Trials / Completed
CompletedNCT02800070
Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease
Clinical Pilot Study of Autologous Stem Cell Transplantation of Cluster of Differentiation 34 Positive (CD34+) Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 5 (actual)
- Sponsor
- University Health Network, Toronto · Academic / Other
- Sex
- Male
- Age
- 18 Years – 50 Years
- Healthy volunteers
- Not accepted
Summary
This is a first-in-human study for the treatment of Fabry disease. Eligible patients will have an autologous stem cell transplantation using CD34+ cells that are transduced with the lentivirus vector containing the human alpha-gal A gene. The researchers of this study would like to see if the re-introduction of transduced cells will help increase the levels of alpha-gal A enzyme levels and to determine the safety and toxicity of autologous stem cell transplantation using CD34+ cells transduced with lentivirus vector containing the alpha-gal A gene. This study's objective is to determine the safety and toxicity of lentivirus alpha-gal A transduced CD34+ cells in adult males with Fabry disease.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Lentivirus Alpha-gal A transduced stem cells |
Timeline
- Start date
- 2016-07-01
- Primary completion
- 2024-04-01
- Completion
- 2024-04-01
- First posted
- 2016-06-15
- Last updated
- 2024-04-17
Locations
3 sites across 1 country: Canada
Source: ClinicalTrials.gov record NCT02800070. Inclusion in this directory is not an endorsement.