Top Cities for Cystinosis Clinical Trials
Cystinosis clinical trials are recruiting across 3 cities. Here are the cities with the most active studies:
About Cystinosis
Cystinosis is a rare genetic disorder caused by accumulation of the amino acid cystine in cells, primarily damaging the kidneys and eyes. Without treatment, it leads to kidney failure in childhood. Treatment includes cysteamine therapy to reduce cystine levels.
Clinical trials are advancing new treatments for cystinosis. Currently, 1 studies are recruiting a combined 30 participants across the United States. Research is being conducted by 1 organizations including Novartis Pharmaceuticals.
2026 Cystinosis Research Landscape
As of March 2026, the cystinosis clinical trial landscape includes 1 actively recruiting studies across 3 cities in the United States. These studies are collectively seeking 30 participants, with an average enrollment target of 30 per study.
Research is being led by 1 organization, including Novartis Pharmaceuticals.
Geographically, cystinosis trials are most concentrated in San Diego, California (1 trial); Stanford, California (1 trial); Atlanta, Georgia (1 trial).
Featured Cystinosis Studies
Highlighted recruiting studies for cystinosis, selected by enrollment size and research scope.
DFT383 in Pediatric Participants With Nephropathic Cystinosis
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. This study includes an active arm (Cohort 1)...
Frequently Asked Questions About Cystinosis Clinical Trials
Are there cystinosis clinical trials near me?
Yes, there are 1 cystinosis clinical trials currently recruiting across 3+ cities in the United States, including San Diego, California; Stanford, California; Atlanta, Georgia. Browse the studies above to find one at a location convenient for you.
How do I join a cystinosis clinical trial?
To join a cystinosis clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are cystinosis clinical trials free?
Yes, participation in cystinosis clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of cystinosis treatments are being studied?
Current cystinosis clinical trials are testing a range of approaches. These include new drugs, combination therapies, medical devices, and other interventions sponsored by 1 research organizations.
Is it safe to participate in cystinosis clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated March 1, 2026 from ClinicalTrials.gov
About This Data
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated March 1, 2026 · Data from ClinicalTrials.gov