Clinical trials · Pennsylvania
Pelizaeus-Merzbacher Disease clinical trials in Pennsylvania
We’re tracking 4 recruiting pelizaeus-merzbacher disease studies with a site in Pennsylvania — including Phase 1 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT03047369Recruiting
Myelin brain disorder research biobank for samples and data
This study collects brain-related medical information and biological samples to help researchers better understand leukodystrophies and other white-matter brain disorders. It may help future diagnosis and treatments by building a valuable “biobank” of patient data and samples.
Philadelphia, PennsylvaniaAges Any age - 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 - NCT05659901Recruiting
Study tracks symptoms and test results in PLP1-confirmed PMD
This study follows children with Pelizaeus-Merzbacher disease (PMD) who have a specific genetic change (PLP1 duplication). It aims to learn which body measurements (“biomarkers”) and symptoms change over time, to better understand disease progression.
Philadelphia, PennsylvaniaAges 6 months–17 years - NCT06150716RecruitingPhase 1
Study of a new drug for Pelizaeus Merzbacher disease in boys
This trial is testing an experimental drug (ION356) given by a lumbar puncture to see if it is safe and how it works in boys with Pelizaeus Merzbacher disease (PMD). The drug is designed to target the genetic cause of PMD.
Philadelphia, PennsylvaniaAges 2–17
Pelizaeus-Merzbacher Disease trials by city in Pennsylvania
Where these studies are running in Pennsylvania
Institutions with a site for the recruiting pelizaeus-merzbacher disease studies listed above.
- Investigative Site · 2 studies
- Children's Hospital of Philadelphia
- University of Pennsylvania
- University of Pittsburgh Medical Center
- UPMC Children's Hospital of Pittsburgh
What taking part in a pelizaeus-merzbacher disease study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Pelizaeus-Merzbacher Disease trials in other states
Common questions
- Are there pelizaeus-merzbacher disease clinical trials in Pennsylvania?
- Yes. We're currently tracking 4 recruiting pelizaeus-merzbacher disease studies with a site in Pennsylvania, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a pelizaeus-merzbacher disease study in Pennsylvania?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Pennsylvania to take part?
- Not necessarily. These studies have a site in Pennsylvania, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a pelizaeus-merzbacher disease trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New pelizaeus-merzbacher disease studies open in Pennsylvania regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
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.