Clinical trials
Congenital Disorders clinical trials
Below are recruiting congenital disorders clinical trials, each written for real people, not researchers. We’re tracking 55 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - NCT07845825RecruitingEarly Phase 1
Aprepitant for CLIFAHDD syndrome
This trial tests a drug called aprepitant to see if it can help people with CLIFAHDD, a rare genetic condition that affects movement and development. The study will check if the drug is safe and if it improves symptoms.
San Antonio, TexasAges 6 months+ - 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.
Los Angeles, CaliforniaAges Any age - NCT06349668RecruitingPhase 3
Comparing spinal morphine and IV lidocaine for robotic kidney surgery
This trial compares two ways to manage pain during robotic-assisted kidney or ureter surgery: a spinal injection of morphine or an intravenous infusion of lidocaine. The goal is to see which method provides better pain relief and recovery.
KalmarAges 18 years+ - NCT07587242RecruitingPhase 3
Testing a New Therapy for DMD with Exon 44 Mutations
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
Westmead, New South WalesAges 7–16 - NCT06092346Recruiting
Understanding rare purine and pyrimidine metabolism disorders
This study aims to learn more about rare disorders of purine and pyrimidine metabolism (DPPM), which can affect the brain, immune system, kidneys, and muscles. Researchers will study people with these disorders, their family members, and healthy volunteers to better understand the condition over time.
Bethesda, MarylandAges 1 month–100 years - NCT03775954Recruiting
Fetal heart testing in high-risk pregnancies after baby loss
This study checks unborn babies’ heart signals using special scans during pregnancy and then checks the baby’s heart shortly after birth. It may help researchers understand why certain high-risk pregnancies can lead to fetal demise and improve care.
Madison, WisconsinAges 18 years+
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Common questions
- Are there clinical trials for congenital disorders?
- Yes. Clin2 currently lists 55 recruiting congenital 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 congenital 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 congenital 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.