Clinical trial • Phase III • Cardiology|Rare Disease

ALXN2220 for Transthyretin amyloid cardiomyopathy (ATTR-CM)

Phase III trial of ALXN2220 for Transthyretin amyloid cardiomyopathy (ATTR-CM).

Overview

Trial Therapeutic Area
Cardiology|Rare Disease
Trial Disease
Transthyretin amyloid cardiomyopathy (ATTR-CM)
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
15-12-2023
First CTIS Authorization Date
12-04-2024

Trial design

Randomised, placebo iv q4w: participants on standard therapy + placebo iv every 4 weeks (placebo comparator arm described as placebo iv q4w).-controlled Phase III trial in Austria, Spain, France and others.

Randomised
Yes
Comparator
PLACEBO IV q4w: Participants on standard therapy + PLACEBO IV every 4 weeks (placebo comparator arm described as PLACEBO IV q4w).
Target Sample Size
654
Trial Duration For Participant
730

Eligibility

Recruits 654 No vulnerable populations selected. Participants are adults (≥18 years). Informed consent is required from adult participants; subject information and informed consent forms (L1_SIS and ICF adults and related translations) are provided..

Pregnancy Exclusion
Female participant is eligible to participate if she is not pregnant or breastfeeding, and is a woman of nonchildbearing potential or is a WOCBP and using a highly effective contraceptive method, and agrees not to donate eggs.
Vulnerable Population
No vulnerable populations selected. Participants are adults (≥18 years). Informed consent is required from adult participants; subject information and informed consent forms (L1_SIS and ICF adults and related translations) are provided.

Inclusion criteria

  • {"criterion_text":"- Male or female ≥ 18 years to ≤ 90 years of age at time of randomization.\n- If treated with locally approved TTR gene silencing agent for ATTR amyloidosis (except as listed in Section 6.9.2 of the protocol), must be on a stable dose for at least 90 calendar days prior to Screening\n- Male and/or female (according to their reproductive organs and functions assigned by chromosomal complement). Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Female participant is eligible to participate if she is not pregnant or breastfeeding, and is a woman of nonchildbearing potential or is a WOCBP and using a highly effective contraceptive method, and agrees not to donate eggs. Male participants are eligible to participate if they agree to refrain from donating fresh unwashed semen plus abstinent from heterosexual intercourse or must agree to use contraception/barrier,\n- Diagnosis of ATTR-CM with either wild-type or a variant TTR genotype based on one of the following: a. Endomyocardial biopsy with confirmatory TTR amyloid typing by either immunohistochemistry or mass spectrometry; OR b. Grade 2 or 3 cardiac uptake on 99mTc scintigraphy (99mTc DPD, 99mTc PYP, or 99mTc HMDP) in the absence of monoclonal gammopathy; OR c. Grade 2 or 3 cardiac uptake on 99mTc scintigraphy (99mTc DPD, 99mTc PYP, or 99mTc HMDP) AND confirmatory TTR amyloid typing by either immunohistochemistry or mass spectrometry in tissue biopsy in the presence of monoclonal gammopathy\n- End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening\n- NT-proBNP > 2000 pg/mL measured by a central laboratory at Screening\n- Treatment with a loop diuretic for at least 30 days prior to Screening\n- History of heart failure as documented by one of the following events within 1 year prior to Screening: a. heart failure hospitalization b. urgent heart failure visit c. episode of volume overload documented by NT-proBNP > 2000 pg/mL (or equivalent BNP)\n- NYHA Class II-IV at Screening\n- Life expectancy of ≥ 6 months as per the Investigator’s judgment"}

Exclusion criteria

  • {"criterion_text":"- Known leptomeningeal amyloidosis\n- Participants with renal failure requiring dialysis or who have an eGFR by CKD-Epi formula < 20 mL/min/1.73 m2 measured by a central laboratory at Screening\n- Polyneuropathy secondary to ATTR amyloidosis requiring a wheelchair (ie, PND score IV)\n- Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis\n- Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening\n- Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment.\n- LVEF < 30% on echocardiography"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Total occurrence of ACM and CV clinical events during the Blinded Treatment Period","definition_or_measurement_approach":"As described in the protocol: assessed by the total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events during the Blinded Treatment Period (difference between ALXN2220 and placebo groups)."}

