Clinical trial • Phase III • Cardiology|Rare Disease

Vutrisiran for Hereditary (hATTR) transthyretin amyloidosis with cardiomyopathy|Wild-type (wtATTR) transthyretin amyloidosis with cardiomyopathy

Phase III trial of Vutrisiran for Hereditary (hATTR) transthyretin amyloidosis with cardiomyopathy|Wild-type (wtATTR) transthyretin amyloidosis with cardi…

Overview

Trial Therapeutic Area
Cardiology|Rare Disease
Trial Disease
Hereditary (hATTR) transthyretin amyloidosis with cardiomyopathy|Wild-type (wtATTR) transthyretin amyloidosis with cardiomyopathy
Trial Stage
Phase III
Drug Modality
Oligonucleotide|Other
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
04-10-2024
First CTIS Authorization Date
18-11-2024

Trial design

Randomised, open-label, placebo (0.9% sodium chloride) administered as subcutaneous injection every 12 weeks ±7 days up to 36 months (placebo comparator arm). active treatment arm: vutrisiran 25 mg sc every 12 weeks ±7 days up to 36 months.-controlled Phase III trial in Croatia, Sweden, Poland and others.

Randomised
Yes
Open Label
Yes
Comparator
Placebo (0.9% Sodium chloride) administered as subcutaneous injection every 12 weeks ±7 days up to 36 months (placebo comparator arm). Active treatment arm: Vutrisiran 25 mg SC every 12 weeks ±7 days up to 36 months.
Target Sample Size
250
Trial Duration For Participant
1095

Eligibility

Recruits 250 The record indicates vulnerable population selection. Inclusion criterion 9 requires the patient "is able to understand and is willing and able to comply with the study requirements and to provide written informed consent." Country-specific subject information and informed consent forms are provided (multiple country versions). No explicit assent process for minors is specified in the available criteria..

Pregnancy Exclusion
27. Female patient is pregnant, planning a pregnancy, or breast-feeding
Vulnerable Population
The record indicates vulnerable population selection. Inclusion criterion 9 requires the patient "is able to understand and is willing and able to comply with the study requirements and to provide written informed consent." Country-specific subject information and informed consent forms are provided (multiple country versions). No explicit assent process for minors is specified in the available criteria.

Inclusion criteria

  • {"criterion_text":"- 1. Age 18 to 85 years\n- 10. Patient agrees to sign a separate medical records release form, where allowed by local regulations, to allow for the collection of information on vital status, cardiac transplant procedures, leftventricular assist device placement, and hospitalizations, for the duration of the DB Period of the study.\n- 2. Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hATTR amyloidosis with cardiomyopathy or wtATTR amyloidosis with cardiomyopathy\n- 3. Medical history of HF with at least 1 prior hospitalization for HF (not due to arrhythmia or a conduction system disturbance treated with a permanent pacemaker) OR clinical evidence of HF (with or without hospitalization) manifested by signs and symptoms of volume overload or elevated intracardiac pressures (eg, elevated jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or auscultation, peripheral edema) that currently requires treatment with a diuretic.\n- 4. Patient meets one of the following criteria: a. Tafamidis-naïve and not actively planning to commence treatment with tafamidis during the first 12 months following randomization (per exclusion criterion #7); or b. On tafamidis (Note: must be on-label use of commercial tafamidis per an approved cardiomyopathy indication and dose in the country of use)\n- 5. Patient is clinically stable, with no CV-related hospitalizations within 6 weeks prior to randomization, as assessed by the Investigator.\n- 6. Screening NT-proBNP >300 ng/L and <8500 ng/L; in patients with permanent or persistent atrial fibrillation, Screening NT-proBNP >600 ng/L and <8500 ng/L.\n- 7. Able to complete ≥150 meters on the 6-MWT at Screening.\n- 8. Have a Karnofsky performance status of ≥60%.\n- 9. Patient is able to understand and is willing and able to comply with the study requirements and to provide written informed consent."}

