Phenylketonuria (PKU) and Hyperphenylalaninemia research hub
8 indexed studies · 6 currently recruiting
- not yet recruitingNA
Acceptability and Tolerance Market Research of a Slow Release, Phenylalanine-free Protein Substitute for the Dietary Management of Phenylketonuria
This study looks at a new type of protein supplement for children and adults with PKU. It's a slow-release tablet designed to be easier to take. Researchers want to see if people find it acceptable and if they tolerate it well, helping improve choices for managing PKU.
United Kingdom - active not recruitingPHASE3
A Study of Donanemab (LY3002813) in Participants With Early Symptomatic Alzheimer's Disease (TRAILBLAZER-ALZ 5)
This study is testing a new medication called donanemab for people in the early stages of Alzheimer's disease. Researchers want to see if it's safe and helps improve or slow memory and thinking problems. It's a key step in understanding potential new treatments for this condition.
Argentina · Australia · China - recruitingPHASE3
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
This study looks at a new medicine called AOC 1044 (or delpacibart zotadirsen) for boys with Duchenne Muscular Dystrophy (DMD) who have a specific genetic change (exon 44 skipping). It aims to see if the medicine is safe and helps improve the condition, compared to a dummy medicine.
Belgium · France · Germany - recruitingNA
Prospective Cohort Study of Neurogenetic Diseases
This study looks at rare, inherited nerve conditions to understand them better. Researchers want to create a central point for information and samples to help develop new treatments. It involves regular check-ups and sample collection over three years for those with an identified condition.
France - recruitingNA
Slowing Cognitive Decline in Alpha-synucleinopathies by Enhancing Physical Activity
This study looks at how increasing physical activity might help slow down memory and thinking problems in people with a condition called REM sleep behaviour disorder (iRBD). This condition can sometimes lead to Parkinson's disease. Participants will use a smartphone app to boost their activity levels.
Germany - recruitingNA
Treating Depression on a Day-to-day Basis: Development of a Tool for Physicians Based on a Smartphone Application
This study is checking if a smartphone app can help doctors better understand and treat depression. It aims to improve how doctors provide care by looking at daily symptoms and how well people take their medication, using a mobile phone tool to gather information.
France - recruitingPHASE4
Cognitive Outcomes After Dexmedetomidine Sedation in Cardiac Surgery Patients
This study looks at whether a medicine called Dexmedetomidine can help prevent memory problems and confusion after heart surgery. Researchers want to see if it improves recovery and reduces how long people stay in hospital.
Canada - active not recruitingPHASE3
A Study of Different Donanemab (LY3002813) Dosing Regimens in Adults With Early Alzheimer's Disease (TRAILBLAZER-ALZ 6)
This study is looking at different ways of giving a medicine called donanemab for early Alzheimer's disease. Researchers want to see how different doses affect side effects and which patients might be more likely to experience them. It's for adults aged 60-85.
United States · United Kingdom