Clinical trials
Orphan Diseases clinical trials
Below are recruiting orphan diseases clinical trials, each written for real people, not researchers. We’re tracking 4 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06729554Recruiting
Education and support program for rare disease kids
This study tests a targeted educational and support program for children and teens with rare diseases. It helps families learn more about the condition and cope better with day-to-day challenges.
Graz, AustriaAges 5–20 - NCT06615206Recruiting
First CRISPR RNA therapy for MECP2 duplication syndrome
This trial tests a new CRISPR therapy that edits RNA to treat a rare genetic condition called MECP2 duplication syndrome (MDS). It is for boys aged 2 to 18 who have stable seizures and no other major health problems, with the goal of improving symptoms and safety.
Beijing, Beijing MunicipalityAges 2–18 - NCT02743845Recruiting
Find genetic answers for rare, possibly inherited conditions
This trial looks for gene changes that may explain rare conditions, even when the diagnosis is not fully understood. It may help families who have a rare condition or who are related to someone with one.
Boston, MassachusettsAges Any age - NCT06219421Recruiting
Using facial photos to understand genetic conditions
This study uses regular front and side facial photos to help doctors better understand facial features in people with genetic conditions or after surgery. It may help improve how we diagnose and care for these conditions.
ParisAges Any age
Hear when a new Orphan Diseases trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
Common questions
- Are there clinical trials for orphan diseases?
- Yes. Clin2 currently lists 4 recruiting orphan diseases 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 orphan diseases 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 orphan diseases 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.