Secondary endpoints

  • {"endpoint_text":"- Change from baseline in KCCQ-OS to 24 months","definition_or_measurement_approach":"Change from baseline in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) at 24 months."}
  • {"endpoint_text":"- Time to CV related mortality","definition_or_measurement_approach":"Time-to-event analysis for cardiovascular-related death."}
  • {"endpoint_text":"- Change from baseline in 6MWT to 24 months","definition_or_measurement_approach":"Change from baseline in 6-minute walk test (6MWT) at 24 months."}
  • {"endpoint_text":"- Rate of CV clinical events","definition_or_measurement_approach":"Incidence/rate of prespecified cardiovascular clinical events as defined in the protocol."}
  • {"endpoint_text":"- Time to ACM","definition_or_measurement_approach":"Time-to-event analysis for all-cause mortality."}
  • {"endpoint_text":"- Incidence of TEAEs and treatment-emergent SAEs. Changes from baseline in physical examination, vital signs, clinical laboratory tests, and 12-lead ECGs","definition_or_measurement_approach":"Safety assessments including incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and changes from baseline in physical exam, vitals, labs, and 12-lead ECGs."}
  • {"endpoint_text":"- ADA incidence and NAb incidence, response categories and titer","definition_or_measurement_approach":"Assessment of anti-drug antibody (ADA) incidence and neutralizing antibody (NAb) incidence, including response categories and titers."}
  • {"endpoint_text":"- Serum ALXN2220 concentration","definition_or_measurement_approach":"Pharmacokinetic measurement of serum ALXN2220 concentrations."}

Recruitment

Registry Or Advocacy Recruitment
True, Fondazione Toscana Gabriele Monasterio (listed as patient organisation/association among trial sites); PAG (patient advocacy group) materials are referenced (PAG Study Intro Letter).
Digital Remote Recruitment
True, materials include Social Posts, Website_Newsletter Listings, Landing Page materials, and Patient Information Video indicating digital/remote recruitment channels.
Planned Sample Size
654
Recruitment Window Months
54
Consent Approach
Informed consent is obtained from adult participants (≥18 years). Subject information and informed consent forms (L1_SIS and ICF adults and related addenda) are provided; language-specific ICFs and patient-facing documents are available (multiple translations: EN, DE, ES, FR, IT, NL, PL, GR/EL, SE, CZ, DK and others as provided). No assent procedures are indicated (study population adults only).

Methods

  • Recruitment Brochure — patient-facing brochure (country-specific versions present, e.g., DE, ES, IT) targeting potential participants and referring clinicians.
  • Half Page Ad (e.g., DE-AT) — print advertisement targeting potential participants in Austria/Germany.
  • Recruitment Poster — clinic/hospital posters for visual outreach at sites.
  • Social Posts — social media posts for outreach to potential participants (country-specific social post materials available).
  • Website / Newsletter Listing — online listings on websites and newsletters (country-specific landing pages and website materials available).
  • Patient Information Video — video materials to inform potential participants about the study.
  • PI-to-Participant Invitation to Trial Letter — direct invitation letter from principal investigators to potential participants.
  • PAG Study Intro Letter — engagement with patient advocacy groups (PAG) and patient organizations.
  • Study Comfort Items Memo / Study Intro materials — site materials to support recruitment and participant engagement.

Geography

Total Number Of Sites
128
Total Number Of Participants
654

Austria

Earliest CTIS Part Ii Submission Date
05-04-2024
Latest Decision Or Authorization Date
24-04-2026
Processing Time Days
749
Number Of Sites
5
Number Of Participants
12

Sites

Site Name
Klinikum Wels-Grieskirchen GmbH
Department Name
"Abteilung für Innere Medizin II Kardiologie und Intensivmedizin "
Principal Investigator Name
Thomas Weber
Principal Investigator Email
Thomas.Weber@klinikum-wegr.at
Contact Person Name
Thomas Weber
Contact Person Email
Thomas.Weber@klinikum-wegr.at
Site Name
Medizinische Universitaet Innsbruck
Department Name
Department Of Internal Medicine III, Division of Cardiology and Angiology
Principal Investigator Name
Gerhard Pölzl
Principal Investigator Email
gerhard.poelzl@tirol-kliniken.at
Contact Person Name
Gerhard Pölzl
Site Name
Medical University Of Vienna
Department Name
Department Of Internal Medicine II, Division of Cardiology
Principal Investigator Name
Roza Badr-Eslam
Principal Investigator Email
roza.badreslam@meduniwien.ac.at
Contact Person Name
Roza Badr-Eslam
Site Name
Stadt Wien Wiener Gesundheitsverbund
Department Name
3. Medizinische Abteilung
Principal Investigator Name
Stephan Dobner
Principal Investigator Email
stephan.dobner@gesundheitsverbund.at
Contact Person Name
Stephan Dobner
Site Name
Medical University Of Graz
Department Name
Department Of Internal Medicine, Division of Cardiology
Principal Investigator Name
Nicolas Verheyen
Principal Investigator Email
nicolas.verheyen@medunigraz.at
Contact Person Name
Nicolas Verheyen
Contact Person Email
nicolas.verheyen@medunigraz.at

