This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Safety and tolerability: Total number of adverse events (AEs) per participant (pre- and post-dose to end of study)
Timeframe: From the first pre-dose safety visit to the end of the treatment period (e.g., 24 months post-dose) or the participant's last safety-assessment visit, whichever occurs later.
Safety and tolerability: Aggregate number of adverse events (AEs) by National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) severity (pre-dose and post-dose to end of study)
Timeframe: From the first pre-dose safety visit to the end of the treatment period (e.g., 24 months post-dose) or the participant's last safety-assessment visit, whichever occurs later.
Safety and tolerability: Aggregate number of adverse events (AEs) by relationship to study treatment (pre-dose and post-dose to end of study)
Timeframe: From the first pre-dose safety visit to the end of the treatment period (e.g., 24 months post-dose) or the participant's last safety-assessment visit, whichever occurs later.
Safety and tolerability: Clinical laboratory values for white blood cell, absolute monocyte, and platelet counts at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.
Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion
Safety and tolerability: Systolic and diastolic blood pressure at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.
Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion
Safety and tolerability: Heart rate at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.
Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion
Safety and tolerability: Body temperature at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.
Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion
Safety and tolerability: Respiratory rate at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.
Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion
Safety and tolerability: Electrocardiogram (ECG) ventricular rates at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.
Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion
Safety and tolerability: Electrocardiogram (ECG) intervals including PR interval, QRS interval, QT interval and QTc Bazett at baseline, 12 month and 24 month post-infusion study visits reported at individual participant level.
Timeframe: Baseline, 12 months post-infusion, 24 months post-infusion
Safety: Number of dosed participants showing evidence (positive or negative) of genotoxicity as determined by polyclonal expansion and insertional mutagenesis.
Timeframe: Up to 24 months post transplant.