Study Summary
This trial will test a new treatment for cystinosis, a rare disease that affects the metabolism of the amino acid cystine.
Eligible Conditions
- Cystinosis
- Lysosomal Storage Diseases
Treatment Effectiveness
Effectiveness Progress
Study Objectives
4 Primary · 3 Secondary · Reporting Duration: Up to 24 months post transplant.
Month 24
Evaluation of efficacy - Change in Quality of Life
Evaluation of efficacy - Change in cystine levels
Therapeutic procedure
Evaluation of safety - Absence of Replication-Competent Lentivirus (RCL)
Evaluation of safety - Absence of Severe Adverse Events (SAEs) due to the investigational product
Evaluation of safety - Absence of genotoxicity
Evaluation of safety - Event-free survival
Trial Safety
Safety Progress
Trial Design
2 Treatment Groups
CTNS-RD-04 Gene Therapy
1 of 2
Gene Therapy with CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for...
1 of 2
Experimental Treatment
6 Total Participants · 2 Treatment Groups
Primary Treatment: CTNS-RD-04 · No Placebo Group · Phase 1 & 2
CTNS-RD-04 Gene Therapy
Genetic
Experimental Group · 1 Intervention: CTNS-RD-04 · Intervention Types: GeneticGene Therapy with CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for LentiBOOST)
Genetic
Experimental Group · 1 Intervention: CTNS-RD-04 or CTNS-RD-04-LB (where the suffix "-LB" stands for LentiBOOST) · Intervention Types: GeneticTrial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 24 months post transplant.
Who is running the clinical trial?
University of California, San DiegoLead Sponsor
1,027 Previous Clinical Trials
1,796,015 Total Patients Enrolled
3 Trials studying Cystinosis
420 Patients Enrolled for Cystinosis
Cystinosis Research FoundationOTHER
3 Previous Clinical Trials
20,770 Total Patients Enrolled
3 Trials studying Cystinosis
20,770 Patients Enrolled for Cystinosis
California Institute for Regenerative Medicine (CIRM)OTHER
59 Previous Clinical Trials
3,254 Total Patients Enrolled
Stephanie Cherqui, Ph.D.Principal Investigator - University of California, San Diego (UCSD)
University of California, San Diego
Eligibility Criteria
Age 18+ · All Participants · 10 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:You are a male or female subject ≥ 14 years of age.
You must be at least one year post kidney transplant status.
References
- Harrison, Frank, Brian A Yeagy, Celine J Rocca, Donald B Kohn, Daniel R Salomon, and Stephanie Cherqui. 2013. “Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis”. Molecular Therapy. Elsevier BV. doi:10.1038/mt.2012.214.
- Naphade S, Sharma J, Gaide Chevronnay HP, Shook MA, Yeagy BA, Rocca CJ, Ur SN, Lau AJ, Courtoy PJ, Cherqui S. Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes. Stem Cells. 2015 Jan;33(1):301-9. doi: 10.1002/stem.1835.
- Stephanie Cherqui 2019. "Stem Cell Gene Therapy for Cystinosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03897361.
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Conditions
Cancer Related
Treatments