Condition

Batten Disease clinical trials and research

• 7Clinical trials
• 2Countries

Newest Batten Disease clinical trials

All recruiting trials (7)

• Biobank for studying ALS and Alzheimer's diseases

  This study is collecting blood, skin samples, and health information from people to see how genetics might help us understand and find treatments for ALS and Alzheimer's disease.

  Recruiting New York Stem Cell Foundation Research Institute · New York, New York · NCT06203106

• Observational study on the natural history of Batten disease

  This study looks at how Batten disease affects patients over time by collecting information about their symptoms and health to help understand the disease better and support future treatments.

  Recruiting Universitätsklinikum Hamburg-Eppendorf · Hamburg · NCT04613089

• Long-term observation of rare genetic brain disorders

  This study is tracking how rare genetic brain disorders progress over time to better understand their effects on patients.

  Recruiting University of Pittsburgh · Pittsburgh, Pennsylvania · NCT03333200

• Investigating biomarkers for Juvenile Neuronal Ceroid Lipofuscinosis

  This study is trying to find specific markers in blood and other samples from people with Batten disease to help track the disease's progress and support future treatments.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT03307304

• Registry for inherited retinal degenerative diseases

  This study is creating a registry for people with rare inherited eye diseases to help track their health and experiences over time, making it easier to understand these conditions and support research.

  Recruiting Foundation Fighting Blindness · Columbia, Maryland · NCT02435940

• Using umbilical cord blood cells to treat inherited metabolic diseases

  This study is testing if a new treatment using special cells from umbilical cord blood can help people with inherited metabolic diseases and early signs of nerve damage feel better when combined with standard cord blood transplants.

  Recruiting Phase 1 Duke University · Durham, North Carolina · NCT02254863

• Investigating Batten disease and its effects

  This study is trying to understand how Batten disease affects movement, behavior, and daily life by collecting information from people with the condition over time.

  Recruiting University of Rochester · Rochester, New York · NCT01873924

Related topics

Quick answers

How many Batten Disease clinical trials are recruiting right now?

7, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 2 countries. The most sites are in United States (6 trials), Germany (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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