Spain

Earliest CTIS Part Ii Submission Date
11-03-2024
Latest Decision Or Authorization Date
24-04-2026
Processing Time Days
773
Number Of Sites
13
Number Of Participants
49

Sites

Site Name
Complexo Hospitalario Universitario A Coruna
Department Name
Cardiology
Principal Investigator Name
Maria Crespo-Leiro
Principal Investigator Email
marisa.crespo.leiro@sergas.es
Contact Person Name
Maria Crespo-Leiro
Contact Person Email
marisa.crespo.leiro@sergas.es
Site Name
Hospital Universitario Virgen De La Victoria
Department Name
Cardiology
Principal Investigator Name
Jose Manuel Garcia Pinilla
Principal Investigator Email
pinilla@secardiologia.es
Contact Person Name
Jose Manuel Garcia Pinilla
Contact Person Email
pinilla@secardiologia.es
Site Name
Hospital Universitario De Jaen
Department Name
Cardiology
Principal Investigator Name
Jose Angel Urbano-Moral
Principal Investigator Email
jaurbanomoral@gmail.com
Contact Person Name
Jose Angel Urbano-Moral
Contact Person Email
jaurbanomoral@gmail.com
Site Name
Bellvitge University Hospital
Department Name
Cardiology
Principal Investigator Name
Jose Gonzalez Castello
Principal Investigator Email
jgonzalez@bellvitgehospital.cat
Contact Person Name
Jose Gonzalez Castello
Site Name
Complexo Hospitalario Universitario De Santiago
Department Name
Cardiology
Principal Investigator Name
Oscar Otero García
Principal Investigator Email
oscar.otero.garcia@sergas.es
Contact Person Name
Oscar Otero García
Contact Person Email
oscar.otero.garcia@sergas.es
Site Name
Clinica Universidad De Navarra (Pamplona)
Department Name
Cardiology
Principal Investigator Name
Juan Jose Gavira Gomez
Principal Investigator Email
jjgavira@unav.es
Contact Person Name
Juan Jose Gavira Gomez
Contact Person Email
jjgavira@unav.es
Site Name
Hospital Universitario De Salamanca
Department Name
Cardiology
Principal Investigator Name
Rocio Eiros Bachiller
Principal Investigator Email
reirosb@saludcastillayleon.es
Contact Person Name
Rocio Eiros Bachiller
Contact Person Email
reirosb@saludcastillayleon.es
Site Name
Hospital Son Llatzer
Department Name
Cardiology
Principal Investigator Name
Tomas Ripoll-Vera
Principal Investigator Email
tripoll@hsll.es
Contact Person Name
Tomas Ripoll-Vera
Contact Person Email
tripoll@hsll.es
Site Name
Hospital Clinico Universitario De Valencia
Department Name
Cardiology
Principal Investigator Name
Julio Eduardo Nuñez Villota
Principal Investigator Email
yulnunez@gmail.com
Contact Person Name
Julio Eduardo Nuñez Villota
Contact Person Email
yulnunez@gmail.com
Site Name
University Clinical Hospital Virgen De La Arrixaca
Department Name
Cardiology
Principal Investigator Name
Domingo Andres Pascual Figal
Principal Investigator Email
dpascual@um.es
Contact Person Name
Domingo Andres Pascual Figal
Contact Person Email
dpascual@um.es
Site Name
Hospital Universitario Puerta De Hierro De Majadahonda
Department Name
Cardiology
Principal Investigator Name
Pablo Garcia Pavia
Principal Investigator Email
pablogpavia@yahoo.es
Contact Person Name
Pablo Garcia Pavia
Contact Person Email
pablogpavia@yahoo.es
Site Name
Clinica Universidad De Navarra (Madrid)
Department Name
Cardiology
Principal Investigator Name
Juan Jose Gavira Gomez
Principal Investigator Email
jjgavira@unav.es
Contact Person Name
Juan Jose Gavira Gomez
Contact Person Email
jjgavira@unav.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Internal medicine
Principal Investigator Name
Fernando Martinez Valle
Principal Investigator Email
fernando.martinez@vallhebron.cat
Contact Person Name
Fernando Martinez Valle

France

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
23-04-2026
Processing Time Days
768
Number Of Sites
13
Number Of Participants
56

