Study on the Effectiveness and Safety of ALXN2220 for Adults with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

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What is this study about?

This clinical trial is focused on studying a condition known as Transthyretin Amyloid Cardiomyopathy (ATTR-CM). ATTR-CM is a heart disease caused by the buildup of abnormal proteins called amyloids in the heart tissue, which can lead to heart failure. The study is testing a new treatment called ALXN2220, which is a type of medication known as a monoclonal antibody. Monoclonal antibodies are proteins made in a lab that can bind to specific substances in the body, in this case, the amyloid proteins, to help remove them.

The purpose of this study is to evaluate the effectiveness and safety of ALXN2220 in adults with ATTR-CM. Participants in the study will receive either the ALXN2220 treatment or a placebo, which is a substance with no active medication. The study will be conducted over a period of time, during which participants will receive regular injections of the treatment or placebo. Throughout the study, participants will be monitored for any changes in their heart condition and overall health.

The study aims to determine if ALXN2220 can help reduce the symptoms and progression of ATTR-CM by comparing the health outcomes of those receiving the treatment to those receiving the placebo. Participants will undergo various health assessments, including heart function tests and physical examinations, to track their progress. The study will provide valuable information on whether ALXN2220 is a safe and effective treatment option for people with ATTR-CM.

1 randomization

Upon joining the study, participants are randomly assigned to receive either the ALXN2220 medication or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

2 treatment administration

Participants receive the assigned treatment as a solution for infusion. The specific dosage and frequency are determined by the study protocol and are administered under medical supervision.

3 blinded treatment period

During this period, the effectiveness and safety of ALXN2220 are evaluated. Participants continue to receive their assigned treatment and undergo regular assessments to monitor their health and any potential side effects.

4 primary endpoint evaluation

The main goal is to assess the difference in the occurrence of cardiovascular events and overall health status between the ALXN2220 and placebo groups. This includes monitoring for any adverse events and changes in health indicators.

5 secondary endpoint evaluation

Additional outcomes are measured, such as changes in physical function and quality of life over a 24-month period, as well as the incidence of any treatment-related adverse effects.

6 study completion

The study is expected to conclude by October 31, 2028. Participants will have contributed valuable data to assess the potential benefits and risks of ALXN2220 for treating transthyretin amyloid cardiomyopathy.

Who Can Join the Study?

  • Must be a male or female aged between 18 and 90 years old.
  • If using a treatment for ATTR amyloidosis, the dose must be stable for at least 90 days before the study starts.
  • Participants must use contraception according to local rules. Women must not be pregnant or breastfeeding and should use effective birth control if they can have children. Men must agree to use contraception and not donate sperm.
  • Must have a confirmed diagnosis of ATTR-CM (a type of heart condition) with specific tests showing heart involvement.
  • The thickness of the heart’s septal wall must be 12 mm or more, as measured by an ultrasound of the heart.
  • Must have a NT-proBNP level greater than 2000 pg/mL, which is a blood test that helps assess heart function.
  • Must have been treated with a loop diuretic (a type of medication that helps remove excess fluid) for at least 30 days before the study.
  • Must have a history of heart failure, shown by events like hospitalization for heart failure, urgent visits for heart failure, or episodes of fluid overload.
  • Must be classified as NYHA Class II-IV, which describes the severity of heart failure symptoms.
  • Must have a life expectancy of at least 6 months, as judged by the study doctor.

Who Cannot Join the Study?

  • Patients who are not adults cannot participate.
  • Patients who do not have Transthyretin Amyloid Cardiomyopathy (ATTR-CM) cannot participate. ATTR-CM is a condition where a protein called transthyretin builds up in the heart, affecting its function.
  • Patients who are part of a vulnerable population, such as those who cannot make decisions for themselves, cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medizinische Universitaet Innsbruck Innsbruck Austria
Azienda Ospedaliero Universitaria Careggi Florence Italy
Oslo Universitetssykehus HF Oslo Norway
Medizinische Hochschule Hannover Hanover Germany
Bellvitge University Hospital L'hospitalet De Llobregat Spain
Medical University Of Vienna Vienna Austria
Hospital Universitario De Salamanca Salamanca Spain
Azienda Ospedaliera Universitaria Federico II Di Napoli Naples Italy
Universitaetsklinikum Heidelberg AöR Heidelberg Germany
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Medical University Of Graz Graz Austria
University Hospital Maastricht Maastricht The Netherlands
Universitaet Leipzig Leipzig Germany
Oncopole Claudius Regaud Toulouse France
Hospital Universitario De Navarra Pamplona Spain
Université Libre de Bruxelles – Hôpital Erasme Brussels Belgium

