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

Cystinosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05146830
• Other study ID #: CTNS-RD-04-LTFU01
• Status: Enrolling by invitation
• Start date: November 14, 2022
• Est. completion date: November 30, 2036

Study information

• Verified date: June 2024
• Source: University of California, San Diego
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 50
• Est. completion date: November 30, 2036
• Est. primary completion date: November 30, 2036
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years to 50 Years

Eligibility

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 Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.

Related Conditions & MeSH terms

• Cystinosis

Intervention

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

Locations

Country	Name	City	State
United States	University of California San Diego	La Jolla	California

Sponsors (1)

Lead Sponsor	Collaborator
Stephanie Cherqui

Country where clinical trial is conducted

United States, 

References & Publications (2)

Harrison F, Yeagy BA, Rocca CJ, Kohn DB, Salomon DR, Cherqui S. Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis. Mol Ther. 2013 Feb;21(2):433-44. doi: 10.1038/mt.2012.214. Epub 2012 Oct 23. — View Citation

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- — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of clinically significant Adverse Events (AEs) and Serious Adverse Events (SAEs)		Baseline to Year 15 post gene therapy
Primary	Number of participants with clinically relevant abnormalities, as assessed by vital sign (heart rate, pulse rate, and temperature)		Baseline to Year 15 post gene therapy
Primary	Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests (chemistry and hematology)		Baseline to Year 15 post gene therapy
Primary	Number of participants with clinically relevant abnormalities, as assessed by by electrocardiograms (ECGs) (rate, rhythm, intervals)		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in Corneal cystine crystal score (CCCS) as assessed by in vivo confocal microscopy (IVCM)		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in Renal glomerular and tubular functions measured by glomerular filtration rate (GFR)		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in vision function as assessed by ophthalmology exams		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in grip strength measured by dynamometry		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in respiratory function measured by spirometry		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in bone density assessed by dual-energy X-ray absorptiometry (DEXA)		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in muscle mass assessed by dual-energy X-ray absorptiometry (DEXA)		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in Endocrine function measured by fasting glucose, thyroid function, and gonadotropin levels		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in neurological function assessed by neurological exam (mental status, coordination, sensory, reflexes, and visual motor integration)		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in psychometric function assessed by neurological exam (memory, oromotor function, intelligence quotient (IQ))		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in pill/injection count related to cystinosis treatment		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in Cystinosin (CTNS) as assessed by quantitative Polymerase Chain Reaction (qPCR)		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in mean Vector Copy Number (VCN) as assessed by quantitative Polymerase Chain Reaction (qPCR)		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in Cystine levels in leukocytes measured by mass spectrometry		Baseline to Year 15 post gene therapy
Secondary	Change from baseline in ovarian reserve and menstrual cycle as assessed by anti-Müllerian hormone (AMH) and gynecology exams		Baseline to Year 15 post gene therapy
Secondary	Change in male reproductive potential as assessed by urology exams (sperm count, motility, and morphology)		Baseline to Year 15 post gene therapy
Secondary	Absence of Replication Competent Lentivirus (RCL) as assessed by Elisa assay		Baseline to Year 15 post gene therapy