Exclusion criteria

  • {"criterion_text":"- 1. Has known primary amyloidosis(AL amyloidosis)or leptomeningeal amyloidosis\n- 19. Has persistent elevation of systolic (>170 mmHg) or diastolic (>100 mmHg) blood pressure that is considered uncontrolled by physician\n- 2. NYHA Class IV heart failure; or NYHA Class III heart failure AND ATTR Amyloidosis Disease Stage 3 (defined as NT-proBNP >3000 ng/L and eGFR <45 ml/min)\n- 20. Has untreated hypo- or hyperthyroidism\n- 21. Has an active infection requiring systemic antiviral, antiparasitic or antimicrobial therapy that will not be completed prior to dosing (Day 1)\n- 22. Prior or anticipated (during the first 12 months after randomization) heart, liver or other organ transplant or implantation of left-ventricular assist device\n- 23. History of multiple drug allergies; or history of allergic reaction to any component of or excipient in the study drug\n- 24. History of intolerance to SC injection(s) or significant abdominal scarring that could potentially hinder study drug administration or evaluation of local tolerability\n- 25. Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation\n- 26. Is not willing to comply with the contraceptive requirements during the study period\n- 27. Female patient is pregnant, planning a pregnancy, or breast-feeding\n- 10. Currently taking doxycycline, ursodeoxycholic acid or tauroursodeoxycholic acid; if previously on any of these agents, must have completed a 30-day wash-out prior to dosing (Day 1)\n- 28. Unwilling or unable to limit alcohol consumption throughout the course of the study\n- 29. History of alcohol abuse, within the last 12 months before Screening, in the opinion of the Investigator\n- 3. Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV (requires cane or stick to walk due to polyneuropathy, or is wheelchair bound) at the Screening visit\n- 30.History of illicit drug abuse within the past 5 years that in the opinion of the Investigator would interfere with compliance with study procedures or follow-up visits\n- 4. Has any of the following laboratory parameter assessments at Screening: a. AST or ALT levels >2.0 × ULN; b. Total bilirubin >2.0 × ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is <2 × ULN); c. International normalized ratio (INR) >1.5 (unless patients were on anticoagulant therapy in which case excluded if INR >3.5)\n- 5. Has eGFR <30 mL/min/1.73 m2 (using the modification of diet in renal disease [MDRD] formula) at Screening\n- 6. Has known human immunodeficiency virus infection; or evidence of current or chronic hepatitis C virus or hepatitis B virus infection\n- 7. Tafamidis-naïve patients (per inclusion criterion #4a) for whom the Investigator actively plans or anticipates commencing treatment with tafamidis either during the Screening Period or the first 12 months following randomization, taking into consideration clinical status, patient preference and/or commercial availability of tafamidis\n- 8. Received prior TTR-lowering treatment (including revusiran, patisiran or inotersen) or participated in a gene therapy trial for hATTR amyloidosis\n- 9. Currently taking diflunisal; if previously on this agent, must have at least a 30-day wash-out prior to dosing (Day 1)\n- 11. Unwilling to avoid any concurrent treatment with diflunisal, ursodeoxycholic acid/tauroursodeoxycholate/doxycycline, or TTR lowering agents (eg, patisiran, inotersen)\n- 12. Current or future participation in another investigational device or drug study, scheduled to occur during this study, or has received an investigational agent or device within 30 days (or 5 half-lives of the investigational drug, whichever is longer) prior to dosing (Day 1). In the case of investigational TTR stabilizer drugs, washout for 3 months prior to dosing (Day 1) is required; this does not apply to patients who are on tafamidis at baseline (inclusion criterion #4)\n- 13. Requires treatment with or is unwilling to avoid any concurrent treatment with nondihydropyridine calcium channel blockers (eg, verapamil, diltiazem)\n- 14. Other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to ischemic heart disease (eg, prior myocardial infarction with documented history of cardiac enzymes and ECG changes) that the Investigator feels is a significant contributor or the predominant cause of the patient's heart failure\n- 15. Unstable congestive heart failure (CHF) (including patients who require adjustment of existing diuretics or addition of new diuretics at time of Screening for purposes of achieving optimal management of CHF)\n- 16. Had acute coronary syndrome or unstable angina within the past 3 months\n- 17. Has history of sustained ventricular tachycardia or aborted ventricular fibrillation\n- 18. Has history of atrioventricular nodal or sinoatrial nodal dysfunction for which a pacemaker is indicated but will not be placed"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Composite outcome of all-cause mortality and recurrent CV events (CV hospitalizations and urgent heart failure [HF] visits) in both the overall population and the vutrisiran monotherapy subgroup (defined as patients not on tafamidis at study baseline).","definition_or_measurement_approach":"Composite endpoint defined as all-cause mortality plus recurrent cardiovascular events (cardiovascular hospitalizations and urgent heart failure visits); evaluated in the overall population and in the vutrisiran monotherapy subgroup (patients not on tafamidis at baseline)."}