Sites

Site Name
Assistance Publique Hopitaux De Paris (Creteil)
Department Name
Cardiologie
Principal Investigator Name
Thibaud Damy
Principal Investigator Email
thibaud.damy@aphp.fr
Contact Person Name
Thibaud Damy
Contact Person Email
thibaud.damy@aphp.fr
Site Name
Hospices Civils De Lyon
Department Name
Cardiologie
Principal Investigator Name
Nathan Mewton
Principal Investigator Email
nathan.mewtonl@chu-lyon.fr
Contact Person Name
Nathan Mewton
Contact Person Email
nathan.mewtonl@chu-lyon.fr
Site Name
Centre Hospitalier Regional Universitaire De Tours
Department Name
Cardiologie
Principal Investigator Name
Anne Bernard
Principal Investigator Email
anne.bernard@univ-tours.fr
Contact Person Name
Anne Bernard
Contact Person Email
anne.bernard@univ-tours.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Cardiologie
Principal Investigator Name
Olivier Lairez
Principal Investigator Email
lairez.o@chu-toulouse.fr
Contact Person Name
Olivier Lairez
Contact Person Email
lairez.o@chu-toulouse.fr
Site Name
Centre Hospitalier Universitaire De Rennes
Department Name
Cardiologie
Principal Investigator Name
Erwan Donal
Principal Investigator Email
erwan.donal@chu-rennes.fr
Contact Person Name
Erwan Donal
Contact Person Email
erwan.donal@chu-rennes.fr
Site Name
Centre Hospitalier Universitaire De Nantes
Department Name
Cardiologie
Principal Investigator Name
Nicolas Piriou
Principal Investigator Email
nicolas.piriou@chu-nantes.fr
Contact Person Name
Nicolas Piriou
Contact Person Email
nicolas.piriou@chu-nantes.fr
Site Name
Centre Hospitalier Universitaire De Poitiers
Department Name
Cardiologie
Principal Investigator Name
Claire Bouleti
Principal Investigator Email
claire.bouleti@chu-poitiers.fr
Contact Person Name
Claire Bouleti
Contact Person Email
claire.bouleti@chu-poitiers.fr
Site Name
Institut de Cardiologie (Paris)
Department Name
Cardiologie / Institut de Cardiologie
Principal Investigator Name
Emmanuelle Begot
Principal Investigator Email
e.begot@ihuican.org
Contact Person Name
Emmanuelle Begot
Contact Person Email
e.begot@ihuican.org
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Cardiologie
Principal Investigator Name
François Roubille
Principal Investigator Email
f-roubille@chu-montpellier.fr
Contact Person Name
François Roubille
Contact Person Email
f-roubille@chu-montpellier.fr
Site Name
Assistance Publique Hopitaux De Paris (Paris)
Department Name
Cardiologie, Insuffisance cardiaque
Principal Investigator Name
Vincent Algalarrondo
Principal Investigator Email
vincent.algalarrondo@aphp.fr
Contact Person Name
Vincent Algalarrondo
Contact Person Email
vincent.algalarrondo@aphp.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
UMCV
Principal Investigator Name
Patricia Réant
Principal Investigator Email
patricia.reant@chu-bordeaux.fr
Contact Person Name
Patricia Réant
Contact Person Email
patricia.reant@chu-bordeaux.fr
Site Name
Assistance Publique Hopitaux De Marseille
Department Name
Cardiologie, Insuffisance cardiaque
Principal Investigator Name
Gilbert Habib
Principal Investigator Email
gilbert.habib@ap-hm.fr
Contact Person Name
Gilbert Habib
Contact Person Email
gilbert.habib@ap-hm.fr
Site Name
Centre Hospitalier Universitaire De Rennes (additional site)
Department Name
Cardiologie (additional contact)
Principal Investigator Name
Erwan Donal
Principal Investigator Email
erwan.donal@chu-rennes.fr
Contact Person Name
Erwan Donal
Contact Person Email
erwan.donal@chu-rennes.fr

Germany

Earliest CTIS Part Ii Submission Date
19-03-2024
Latest Decision Or Authorization Date
22-04-2026
Processing Time Days
763
Number Of Sites
13
Number Of Participants
52

