Congenital hypothyroidism

This study offers expanded access to an experimental stem cell treatment for up to 7 adults with Multiple System Atrophy (MSA), a rare and serious brain disorder. Participants will receive 12 intravenous infusions and 6 spinal injections of donor stem cells over 44 weeks. The goa…

This study looks at three forms of levothyroxine (a thyroid hormone medicine) given to babies diagnosed with congenital hypothyroidism. Researchers will compare a new oral solution to standard liquid drops and tablets, checking how well they control hormone levels and affect deve…

This early-phase trial tests a drug called Aleeto, a nerve repair protein made from stem cells, in 20 people with Multiple System Atrophy (MSA), a rare brain disease. The study checks if Aleeto is safe and might help with symptoms. Participants receive the drug via injection into…

This study tests a computer model called OptiThyDose that helps doctors find the best dose of thyroid medication for children with congenital hypothyroidism or Graves' disease. The model uses past lab results to keep thyroid hormone levels in a healthy range. About 150 children w…

This study is testing a new blood test to help doctors diagnose Parkinson's disease and similar brain disorders more accurately and earlier. Researchers will use a technique called RT-QuIC to detect misfolded proteins in blood samples from 458 participants, including healthy peop…

This study is looking at the genes of 350 children born with permanent congenital hypothyroidism, even though their thyroid gland is in the right place. Researchers will use advanced genetic sequencing to find DNA changes that might explain the condition. The goal is to better un…

This study looks at how babies aged 6 to 18 months with congenital hypothyroidism (a thyroid condition present at birth) develop their motor skills, sensory processing, and neurological health. Researchers will assess 40 infants who have been on treatment since birth, using stand…

This study looks at whether measuring a different thyroid hormone (fT3) or the ratio of two hormones (fT3/fT4) can give a clearer picture of thyroid health in people taking levothyroxine. It includes 500 adults with various types of hypothyroidism who are on a stable dose of the …