Secondary endpoints

  • {"endpoint_text":"- Change from baseline in 6-minute walk test (6-MWT)","definition_or_measurement_approach":"Change from baseline in distance (meters) walked on the 6-minute walk test at specified follow-up timepoints."}
  • {"endpoint_text":"- Change from baseline in the Kansas City Cardiomyopathy QuestionnaireOverall Summary (KCCQ-OS)","definition_or_measurement_approach":"Change from baseline in KCCQ Overall Summary score (patient-reported measure of health status/quality of life) at prespecified timepoints."}
  • {"endpoint_text":"- All-cause mortality","definition_or_measurement_approach":"Vital status assessed during study follow-up; death from any cause counted as event."}
  • {"endpoint_text":"- Change from baseline in NYHA Class","definition_or_measurement_approach":"Change from baseline in New York Heart Association functional class as assessed by investigator at scheduled visits."}

Recruitment

Planned Sample Size
250
Recruitment Window Months
72
Consent Approach
Written informed consent is required from each participant (inclusion criterion: participant "is able to understand ... and to provide written informed consent"). Country-specific main ICFs and related ICFs are provided (examples in Croatian, Swedish, Polish, Danish, Spanish, English (IE), Dutch, German, Lithuanian, Portuguese, French and others as indicated in the document list). Separate pregnancy ICFs, pregnancy-partner ICFs and caregiver/partner forms are also provided where applicable. No explicit assent process for minors is specified in the available documents.

Geography

Total Number Of Sites
41
Total Number Of Participants
441

Croatia

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
03-12-2024
Processing Time Days
68
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
University Hospital Centre Zagreb
Department Name
Department for Cardiovascular Diseases
Contact Person Name
Davor Miličić
Contact Person Email
predstojnik.skz@kbc-zagreb.hr

Sweden

Earliest CTIS Part Ii Submission Date
27-09-2024
Latest Decision Or Authorization Date
18-11-2024
Processing Time Days
52
Number Of Sites
2
Number Of Participants
14

Sites

Site Name
University Hospital Of Northern Sweden
Department Name
Hjartcentrum Kliniskt Forskningscentrum
Contact Person Name
Bjorn Pilebro
Site Name
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department Name
Hjartmottagningen Kardiologens Forskningsenhet
Contact Person Name
Entela Bollano
Contact Person Email
entela.bollano@vgregion.se

Poland

Earliest CTIS Part Ii Submission Date
27-09-2024
Latest Decision Or Authorization Date
24-11-2024
Processing Time Days
58
Number Of Sites
2
Number Of Participants
37

Sites

Site Name
Synexus Polska Sp. z o.o.
Department Name
Oddział w Gdańsku
Contact Person Name
Milena Kowalewska-Celejewska
Site Name
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Department Name
Centrum Chorób Serca
Contact Person Name
Ewa Jankowska
Contact Person Email
ewa.jankowska@umed.wroc.pl

Denmark

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
19-11-2024
Processing Time Days
54
Number Of Sites
2
Number Of Participants
30

Sites

Site Name
Odense University Hospital
Department Name
Hjertemedicinsk Afdeling
Contact Person Name
Jens Flensted Lassen
Contact Person Email
jens.flensted.lassen@rsyd.dk
Site Name
Aarhus University Hospital
Department Name
Hjertemedicinsk Afdeling B
Contact Person Name
Steen Poulsen
Contact Person Email
steepoul@rm.dk

Spain

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
19-11-2024
Processing Time Days
54
Number Of Sites
6
Number Of Participants
100

Sites

Site Name
Bellvitge University Hospital
Department Name
Cardiology Department
Contact Person Name
José González Costello
Site Name
Hospital Universitario Juan Ramon Jimenez
Department Name
Cardiology Department
Contact Person Name
Ana Manovel Sánchez
Site Name
Hospital Universitario Puerta De Hierro De Majadahonda
Department Name
Cardiology Department
Contact Person Name
Pablo García Pavia
Contact Person Email
pablogpavia@yahoo.es
Site Name
Hospital Universitario Virgen De La Victoria
Department Name
Cardiology Department
Contact Person Name
José Manuel García Pinilla
Contact Person Email
pinilla@secardiologia.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Internal Medicine Department
Contact Person Name
Fernando Martínez Valle
Site Name
Hospital Universitario Basurto
Department Name
Cardiolog Department
Contact Person Name
Ainara Lozano-Bahamonde

Ireland

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
18-11-2024
Processing Time Days
53
Number Of Sites
2
Number Of Participants
6