Other Sites

Site Name City Country Status
Azienda Sanitaria Universitaria Giuliano Isontina Trieste Italy
Azienda Ospedaliero-Universitaria Sant Andre Rome Italy
Fondazione Toscana Gabriele Monasterio Pisa Italy
Centre Hospitalier Universitaire De Poitiers Poitiers France
Region Vaesterbotten Umea Sweden
Universitaetsklinikum Schleswig-Holstein AöR Kiel Germany
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Klinikum Wels-Grieskirchen GmbH Wels Austria
St. Olavs Hospital HF Trondheim Norway
Hospital Universitario De Jaen Jaen Spain
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego Warsaw Poland
HOPA MVZ GmbH Hamburg Germany
Azienda Ospedaliera di Padova Padua Italy
St James’s Hospital Dublin Ireland
Hospital Clinico Universitario De Valencia Valencia Spain
Odense University Hospital Odense Denmark
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Region Skane Skanes Universitetssjukhus Lund Sweden
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Alexandra Hospital Athens Greece
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Karolinska University Hospital Solna Sweden
Az St-Jan Brugge-Oostende A.V. Brugge Belgium
Ospedale San Giovanni Bosco Turin Italy
University Clinical Hospital Virgen De La Arrixaca Murcia Spain
Centre Hospitalier Universitaire De Montpellier Montpellier France
Universidade De Santiago De Compostela Santiago De Compostela Spain
Hospital Son Llatzer Palma Spain
Fakultni Nemocnice U Sv Anny V Brne Brno-Stred Czechia
Justus-Liebig-Universitaet Giessen Giessen Germany
Centre Hospitalier Universitaire De Nantes Nantes France
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Uniwersytecki Szpital Kliniczny W Poznaniu Poznan Poland
Centre Hospitalier Universitaire De Rennes Rennes France
Ziekenhuis Oost Limburg Genk Belgium
Rigshospitalet Copenhagen Denmark
A.O.U. Policlinico G. Martino Di Messina Messina Italy
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Universita’ Degli Studi Di Ferrara Ferrara Italy
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Stadt Wien Wiener Gesundheitsverbund Vienna Austria
Universitätsklinikum des Saarlandes – Homburg/Saar, Klinik für Urologie und Kinderurologie Homburg Germany
Hospital Universitario Virgen De La Victoria Malaga Spain
Ospedale Giovan Battista Morgagni-Luigi Pierantoni Di Forlì Forli' Italy
Uvlucrawdysozmzrdyvnn Euhnk Aoy Essen Germany
Isyiwpwoq Fhb Cygdfore Arz Ebxytsmycejh Mmdxzifj Prague Czechia
Ultuyviuhqak Mqmrzhk Cipzmdc Gdtcqxkce Groningen The Netherlands
Urxzzcwnga Hpmrmlsi Crdtesv Cologne Germany
Anocqgynik Pavidwsi Hntyjkgl Dh Msxalqvnq Marseille France
Afzglihm Umwmpotkpc Hqqoduvj Lorenskog Norway
Evhyuei Ueyxbzruxhrs Mwbkbcq Cguosqm Rnrnrmhxe (koiwoeu Mph Rotterdam The Netherlands
Arjcbe Ucuhupzlyv Hikdkews Aarhus Denmark
Cuzbso Hwxgzsdtbys Rmslhcza Uplwwzyjrzzlu Dz Tutbi Tours France
Urjlpnaidqdmkppxcwblv Mbgbirnw Akv Munster Germany
Kfinbitn djc Uqjzouuffnmw Mjasfrhw Aqq Munich Germany
Uhacujtfpehwjnvnhoaxr Wrjhvtoxg Ate Wuerzburg Germany
Aywtsta Upyjo Sdhmdxibk Lukbwb Df Bwfucfd Bologna Italy
Hjupx Mabnw Oe Rmbxwmx Hm Aalesund Norway
Uiwhelwhotnutu Cwxthpo Kxqnfowbj Gdansk Poland
Hvhjydti Vomc dscfcnds Barcelona Spain
Hbaviaet Uqnjnehmfcrvj do A Cwkvtp A Coruna Galicia Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
23.04.2024
Belgium Belgium
Not recruiting
23.04.2024
Czechia Czechia
Not recruiting
23.04.2024
Denmark Denmark
Not recruiting
23.04.2024
France France
Not recruiting
23.04.2024
Germany Germany
Not recruiting
23.04.2024
Greece Greece
Not recruiting
23.04.2024
Ireland Ireland
Not recruiting
23.04.2024
Italy Italy
Not recruiting
23.04.2024
Norway Norway
Not recruiting
23.04.2024
Poland Poland
Not recruiting
23.04.2024
Spain Spain
Not recruiting
23.04.2024
Sweden Sweden
Not recruiting
23.04.2024
The Netherlands The Netherlands
Not recruiting
23.04.2024

Trial locations

Investigated drugs:

ALXN2220 is a medication being studied for its potential to treat transthyretin amyloid cardiomyopathy (ATTR-CM). This condition involves the buildup of abnormal protein deposits in the heart, which can lead to heart problems. ALXN2220 is designed to help reduce these protein deposits, potentially improving heart function and reducing related health issues. The study aims to evaluate how effective and safe ALXN2220 is for patients with this condition.

Investigated diseases:

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) – This is a rare heart condition caused by the buildup of abnormal protein deposits called amyloid in the heart tissue. These deposits can lead to the stiffening of the heart muscle, making it difficult for the heart to pump blood effectively. Over time, this can result in symptoms such as fatigue, shortness of breath, and swelling in the legs and feet. The disease typically progresses slowly, but the accumulation of amyloid can eventually lead to more severe heart problems. ATTR-CM is often seen in older adults and can be hereditary or occur without a family history. The condition requires careful monitoring to manage its impact on heart function.

Trial ID:
2023-506669-70-00
Protocol code:
ALXN2220-ATTRCM-301
Trial Phase:
Therapeutic confirmatory (Phase III)

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