Clinical trials
Batten Disease clinical trials
Below are recruiting batten disease clinical trials, each written for real people, not researchers. We’re tracking 9 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT04613089Recruiting
Study of Batten disease progress and clinical changes over time
This trial tracks how Batten disease (NCL) affects the body and how symptoms change over time. If you have a confirmed genetic (molecular) diagnosis—especially CLN2 with Brineura treatment—it may help researchers understand disease patterns and care needs.
HamburgAges Any age - NCT06203106Recruiting
NYSCF Scientific Discovery Biobank
This study collects blood, saliva, or skin samples from people with certain neurological conditions and from healthy volunteers to help researchers better understand and find treatments for these diseases.
New York, New YorkAges 4 weeks+ - NCT03333200Recruiting
Study of genetic brain diseases over time
This trial follows people with genetic neurodegenerative (brain-wasting) disorders over time to better understand how the condition changes. The goal is to learn patterns that could help future treatments or care plans.
Pittsburgh, PennsylvaniaAges Any age - NCT01873924Recruiting
Study of Batten disease symptoms and brain function
This study looks at how Batten disease affects the body and thinking/brain function. It may help researchers better understand the condition and how symptoms change over time.
Rochester, New YorkAges Any age - NCT03862274Enrolling by invitation
Study of development in children with CLN2 due to TPP1 changes
This study looks at how children’s development progresses in CLN2 disease caused by a specific enzyme problem (TPP1). It may help doctors better understand outcomes and guide future care for children with this diagnosis.
Columbus, OhioAges Any age - NCT05007990Recruiting
Interview study of caregiving networks over a person’s life
This study uses interviews and questionnaires to better understand who makes up a caregiver’s support network when caring for someone with a long-term health condition. Your answers may help researchers understand how caregiving changes over time and across different health situations.
Bethesda, MarylandAges 18–100 - NCT02435940Recruiting
Registry for inherited eye vision conditions
This study is a registry, meaning it collects information about people with inherited retinal (retina) diseases. It may help researchers learn how these conditions progress and connect you with future studies or treatments.
Columbia, MarylandAges Any age - NCT02254863RecruitingPhase 1
Stem cell transplant into the spinal fluid for rare brain diseases
This Phase 1 trial tests a transplant using special donated stem-cell–like cells placed into the spinal fluid to treat certain inherited brain diseases. It is mainly designed to check safety, and it may offer benefit by targeting brain and nerve damage.
Durham, North CarolinaAges 1 week–22 years - NCT03307304Recruiting
Studying different forms of childhood brain-wasting disease
This study looks at people with CLN3 and other related forms of NCL (a childhood brain-wasting illness) to better describe symptoms and disease changes over time. You may be followed with regular check-ins, medical records, and sometimes NIH visits or sample collection.
Bethesda, MarylandAges 1 week–100 years
Hear when a new Batten Disease trial opens
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Common questions
- Are there clinical trials for batten disease?
- Yes. Clin2 currently lists 9 recruiting batten disease studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a batten disease trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a batten disease trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.