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Klinik für Kardiologie, Angiologie und Intensivmedizin
Principal Investigator Name
Sebastian Spethmann
Principal Investigator Email
sebastian.spethmann@dhzc-charite.de
Contact Person Name
Sebastian Spethmann
Site Name
Universitaetsklinikum Schleswig-Holstein
Department Name
Klinik für Kardiologie, Angiologie und internistische Intensivmedizin
Principal Investigator Name
Oliver Müller
Principal Investigator Email
Oliver.Mueller@uksh.de
Contact Person Name
Oliver Müller
Contact Person Email
Oliver.Mueller@uksh.de
Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Kardiologie und Angiologie
Principal Investigator Name
Tienush Rassaf
Principal Investigator Email
Tienush.Rassaf@uk-essen.de
Contact Person Name
Tienush Rassaf
Contact Person Email
Tienush.Rassaf@uk-essen.de
Site Name
Medizinische Hochschule Hannover
Department Name
Medizinische Hochschule Hannover (MHH)
Principal Investigator Name
Udo Bavendiek
Principal Investigator Email
Bavendiek.udo@mh-hannover.de
Contact Person Name
Udo Bavendiek
Contact Person Email
Bavendiek.udo@mh-hannover.de
Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Medizinische Klinik I Kardiologie
Principal Investigator Name
Stephan Störk
Principal Investigator Email
stoerk_s@ukw.de
Contact Person Name
Stephan Störk
Contact Person Email
stoerk_s@ukw.de
Site Name
HOPA MVZ GmbH
Department Name
Hämatologisch-Onkologische Praxis Altona (HOPA)
Principal Investigator Name
Timon Hansen
Principal Investigator Email
timon.hansen@hopa.de
Contact Person Name
Timon Hansen
Contact Person Email
timon.hansen@hopa.de
Site Name
Universitaet Leipzig
Department Name
Innere Medizin – Klinik und Poliklinik für Kardiologie
Principal Investigator Name
Daniell Lavall
Principal Investigator Email
daniel.lavall@medizin.uni-leipzig.de
Contact Person Name
Daniell Lavall
Site Name
Universitaetsklinikum Giessen und Marburg GmbH
Department Name
Med. Klinik I, Kardiologie / Angiologie
Principal Investigator Name
Birgit Aßmus
Principal Investigator Email
birgit.assmus@innere.med.uni-giessen.de
Contact Person Name
Birgit Aßmus
Site Name
Universitaetsklinikum Muenster AöR
Department Name
Herz-MRT-Zentrum Sektion für Herzbildgebung - Klinik für Kardiologie I
Principal Investigator Name
Ali Yilmaz
Principal Investigator Email
ali.yilmaz@ukmuenster.de
Contact Person Name
Ali Yilmaz
Contact Person Email
ali.yilmaz@ukmuenster.de
Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Medizinische Klinik und Poliklinik I
Principal Investigator Name
Stephanie Schwarting
Principal Investigator Email
Stephanie.Bieber@med.uni-muenchen.de
Contact Person Name
Stephanie Schwarting
Site Name
University Hospital Cologne AöR
Department Name
Herzzentrum der Universität zu Köln
Principal Investigator Name
Roman Pfister
Principal Investigator Email
roman.pfister@uk-koeln.de
Contact Person Name
Roman Pfister
Contact Person Email
roman.pfister@uk-koeln.de
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Innere Medizin 3 / Kardiologie
Principal Investigator Name
Fabian aus dem Siepen
Principal Investigator Email
Fabian.Siepen@med.uni-heidelberg.de
Contact Person Name
Fabian aus dem Siepen
Site Name
Universitaet Des Saarlandes
Department Name
Klinik für Innere Medizin III Kardiologie, Angiologie und Internistische Intensivmedizin
Principal Investigator Name
Ingrid Kindermann
Principal Investigator Email
Ingrid.Kindermann@uks.eu
Contact Person Name
Ingrid Kindermann
Contact Person Email
Ingrid.Kindermann@uks.eu

Italy

Earliest CTIS Part Ii Submission Date
26-02-2024
Latest Decision Or Authorization Date
22-04-2026
Processing Time Days
786
Number Of Sites
13
Number Of Participants
47

