- recruitingPHASE3
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
This study looks at a new drug, acoramidis, for people who have a faulty gene that can cause a condition called ATTR. It aims to see if the drug can stop or slow down the disease from developing in people who don't yet have symptoms.
United States · Argentina · Australia - authorisedTherapeutic confirmatory (Phase III)
A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients with Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)
This study is looking at a medicine called Acoramidis for people recently diagnosed with a heart condition called variant transthyretin amyloidosis. Doctors want to see how safe the medicine is and if it helps with heart-related problems over a longer period.
Germany · Portugal · Netherlands - recruiting
Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
The MaesTTRo study is looking at people living with a condition called transthyretin amyloidosis (ATTR). It will follow them over time to understand how the condition develops and how current treatments are working in real life. It aims to gather information from around the world.
United States · Canada · China - active not recruiting
Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran
This study looks at how new medicines, Patisiran or Vutrisiran, affect the hearts of people in the UK with a specific type of inherited amyloidosis (ATTRv) that also causes nerve problems. It aims to understand if these treatments help their hearts over two years.
France - ongoing, recruitingHuman Pharmacology (Phase I)- Bioequivalence Study
A Study to Establish the Bioequivalence of Free Acid Tafamidis 61 mg Capsule and Tablet, and Evaluate the Safety and Tolerability of the 61 mg Tablet in Healthy Adults Under Fed Conditions
This study is looking at a new tablet form of a medicine called tafamidis. It checks if the new tablet works in the same way as the current capsule. This helps make sure patients with a condition called transthyretin amyloidosis can have a new, easy-to-use option.
Belgium - ongoing, recruitingHuman Pharmacology (Phase I)- Bioequivalence Study
A study to establish the bioequivalence of free acid tafamidis 61 mg capsule and tablet, and evaluate the safety and tolerability of the 61 mg tablet in healthy adults
Researchers are comparing two forms of a tafamidis medicine (capsule and tablet) in healthy adults. This is to check if both forms deliver the medicine to the body in the same way. They will also look at how safe the tablet form is.
Belgium