CTNS-RD-04 for Cystinosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Cystinosis+1 MoreCTNS-RD-04 - Genetic
Eligibility
18+
All Sexes
What conditions do you have?
Select

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

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

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: Genetic
Gene 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: Genetic

Trial 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.