Sites

Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
Dipartimento di Medicina Clinica e Molecolare
Principal Investigator Name
Maria Beatrice Musumeci
Principal Investigator Email
Beatrice.musumeci@uniroma1.it
Contact Person Name
Maria Beatrice Musumeci
Contact Person Email
Beatrice.musumeci@uniroma1.it
Site Name
Azienda Ospedaliera Universitaria Gaetano Martino Messina
Department Name
UTIC Cardiologia
Principal Investigator Name
Gianluca Di Bella
Principal Investigator Email
gianluca.dibella@unime.it
Contact Person Name
Gianluca Di Bella
Contact Person Email
gianluca.dibella@unime.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
UOC Medicina Generale 2 - Centro Amiloidosi Sistemiche e Malattie ad Alta Complessità
Principal Investigator Name
Giovanni Palladini
Principal Investigator Email
giovanni.palladini@unipv.it
Contact Person Name
Giovanni Palladini
Contact Person Email
giovanni.palladini@unipv.it
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Dipartimento Malattie Cardio-Toraco-Vascolare, Cardiologia
Principal Investigator Name
Simone Longhi
Principal Investigator Email
simone.longhi@aosp.bo.it
Contact Person Name
Simone Longhi
Contact Person Email
simone.longhi@aosp.bo.it
Site Name
Cattinara Hospital
Department Name
Dipartimento Cardiotoracovascolare
Principal Investigator Name
Marco Merlo
Principal Investigator Email
marco.merlo@asugi.sanita.fvg.it
Contact Person Name
Marco Merlo
Site Name
Azienda Ospedaliera Dei Colli (Naples)
Department Name
Dipartimento di Scienze mediche traslazionali- Unità di Malattie Genetiche e Rare Cardiovascolari
Principal Investigator Name
Giuseppe Limongelli
Principal Investigator Email
limongelligiuseppe@libero.it
Contact Person Name
Giuseppe Limongelli
Contact Person Email
limongelligiuseppe@libero.it
Site Name
University Hospital Of Ferrara
Department Name
Cardiovascular Medicine
Principal Investigator Name
Matteo Serenelli
Principal Investigator Email
cardiologia@ospfe.it
Contact Person Name
Matteo Serenelli
Contact Person Email
cardiologia@ospfe.it
Site Name
Fondazione Toscana Gabriele Monasterio
Department Name
U.O. Complessa di Cardiologia e Medicina Cardiovascolare
Principal Investigator Name
Giuseppe Vergaro
Principal Investigator Email
vergaro@ftgm.it
Contact Person Name
Giuseppe Vergaro
Contact Person Email
vergaro@ftgm.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
Department of Cardio-Thoracic-Vascular Diseases
Principal Investigator Name
Stefano Carugo
Principal Investigator Email
stefano.carugo@policlinico.mi.it
Contact Person Name
Stefano Carugo
Site Name
Ospedale San Giovanni Bosco
Department Name
SCDU Nefrologia e Dialisi - CMID
Principal Investigator Name
Roberta Fenoglio
Principal Investigator Email
roberta.fenoglio@unito.it
Contact Person Name
Roberta Fenoglio
Contact Person Email
roberta.fenoglio@unito.it
Site Name
Careggi University Hospital
Department Name
Interventional Cardiological Structural SOD
Principal Investigator Name
Francesco Cappelli
Principal Investigator Email
f.cappelli@unifi.it
Contact Person Name
Francesco Cappelli
Contact Person Email
f.cappelli@unifi.it
Site Name
Azienda Ospedale-Universita Padova
Department Name
Cardio-Toraco-Vascolare e Sanità Pubblica
Principal Investigator Name
Alberto Cipriani
Principal Investigator Email
alberto.cipriani@unipd.it
Contact Person Name
Alberto Cipriani
Contact Person Email
alberto.cipriani@unipd.it
Site Name
Ospedale Giovan Battista Morgagni-Luigi Pierantoni Di Forlì
Department Name
Dipartimento Emergenza, Internistico e Cardiologico Forlì e Cesena-U.O.C. di Cardiologia
Principal Investigator Name
Elisa Gardini
Principal Investigator Email
elisa.gardini@auslromagna.it
Contact Person Name
Elisa Gardini
Contact Person Email
elisa.gardini@auslromagna.it

Belgium

Earliest CTIS Part Ii Submission Date
25-03-2024
Latest Decision Or Authorization Date
21-04-2026
Processing Time Days
756
Number Of Sites
3
Number Of Participants
20

Sites

Site Name
Ziekenhuis Oost Limburg
Department Name
Cardiologie
Principal Investigator Name
Matthias Dupont
Principal Investigator Email
Matthias.Dupont@zol.be
Contact Person Name
Matthias Dupont
Contact Person Email
Matthias.Dupont@zol.be
Site Name
Az St-Jan Brugge-Oostende A.V.
Department Name
Cardiologie - Campus Sint-Jan
Principal Investigator Name
Philippe Debonnaire
Principal Investigator Email
cardiologie@azsintjan.be
Contact Person Name
Philippe Debonnaire
Contact Person Email
cardiologie@azsintjan.be
Site Name
Hopital Erasme
Department Name
Cardiologie
Principal Investigator Name
Antoine Bondue
Principal Investigator Email
antoine.bondue@hubruxelles.be
Contact Person Name
Antoine Bondue
Contact Person Email
antoine.bondue@hubruxelles.be

Netherlands

Earliest CTIS Part Ii Submission Date
12-01-2024
Latest Decision Or Authorization Date
23-04-2026
Processing Time Days
831
Number Of Sites
4
Number Of Participants
24

