Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| OZ1-HV1-201 | |||
| OTH/OZ1-INT-1 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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).
This is a randomized (study drug assigned by chance), double-blind (neither the participant nor the physician know the study medication drug name), placebo (an inactive substance that is compared with a drug to test if the drug has a real effect in a clinical trial) controlled study to investigate safety and efficacy of administration of autologous cluster of differentiation 34 (CD34+) cells transduced with placebo or an anti-HIV-ribozyme (OZ1) in participants with HIV-1 infection. The total study duration will be 100 weeks and will include following visits: screening, pre-infusion Days 1-7, Day 8, Week 1, 4, 8, 12, 16, 20, 24, 25, 26, 27, 28, 30, 32, 34, 36, 38, 40, 41, 42, 43, 44, 45, 46, 47, 48, 52, 56, 60, 64, 68, 72, 76, 80, 84, 88, 92, 96 and 100. Before the administration of CD34+ final cell product, a number of procedures will be performed, including the injection of granulocyte colony-stimulating factor (G-CSF) to mobilize the CD34+ cells, apheresis and the transduction of the CD34+ cells with either OZ1 or placebo. Participants will be divided into two groups: one group will receive OZ1-containing CD34+ cells, the other group will receive CD34+ cells alone and will receive a single intravenous infusion (a fluid or a medicine delivered into a vein by way of a needle) of CD34+ cells transduced with either placebo or OZ1 gene transfer product. The final cell product contains approximately 2-20 x 10^7 cells/kilogram autologous CD34+ cell suspension transduced with either placebo or OZ1 gene transfer product. Primary efficacy will be assessed primarily by the amount of HIV ribonucleic acid (RNA) (viral load). Participants' safety will be monitored throughout the study.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
|
| 002 | Placebo Comparator | Placebo Single intravenous infusion of placebo transduced autologous CD34+ cells per kilogram of body weight |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Other | Single intravenous infusion of placebo. |
| |
| OZ1 |
| Measure | Description | Time Frame |
|---|---|---|
| Difference in viral load between the placebo and OZ1 groups. | Week 47 | |
| Difference in viral load between the placebo and OZ1 groups. | Week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| CD4+ cell count | Weeks 41 - 48 | |
| HIV proviral DNA | Weeks 41 - 48 | |
| Thymic function |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Janssen-Cilag Pty Ltd Clinical Trial | Janssen-Cilag Pty Ltd | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Darlinghurst | Australia |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 19219022 | Derived | 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. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Genetic |
Single intravenous infusion of OZ1. |
|
| CD34+ cells | Genetic | Autologous CD34+ cells. |
|
| Weeks 41 - 48 |
| Time to resumption of antiretroviral therapy | Weeks 41 - 48 |
| ID | Term |
|---|---|
| C000721148 | betibeglogene autotemcel |
Not provided
Not provided
Not provided