NCT06910813 · Novartis Pharmaceuticals
DFT383 in Pediatric Participants With Nephropathic Cystinosis
(CYStem)
What this study is about
An where both patients and doctors know the treatment given, multi-center, phase I/II study to assess the safety, tolerability and effectiveness of DFT383 in pediatric participants with nephropathic cystinosis. The purpose of this clinical study is to assess safety, tolerability, and effectiveness of DFT383 in participants aged 2 to ≤ 5 years with nephropathic cystinosis.
View original scientific description
An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis. The purpose of this clinical study is to assess safety, tolerability, and efficacy of DFT383 in participants aged 2 to ≤ 5 years with nephropathic cystinosis. DFT383 is a cellular gene therapy.
Interventions
GENETIC
DFT383
DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy.
Primary outcome measures
Core Phase - Incidence of adverse events (Cohort 1)
Time frame: Up to 32 months
Number and proportion of participants with adverse events (AEs) and serious adverse events (SAEs)
Core Phase - Number of participants with hematological reconstitution (Cohort 1)
Time frame: 42 days post DFT infusion
Hematological reconstitution by Day 42 post-DFT383 infusion
Core Phase - Proportion of participants with reversal of renal Fanconi syndrome (RFS)
Time frame: Up to 32 months
Proportion of participants with reversal of renal Fanconi syndrome (RFS)
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Participants eligible for inclusion in this study must meet all the following criteria: 1. Informed consent in writing from parent(s) or legal guardian(s) must be provided 2. 2 to 5 years of age (including 5 years and 364 days old) at Screening 3. Weight-for-stature is ≥ the third percentile is ≥ 10 kg 4. Oral cysteamine therapy for at least 6 months 5. Historic clinical diagnosis of nephropathic cystinosis 6. Laboratory evidence of of renal fanconi syndrome (RFS) 7. Preserved kidney function (eGFR ≥ 90mL/min/1.73m2) 8. Received all age-appropriate vaccinations Key
Exclusion criteria
- for Cohort 1 and 0 1. A history of kidney transplantation 2. A prior or planned bone marrow or stem cell transplantation or prior treatment with gene therapy 3. History of malignancy 4. A severe or uncontrolled medical disorder 5. Major surgery within 90 days Additional Key exclusion criteria for Cohort 1 - The following exclusion criteria only apply to Cohort 1 only as they ar
Where
- San Diego, California
- Stanford, California
- Atlanta, Georgia
- Houston, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 29, 2026 · Source of record for eligibility and locations