Sites

Site Name
Universitair Medisch Centrum Utrecht
Department Name
Cardiology
Principal Investigator Name
Marish Oerlemans
Principal Investigator Email
m.oerlemans@umcutrecht.nl
Contact Person Name
Marish Oerlemans
Contact Person Email
m.oerlemans@umcutrecht.nl
Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
Cardiology
Principal Investigator Name
Michelle Michels
Principal Investigator Email
m.michels@erasmusmc.nl
Contact Person Name
Michelle Michels
Contact Person Email
m.michels@erasmusmc.nl
Site Name
Universitair Medisch Centrum Groningen
Department Name
Cardiology
Principal Investigator Name
Peter van der Meer
Principal Investigator Email
p.van.der.meer@umcg.nl
Contact Person Name
Peter van der Meer
Contact Person Email
p.van.der.meer@umcg.nl
Site Name
Universiteit Maastricht
Department Name
Cardiology
Principal Investigator Name
Christian Knackstedt
Principal Investigator Email
secretariaat.HVC@mumc.nl
Contact Person Name
Christian Knackstedt
Contact Person Email
secretariaat.HVC@mumc.nl

Ireland

Earliest CTIS Part Ii Submission Date
19-03-2024
Latest Decision Or Authorization Date
20-04-2026
Processing Time Days
761
Number Of Sites
1
Number Of Participants
18

Sites

Site Name
St James's Hospital
Department Name
Cardiology
Principal Investigator Name
Murphy Ross
Principal Investigator Email
RTMurphy@stjames.ie
Contact Person Name
Murphy Ross
Contact Person Email
RTMurphy@stjames.ie

Denmark

Earliest CTIS Part Ii Submission Date
25-06-2024
Latest Decision Or Authorization Date
20-04-2026
Processing Time Days
665
Number Of Sites
3
Number Of Participants
12

Sites

Site Name
Rigshospitalet
Department Name
Hjertemedicinsk klinik B 2142
Principal Investigator Name
Kasper Rossing
Principal Investigator Email
kasper.rossing@regionh.dk
Contact Person Name
Kasper Rossing
Contact Person Email
kasper.rossing@regionh.dk
Site Name
Aarhus Universitetshospital
Department Name
Hjertesygdomme Klinik 1
Principal Investigator Name
Steen Hvitfeldt Poulsen
Principal Investigator Email
steen.hvitfeldt@rm.dk
Contact Person Name
Steen Hvitfeldt Poulsen
Contact Person Email
steen.hvitfeldt@rm.dk
Site Name
Odense University Hospital
Department Name
Hjertemedicinsk Afdeling B
Principal Investigator Name
Michael Maiborg
Principal Investigator Email
Michael.maiborg@rsyd.dk
Contact Person Name
Michael Maiborg
Contact Person Email
Michael.maiborg@rsyd.dk

Czechia

Earliest CTIS Part Ii Submission Date
24-04-2024
Latest Decision Or Authorization Date
21-04-2026
Processing Time Days
727
Number Of Sites
3
Number Of Participants
12

Sites

Site Name
Institute For Clinical And Experimental Medicine
Department Name
Kardiocentrum Klinika kardiologie
Principal Investigator Name
Milos Kubanek
Principal Investigator Email
mikb@ikem.cz
Contact Person Name
Milos Kubanek
Contact Person Email
mikb@ikem.cz
Site Name
Fakultni Nemocnice U Sv Anny V Brne
Department Name
I interni kardiologicka klinika
Principal Investigator Name
Hana Poloczkova
Principal Investigator Email
hana.polockova@fnusa.cz
Contact Person Name
Hana Poloczkova
Contact Person Email
hana.polockova@fnusa.cz
Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
II interni klinika - klinika kardilogie a angiologie
Principal Investigator Name
Tomas Palecek
Principal Investigator Email
tomas.palecek@vfn.cz
Contact Person Name
Tomas Palecek
Contact Person Email
tomas.palecek@vfn.cz

Sweden

Earliest CTIS Part Ii Submission Date
24-04-2024
Latest Decision Or Authorization Date
20-04-2026
Processing Time Days
726
Number Of Sites
4
Number Of Participants
14

