Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Batten Disease Clinical Trials
Batten Disease clinical trials are recruiting across 12 cities. Here are the cities with the most active studies:
Batten Disease Trial Phases
9 research organizations are sponsoring batten disease trials.
About Batten Disease
Batten disease refers to a group of rare, inherited neurological disorders (neuronal ceroid lipofuscinoses) that cause progressive loss of vision, cognition, and motor skills. They primarily affect children. Gene therapy and enzyme replacement therapy are being studied as potential treatments.
Clinical trials are advancing new treatments for batten disease. Currently, 9 studies are recruiting a combined 3,130 participants across the United States. Research is being conducted by 9 organizations including Stanford University, University of Rochester, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and 6 others.
2026 Batten Disease Research Landscape
As of July 2026, the batten disease clinical trial landscape includes 9 actively recruiting studies across 12 cities in the United States. These studies are collectively seeking 3,130 participants, with an average enrollment target of 348 per study.
The research spans multiple phases of development: 1 study is in PHASE1, 1 study is in PHASE4, 1 study is in NA, 1 study is in EARLY_PHASE1. Early-phase studies suggest new therapeutic approaches are being explored for batten disease.
Research is being led by 9 different organizations, including Stanford University, University of Rochester, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Novartis Pharmaceuticals, NYU Langone Health, and 4 others.
Geographically, batten disease trials are most concentrated in Stanford, California (1 trial); Rochester, New York (1 trial); Bethesda, Maryland (1 trial); Lenexa, Kansas (1 trial); San Antonio, Texas (1 trial) and 7 other cities.
Featured Batten Disease Studies
Highlighted recruiting studies for batten disease, selected by enrollment size and research scope.
Novel Serum Markers for Monitoring Response to Anti-Cancer Therapy
The purpose of this study is to measure the levels of serum proteins and other biomarkers in cancer patients and in patients suspected of having cancer. We believe that some of these markers may be useful for confirming the diagnosis or for selecting patients for specific types of cancer therapies. These markers may also help to predict response to therapy, relapse after therapy, and survival afte...
Clinical and Neuropsychological Investigations in Batten Disease
This study aims to assess the natural history of Batten disease (Neuronal Ceroid Lipofuscinosis) by obtaining information about the motor, behavioral, and functional capabilities of individuals with Batten disease. This study will also refine and validate the Unified Batten Disease Rating Scale (UBDRS) as a clinical rating instrument for Batten disease.
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
Background: CLN3, or Batten disease, is a genetic disorder. This deadly disease leads to decline of brain and nervous system functions. Symptoms of CLN3 typically occur between 4 and 7 years of age. They include changes in how a person sees, thinks, and moves. CLN3 can also cause seizures. No effective treatments for the disease are yet known. There is limited testing of potential therapies. Rese...
Frequently Asked Questions About Batten Disease Clinical Trials
Are there batten disease clinical trials near me?
Yes, there are 9 batten disease clinical trials currently recruiting across 12+ cities in the United States, including Stanford, California; Rochester, New York; Bethesda, Maryland. Browse the studies above to find one at a location convenient for you.
How do I join a batten disease clinical trial?
To join a batten disease 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 batten disease clinical trials free?
Yes, participation in batten disease 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 batten disease treatments are being studied?
Current batten disease clinical trials are testing a range of approaches across PHASE1 (1 study), PHASE4 (1 study), NA (1 study), EARLY_PHASE1 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 9 research organizations.
Is it safe to participate in batten disease 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 July 16, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
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 July 16, 2026 · Data from ClinicalTrials.gov