13 years post-infusion: 2/3 Detected Number Analyzed 3 participants
0
13 years post-infusion: 3/3 Detected Number Analyzed 3 participants
0
13 years post-infusion: Detected (Quantifiable) Number Analyzed 3 participants
0
14 years post-infusion: Not Detected Number Analyzed 1 participants
1
14 years post-infusion: 1/3 Detected Number Analyzed 1 participants
0
14 years post-infusion: 2/3 Detected Number Analyzed 1 participants
0
14 years post-infusion: 3/3 Detected Number Analyzed 1 participants
0
14 years post-infusion: Detected (Quantifiable) Number Analyzed 1 participants
0
End of study: Not Detected Number Analyzed 6 participants
6
End of study: 1/3 Detected Number Analyzed 6 participants
0
End of study: 2/3 Detected Number Analyzed 6 participants
0
End of study: 3/3 Detected Number Analyzed 6 participants
0
End of study: Detected (Quantifiable) Number Analyzed 6 participants
0
Time Frame Approximately up to 15 years
Adverse Event Reporting Description The safety population included all participants who were enrolled in the original OTH/OZ1-INT-1 study and continued participation in the long term follow-up OZ1-HV1-202/OZ1HIV2001 study.
 
Arm/Group Title Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Hide Arm/Group Description Participants who received an infusion of final cell product (that is, a cluster of differentiation [CD]34+ cells with or without gene transfer product) in the original study (OTH/OZ1-INT-1) were followed up in this study.
All-Cause Mortality
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Affected / at Risk (%)
Total   0/68 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Affected / at Risk (%)
Total   7/68 (10.29%) 
Injury, poisoning and procedural complications   
Rib Fracture * 1  1/68 (1.47%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Basal Cell Carcinoma * 1  1/68 (1.47%) 
Hodgkin's Disease * 1  1/68 (1.47%) 
Kaposi's Sarcoma * 1  1/68 (1.47%) 
Papillary Thyroid Cancer * 1  1/68 (1.47%) 
Skin Cancer * 1  1/68 (1.47%) 
Testicle Adenoma * 1  1/68 (1.47%) 
Pregnancy, puerperium and perinatal conditions   
Premature Baby * 1  1/68 (1.47%) 
Psychiatric disorders   
Suicidal Ideation * 1  1/68 (1.47%) 
Respiratory, thoracic and mediastinal disorders   
Pulmonary Embolism * 1  1/68 (1.47%) 
Vascular disorders   
Deep Vein Thrombosis * 1  1/68 (1.47%) 
1
Term from vocabulary, MedDRA Version 20.0
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Anti-HIV-1 Ribozyme (OZ1) Transduced Cells
Affected / at Risk (%)
Total   1/68 (1.47%) 
Vascular disorders   
Hypertension * 1  1/68 (1.47%) 
1
Term from vocabulary, MedDRA Version 20.0
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
A copy of the manuscript must be provided to the sponsor for review at least 60 days before submission for publication or presentation. If requested in writing, such publication will be withheld for up to an additional 60 days.
Results Point of Contact
Name/Title: Executive Director Medical & Scientific
Organization: Janssen-Cilag Pty Ltd, Australia
Phone: 844-434-4210
Responsible Party: Janssen-Cilag Pty Ltd
ClinicalTrials.gov Identifier: NCT01177059     History of Changes
Other Study ID Numbers: CR016027
OZ1-HV1-202 ( Other Identifier: Janssen-Cilag Pty Ltd, Australia )
First Submitted: August 5, 2010
First Posted: August 6, 2010
Results First Submitted: November 20, 2018
Results First Posted: December 12, 2018
Last Update Posted: December 12, 2018