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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
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.
Details
| Lead sponsor | University Health Network, Toronto |
|---|---|
| Phase | Phase 1 |
| Status | COMPLETED |
| Enrolment | 5 |
| Start date | 2016-07 |
| Completion | 2024-04 |
Conditions
- Fabry Disease
Interventions
- Lentivirus Alpha-gal A transduced stem cells
Primary outcomes
- Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment — 5 years
Safety measurement will be based on all clinical and laboratory assessment post-baseline. The assessment will be the frequency of clinically notable abnormal vital signs and laboratory values, and the frequency of treatment-related adverse events.
Countries
Canada