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A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

NCT05146830 · Status: ENROLLING_BY_INVITATION · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2024-07-03

No results posted yet for this study

Summary

This is a multinational, long-term follow-up study to assess the long-term safety and durability of CTNS-RD-04 treatment in participants who received a single dose administration of lentiviral gene therapy. No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from the initial date of CTNS-RD-04 infusion.

Conditions

  • Cystinosis

Interventions

OTHER

Safety and Efficacy Assessments

Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.

Sponsors & Collaborators

  • California Institute for Regenerative Medicine (CIRM)

    collaborator OTHER

  • Cystinosis Research Foundation

    collaborator OTHER

  • Novartis Pharmaceuticals

    collaborator INDUSTRY

  • Stephanie Cherqui

    lead OTHER

Principal Investigators

  • Stephanie Cherqui, PhD · University of California, San Diego

Eligibility

Min Age
14 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-11-14
Primary Completion
2036-11-30
Completion
2036-11-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05146830 on ClinicalTrials.gov