Ongoing Clinical Trials for Acquired ATTR Amyloidosis
This article provides information about 2 ongoing clinical trials investigating new treatments for acquired ATTR amyloidosis. These studies are exploring medications that may help prevent disease development or improve quality of life and exercise capacity in affected individuals.
Clinical trial locations
- Belgium
- Bulgaria
- Denmark
- France
- Germany
- Greece
- Ireland
- Italy
- Netherlands
- Portugal
- Spain
- Sweden
Study on Acoramidis for Preventing Transthyretin Amyloidosis in Young People with a Genetic Risk
This phase 3 clinical trial is investigating whether acoramidis (also known as AG10) can prevent the development of transthyretin amyloidosis in people who carry a genetic variant but do not yet have symptoms. This rare disease causes abnormal protein deposits to build up in organs and tissues, most commonly affecting the heart and nerves.
Main inclusion criteria:
- Adults between 18 and 75 years of age
- Confirmed presence of a TTR gene variant known to cause the condition
- Age should be no more than 10 years younger than the typical age when family members develop symptoms
- Willingness to use effective birth control during the study and for 30 days afterward
- Ability to provide informed consent
Main exclusion criteria:
- Already showing signs or symptoms of transthyretin amyloidosis
- Any clinical evidence of the disease at study entry
Focus of the trial: The study aims to determine whether acoramidis can effectively delay or prevent the onset of symptoms in people at genetic risk. Participants will be randomly assigned to receive either acoramidis tablets or a placebo in a double-blind design, meaning neither participants nor researchers will know who receives which treatment. The trial will follow participants for several years until November 2031, monitoring for any development of disease symptoms. The primary focus is comparing the time it takes for symptoms to appear in those taking acoramidis versus those taking placebo.
Investigational drug: Acoramidis is an oral medication that works as a transthyretin stabilizer. It helps keep the transthyretin protein in its normal form, preventing it from misfolding and creating harmful deposits in organs. This stabilizing action aims to prevent the disease from developing in people who carry the genetic variant.
Study on the Impact of Dapagliflozin on Quality of Life and Exercise Capacity in Patients with Transthyretin Cardiac Amyloidosis
This clinical trial is examining whether dapagliflozin, a medication commonly used for diabetes, can improve quality of life and exercise capacity in patients who already have ATTR cardiac amyloidosis. This form of the disease specifically affects the heart, where protein deposits accumulate in heart tissue and make the heart walls stiff, reducing the heart’s ability to pump blood effectively.
Main inclusion criteria:
- Adults between 18 and 90 years of age
- Confirmed diagnosis of ATTR cardiac amyloidosis through specific medical tests, such as heart tissue biopsy or special imaging
- History of heart failure, either with previous hospitalization or current symptoms like swelling, shortness of breath, or fluid in the lungs
- Stable doses of cardiovascular medications (except diuretics) for at least 2 weeks before study entry
- Ability to understand and sign informed consent
Main exclusion criteria:
- Lack of confirmed ATTR cardiac amyloidosis diagnosis
- Inability to safely participate in exercise tests
- Pregnancy or breastfeeding
- Other serious health conditions that might interfere with the study
- Inability to provide informed consent
Focus of the trial: Researchers want to understand how dapagliflozin affects patients’ ability to exercise and their overall well-being. The study will measure changes in how far patients can walk in six minutes, which is a standard way to assess exercise capacity. Additionally, researchers will evaluate improvements in heart-related symptoms and quality of life using specialized questionnaires. Safety will be closely monitored by recording any side effects, and adjustments to other heart medications may be made as needed. The study is expected to conclude in June 2025.
Investigational drug: Dapagliflozin is taken as a film-coated tablet and belongs to a class of medications called SGLT2 inhibitors. While it’s primarily known for lowering blood sugar by helping the kidneys remove glucose from the bloodstream, researchers believe it may also have beneficial effects on heart function. In this trial, dapagliflozin is being studied specifically for its potential to help patients with ATTR cardiac amyloidosis feel better and be more active in their daily lives.
Summary
Currently, there are 2 ongoing clinical trials for acquired ATTR amyloidosis available in Europe. These trials represent two distinct approaches to managing the disease: prevention in at-risk individuals and symptom management in those already affected.
The first trial, testing acoramidis, is widely available across 12 European countries including Italy, France, Denmark, Sweden, Netherlands, Portugal, Spain, Greece, Germany, Belgium, Bulgaria, and Ireland. This prevention-focused study targets individuals who carry the genetic variant but have not yet developed symptoms, representing an important shift toward early intervention.
The second trial, evaluating dapagliflozin, is currently available only in Italy and focuses on improving quality of life and exercise capacity in patients who already have cardiac involvement. This represents a repurposing approach, testing a medication already known for diabetes treatment in a new context.
Both trials reflect ongoing efforts to expand treatment options for this rare condition, with one focusing on disease prevention and the other on managing existing symptoms. Patients interested in participating should discuss eligibility with their healthcare providers.
