A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
Launched by STEPHANIE CHERQUI · Nov 23, 2021
Trial Information
Current as of June 26, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
Participants enrolled in a study where the individual received CTNS-RD-04 will be offered participation in the CTNS-RD-04-LTF01 study. The Baseline visit for the CTNS-RD-04-LTF01 study will likely coincide with the final visit in the parent study. Participants confirmed eligible for the CTNS-RD-04-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for up to 11 years until a total of 15 years have elapsed during which time continued safety, engraftment, and efficacy of CTNS-RD-04 treatment will be assessed.
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • • Participant must have received CTNS-RD-04 in a preceding study
- Exclusion Criteria:
- • • Participant is currently enrolled in an CTNS-RD-04 treatment study. Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.
About Stephanie Cherqui
Stephanie Cherqui is a dedicated clinical trial sponsor with a strong commitment to advancing medical research and improving patient outcomes. With extensive experience in the field, she focuses on innovative therapies and groundbreaking treatments that address unmet medical needs. Her leadership emphasizes collaboration among multidisciplinary teams, rigorous adherence to regulatory standards, and a patient-centered approach. Through her efforts, Stephanie aims to facilitate the development of safe and effective interventions, fostering a culture of scientific excellence and integrity within the clinical research community.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
La Jolla, California, United States
Patients applied
Trial Officials
Stephanie Cherqui, PhD
Principal Investigator
University of California, San Diego
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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