Clinical trials
Rare Disorders clinical trials
Below are recruiting rare disorders clinical trials, each written for real people, not researchers. We’re tracking 38 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06368726Enrolling by invitationPhase 1
Brain stimulation for kids with autism and genetic changes
This trial tests a gentle brain stimulation treatment called tDCS for children with autism or related conditions who also have certain genetic changes. It may help improve symptoms if your child meets the specific health and birth history requirements.
Albuquerque, New MexicoAges 6–11 - NCT04654000Recruiting
Rheopheresis to treat painful skin damage in calciphylaxis
This trial studies whether a treatment called rheopheresis, added to standard care, can help people with calciphylaxis whose skin wounds are getting worse. It may be considered when ulcers or dead (necrotic) skin appear despite conventional treatment.
AngersAges 18 years+ - NCT06478121Recruiting
Understanding how genes affect beta cells
This study looks at how specific genetic changes affect beta cells, which make insulin. It may help people with rare genetic forms of diabetes or other beta cell disorders, and also needs healthy volunteers for comparison.
Exeter, DevonAges 6–99 - NCT06839469Recruiting
Walking study for rare muscle and nerve conditions
This study looks at how children and teens with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk, by having them do walking tests while wearing sensors. The goal is to find better ways to track how these diseases progress and how treatments work over time, without needing blood tests or scans.
Palo Alto, CaliforniaAges 5 years+ - NCT04604626Recruiting
Study rare obesity linked to eating and brain conditions
This study aims to improve diagnosis and treatment planning for people with severe obesity—especially when it may be related to a genetic cause or a brain condition affecting appetite. You may be invited if your case includes clues from your history or symptoms, and you can consent to genetic testing discussions as part of usual care.
La DefenseAges Any age - NCT04880356Recruiting
Study of very rare inherited brain diseases over time
This study follows adults over time who have a very rare inherited brain disease, or whose illness seems like it may run in families. The goal is to better understand these conditions and how they progress.
Milan, MilanoAges 18 years+ - NCT07008612Recruiting
Study of MYT1L syndrome in children and adults
This study looks at MYT1L syndrome, a rare genetic condition that affects brain development. It aims to understand the disorder better by studying people with the syndrome and a comparison group.
RouenAges 6 years+ - NCT06491615Recruiting
eyeGENE study: genetics of rare eye diseases
This study collects DNA samples and medical information from people with rare inherited eye diseases and their close relatives. The goal is to learn more about the genetic causes of these conditions and improve diagnosis and care.
Bethesda, MarylandAges 1 day–120 years - NCT03416114Recruiting
Registry for people with rare primary headache types
This trial is a registry that collects information about rare types of primary headaches in specialized Italian headache clinics. It may help researchers better understand these conditions and improve future care.
FlorenceAges Any age - NCT05544266Recruiting
Study rare diabetes patterns to better guide diagnosis
This study looks at people who may have a “non-typical” form of diabetes, including rare genetic causes or unusual symptoms. It may help your care team understand what type of diabetes you have and whether specific genetic testing could explain it.
Aurora, ColoradoAges Any age - NCT06399952Recruiting
Natural history study for Baker Gordon syndrome
This study follows people with Baker Gordon syndrome over time to better understand the condition. Participants and their caregivers share medical records and complete tests and questionnaires.
Columbia, MissouriAges birth–99 years - NCT06938542Enrolling by invitation
Palliative care needs for kids with rare diseases
This study asks children with rare diseases and their families about what kind of palliative care (comfort and support) they need. It aims to understand how to better help families through their child's illness.
Washington D.C., District of ColumbiaAges 1–99 - NCT04194619Recruiting
Study of pregnancy safety in women with rare blood-vessel diseases
This study looks at pregnancy and the early period after birth in women with rare diseases that affect blood vessels. It may help doctors understand risks and improve care for future pregnancies.
AngersAges 18–45 - NCT06573723Recruiting
Rare disease registry at Hospital Italiano
This study collects information from patients with certain rare diseases to better understand them. If you have one of these conditions and receive care at Hospital Italiano de Buenos Aires, you may be able to join.
Buenos Aires, Buenos AiresAges Any age
Hear when a new Rare Disorders trial opens
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Common questions
- Are there clinical trials for rare disorders?
- Yes. Clin2 currently lists 38 recruiting rare disorders 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 rare disorders 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 rare disorders 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.