Clinical trial
A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing
NCT03897361 is registered in ClinicalTrials.gov with University of California, San Diego as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing
- Lead sponsor
- University of California, San Diego
- Phase
- Phase 1/2
- Overall status
- Completed
- Start date
- July 8, 2019
- Completion date
- September 18, 2024
- Enrollment
- 7 (actual)
- Sites
- 1
Conditions
As the registry lists them; a condition with its own page is linked.