Sites

Site Name
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department Name
Kardiologen
Principal Investigator Name
Charlotta Ljungman
Principal Investigator Email
charlotta.Ljungman@vgregion.se
Contact Person Name
Charlotta Ljungman
Contact Person Email
charlotta.Ljungman@vgregion.se
Site Name
Region Skane Skanes Universitetssjukhus
Department Name
Avdelning för Kardiologi
Principal Investigator Name
Gustav Smith
Principal Investigator Email
gustav.smith@med.lu.se
Contact Person Name
Gustav Smith
Contact Person Email
gustav.smith@med.lu.se
Site Name
Karolinska University Hospital
Department Name
ME Kardiologi
Principal Investigator Name
Jonas Faxén
Principal Investigator Email
jonas.faxen@regionstockholm.se
Contact Person Name
Jonas Faxén
Contact Person Email
jonas.faxen@regionstockholm.se
Site Name
Region Vaesterbotten
Department Name
Skellefteå lasarett, Lasarettsvägen 29D, 931 41 Skellefteå, Forskningsmottagning Plan 2
Principal Investigator Name
Jonas Andersson
Principal Investigator Email
jonas.so.andersson@regionvasterbotten.se
Contact Person Name
Jonas Andersson

Poland

Earliest CTIS Part Ii Submission Date
05-04-2024
Latest Decision Or Authorization Date
24-04-2026
Processing Time Days
749
Number Of Sites
3
Number Of Participants
12

Sites

Site Name
Uniwersyteckie Centrum Kliniczne (Gdansk)
Department Name
II Klinika Kardiologii i Elektroterapii Serca
Principal Investigator Name
Ludmila Danilowicz-Szymanowicz
Principal Investigator Email
ludwik@gumed.edu.pl
Contact Person Name
Ludmila Danilowicz-Szymanowicz
Contact Person Email
ludwik@gumed.edu.pl
Site Name
Uniwersytecki Szpital Kliniczny W Poznaniu
Department Name
Oddzial Kardiologii z Pododdzialem Kardiologii "F" i Pododdzialem Kardiologii "G"
Principal Investigator Name
Ewa Straburzynska-Migaj
Principal Investigator Email
ewa.straburzynska-migaj@skpp.edu.pl
Contact Person Name
Ewa Straburzynska-Migaj
Site Name
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
Department Name
Klinika Chorob Wewnetrznych, Nadcisnienia Tetniczego i Angiologii
Principal Investigator Name
Maciej Sinski
Principal Investigator Email
msinski@wum.edu.pl
Contact Person Name
Maciej Sinski
Contact Person Email
msinski@wum.edu.pl

Greece

Earliest CTIS Part Ii Submission Date
12-01-2024
Latest Decision Or Authorization Date
20-04-2026
Processing Time Days
828
Number Of Sites
1
Number Of Participants
8

Sites

Site Name
Alexandra Hospital
Department Name
Department of Clinical Therapeutics
Principal Investigator Name
Efstathios Kastritis
Principal Investigator Email
ekastritis@med.uoa.gr
Contact Person Name
Efstathios Kastritis
Contact Person Email
ekastritis@med.uoa.gr

Norway

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
22-04-2026
Processing Time Days
646
Number Of Sites
4
Number Of Participants
10

Sites

Site Name
Oslo University Hospital HF
Department Name
Department of cardiology
Principal Investigator Name
Einar Gude
Principal Investigator Email
egude@ous-hf.no
Contact Person Name
Einar Gude
Contact Person Email
egude@ous-hf.no
Site Name
Akershus University Hospital
Department Name
Department of cardiology
Principal Investigator Name
Henrik Shirmer
Principal Investigator Email
henrik.schirmer@medisin.uio.no
Contact Person Name
Henrik Shirmer
Contact Person Email
henrik.schirmer@medisin.uio.no
Site Name
St. Olavs Hospital HF
Department Name
Department of Circulation and Medical Imaging / Clinic of Cardiology
Principal Investigator Name
Hårvard Dalen
Principal Investigator Email
havard.dalen@ntnu.no
Contact Person Name
Hårvard Dalen
Contact Person Email
havard.dalen@ntnu.no
Site Name
Helse Moere Og Romsdal HF
Department Name
Department of cardiology
Principal Investigator Name
Torstein Hole
Principal Investigator Email
torstein.hole@helse-mr.no
Contact Person Name
Torstein Hole
Contact Person Email
torstein.hole@helse-mr.no

Sponsor

Primary sponsor

Full Name
Alexion Pharmaceuticals Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Investigational products

Investigational Product Name
Recombinant human anti-ATTR immunoglobulin G1 (IgG1) monoclonal antibody (mAb)
Active Substance
ALXN2220
Modality
Monoclonal antibody
Routes Of Administration
Solution for infusion (intravenous infusion)
Route
Intravenous (infusion)
Authorisation Status
prodAuthStatus: 1 (per productDictionaryInfo)
Orphan Designation
Yes
Frequency
IV q4w (every 4 weeks)
Investigational Product Name
ALXN2220 Placebo Product
Modality
Other
Frequency
PLACEBO IV q4w
Combination Treatment
Yes

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