Study on Acoramidis for Preventing Transthyretin Amyloidosis in Young People with a Genetic Risk

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What is this study about?

The study focuses on a condition called Transthyretin Amyloidosis, which is a rare disease that can affect the heart and nerves. The trial is investigating a treatment called Acoramidis (AG10), which is taken as a tablet. This treatment is being compared to a placebo to see if it can prevent the disease in people who carry a specific genetic variant but do not yet show symptoms.

The purpose of the study is to determine if Acoramidis can effectively prevent the development of Transthyretin Amyloidosis in individuals who are at risk but currently asymptomatic. Participants will be randomly assigned to receive either the treatment or a placebo. The study will monitor participants over time to see if they develop symptoms of the disease.

The trial is designed to be a long-term study, with participants being followed for several years. The main focus will be on the time it takes for the disease to develop in those taking the treatment compared to those taking the placebo. The study will also look at other health outcomes and any side effects that may occur during the trial period.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, genetic status, and other criteria.

Participants must have a specific genetic variant related to transthyretin amyloidosis and be within the age range of 18 to 75 years.

2 randomization

Participants are randomly assigned to receive either the acoramidis tablet or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

3 medication administration

Participants take the assigned tablet orally. The dosage and frequency are determined by the study protocol and are consistent throughout the trial.

The duration of medication administration continues until the study’s estimated end date in November 2031, unless otherwise specified by the study protocol.

4 regular monitoring

Participants undergo regular health assessments to monitor for any signs of transthyretin amyloidosis or other health changes.

These assessments include physical examinations, vital sign checks, and laboratory tests to ensure participant safety and gather data on the medication’s effects.

5 end of study evaluation

At the conclusion of the study, a final evaluation is conducted to assess the participant’s health and any developments related to transthyretin amyloidosis.

The primary focus is on the time to development of any symptoms or conditions related to the disease.

Who Can Join the Study?

Participants must be willing and able to give a signed informed consent for study procedures. This means they agree to take part in the study after understanding what it involves.
Participants must be between 18 and 75 years old when signing the informed consent.
Participants must have a specific change in their DNA, called a TTR gene variant, which is known to cause the condition. This must be confirmed by a special test before joining the study.
Participants’ age should be no more than 10 years younger than the age when their family members typically show symptoms of the condition, known as PADO. For example, if family members usually show symptoms at 50 years old, participants should be at least 40 years old.
Participants must agree to use highly effective birth control methods during the study and for 30 days after the last dose of the study drug. This applies to both men and women who can have children.

Who Cannot Join the Study?

Individuals who already show signs of Transthyretin Amyloidosis cannot participate. This is a condition where a protein called transthyretin builds up in the body and can cause health problems.
Participants must not have any clinical evidence of the disease at the start of the study. This means they should not have any symptoms or medical findings that suggest they have the condition.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Bellvitge University Hospital	L'hospitalet De Llobregat	Spain
Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz	Mainz	Germany
Hospital Universitario De Salamanca	Salamanca	Spain
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
University Hospital Maastricht	Maastricht	The Netherlands
Oncopole Claudius Regaud	Toulouse	France
Katholieke Universiteit te Leuven	Leuven	Belgium
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal

Other Sites

Site Name	City	Country
Azienda Ospedaliero-Universitaria Sant Andre	Rome	Italy
Fondazione Toscana Gabriele Monasterio	Pisa	Italy
Region Vaesterbotten	Umea	Sweden
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Pellegrin Hospital	Bordeaux	France
Hippokration Hospital	Athens	Greece
Diagnostic-consultative center “Aleksandrovska” EOOD	Sofia	Bulgaria
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Alexandra Hospital	Athens	Greece
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Hospital Son Llatzer	Palma	Spain
Region Midtjylland	Aarhus	Denmark
Centre Hospitalier Universitaire De Rennes	Rennes	France
Centro Hospitalar Universitario De Santo Antonio E.P.E.	Porto	Portugal
A.O.U. Policlinico G. Martino Di Messina	Messina	Italy
CHU De Martinique	Fort De France	France
Bicetre Hospital	Le Kremlin-Bicetre	France
Uwcqaegxgfnf Msqvhul Cvbmogp Gxtfxmaax	Groningen	The Netherlands
Hitxjec Hknfe Mwdtpt &vmsihf 1 rmy Gweemie Edwnsn	Creteil	France
Ezbxvss Ujeemvamqaiz Mndcdjp Cqwupec Rgbqsstcp (ewmsbdg Mrb	Rotterdam	The Netherlands
Aeaxsky Uprck Sunqiulet Ljwavx Dl Bhpuljn	Bologna	Italy
Uewpymhdfg Dqxck Shqry Dk Rcri Ld Svdxdxez	Rome	Italy
Hmcwksuo Uqvzwxartrrix da A Cwirsv	A Coruna Galicia	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	31.01.2025
Bulgaria	Not recruiting	31.01.2025
Denmark	Recruiting	31.01.2025
France	Recruiting	31.01.2025
Germany	Recruiting	31.01.2025
Greece	Recruiting	31.01.2025
Ireland	Recruiting	31.01.2025
Italy	Recruiting	31.01.2025
Portugal	Recruiting	31.01.2025
Spain	Recruiting	31.01.2025
Sweden	Recruiting	31.01.2025
The Netherlands	Recruiting	31.01.2025

Trial locations

Acoramidis is a medication being studied for its ability to prevent a condition called transthyretin amyloidosis (ATTR) in people who carry a specific genetic variant. This condition can lead to problems with the heart or nerves. The trial is focused on individuals who have the genetic variant but do not yet show any symptoms of the disease. The goal is to see if acoramidis can stop or delay the onset of symptoms related to this condition.

Investigated diseases:

Acquired ATTR amyloidosis

Transthyretin Amyloidosis – This is a rare disease where abnormal protein deposits, known as amyloid, accumulate in the body’s tissues and organs. It is caused by mutations in the transthyretin (TTR) gene, leading to the production of unstable proteins that misfold and form amyloid fibrils. These deposits can affect various organs, most commonly the heart and nerves, leading to conditions such as cardiomyopathy and peripheral neuropathy. The disease progresses as more amyloid builds up, causing organ dysfunction and related symptoms. It can manifest in different forms, including hereditary and wild-type, with varying symptoms depending on the organs involved. Early stages may be asymptomatic, but as the disease advances, it can lead to significant health issues.

Trial ID:

2024-513547-82-00

Protocol code:

AG10-501

NCT ID:

NCT06563895

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Acoramidis for Preventing Transthyretin Amyloidosis in Young People with a Genetic Risk

What is this study about?

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