Sites

Site Name
St James's Hospital
Department Name
Cardiology
Contact Person Name
Ross Murphy
Contact Person Email
RTMurphy@STJAMES.IE
Site Name
Mater Misericordiae University Hospital
Department Name
Cardiology
Contact Person Name
Niall Mahon
Contact Person Email
Niall.mahon@ucd.ie

Netherlands

Earliest CTIS Part Ii Submission Date
30-09-2024
Latest Decision Or Authorization Date
21-11-2024
Processing Time Days
52
Number Of Sites
2
Number Of Participants
22

Sites

Site Name
Universitair Medisch Centrum Groningen
Department Name
Cardiology
Contact Person Name
Peter van der Meer
Contact Person Email
p.van.der.meer@umcg.nl
Site Name
Universitair Medisch Centrum Utrecht
Department Name
Cardiology
Contact Person Name
Marish Oerlemans
Contact Person Email
m.oerlemans@umcutrecht.nl

Germany

Earliest CTIS Part Ii Submission Date
31-10-2024
Latest Decision Or Authorization Date
22-11-2024
Processing Time Days
22
Number Of Sites
6
Number Of Participants
50

Sites

Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Kardiologie und Angiologie
Contact Person Name
Tienush Rassaf
Contact Person Email
tienush.rassaf@uk-essen.de
Site Name
Universitaet Muenster
Department Name
Herz-MRT-Zentrum, Sektion für Herzbildgebung Klinik für Kardiologie I
Contact Person Name
Ali Yilmaz
Contact Person Email
ali.yilmaz@ukmuenster.de
Site Name
Synexus Clinical Research GmbH
Department Name
Synexus Clinical Research GmbH
Contact Person Name
Katrin Arelin
Contact Person Email
katrin.arelin@globalaes.com
Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Deutsches Zentrum für Herzinsuffizienz Würzburg
Contact Person Name
Caroline Morbach
Contact Person Email
morbach_c@ukw.de
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Klinik für Kardiologie, Angiologie, Pneumologie
Contact Person Name
Arndt Kristen
Site Name
Universitaetsmedizin Goettingen
Department Name
Herzzentrum Göttingen
Contact Person Name
Frauke Czepluch

Austria

Earliest CTIS Part Ii Submission Date
30-09-2024
Latest Decision Or Authorization Date
25-11-2024
Processing Time Days
56
Number Of Sites
2
Number Of Participants
30

Sites

Site Name
Medical University Of Vienna
Department Name
Medizinische Universität Universitätsklinik für Innere Medizin II, Abteilung für Kardiologie
Contact Person Name
Johannes Kastner
Site Name
Stadt Wien Wiener Gesundheitsverbund
Department Name
c/o Klinik Floridsdorf Abteilung für Kardiologie
Contact Person Name
Andreas Schober

Lithuania

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
20-11-2024
Processing Time Days
55
Number Of Sites
1
Number Of Participants
10

Sites

Site Name
Lietuvos sveikatos mokslu universiteto ligonine Kauno klinikos
Department Name
Hospital of Lithuanian University of Health Sciences Kaunas Clinics, Cardiology clinic
Contact Person Name
Egle Ereminiene
Contact Person Email
eglerem@yahoo.com

Belgium

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
19-11-2024
Processing Time Days
54
Number Of Sites
4
Number Of Participants
9

Sites

Site Name
O.L.Vrouw van Troost
Department Name
Cardiology
Contact Person Name
Tom Sarens
Contact Person Email
tomsarens@hotmail.com
Site Name
Hopital Erasme
Department Name
Cardiology
Contact Person Name
Antoine Bondue
Contact Person Email
antoine.bondue@hubruxelles.be
Site Name
Universitair Ziekenhuis Gent
Department Name
Cardiology
Contact Person Name
Michel De Pauw
Contact Person Email
michel.depauw@ugent.be
Site Name
UZ Leuven
Department Name
Cardiology
Contact Person Name
Johan Van Cleemput
Contact Person Email
johan.vancleemput@uzleuven.be

Hungary

Earliest CTIS Part Ii Submission Date
01-10-2024
Latest Decision Or Authorization Date
20-11-2024
Processing Time Days
50
Number Of Sites
1
Number Of Participants
30

Sites

Site Name
Semmelweis University
Department Name
Belgyógyászati és Hematológiai Klinika
Contact Person Name
Zoltán POZSONYI
Contact Person Email
pozsonyizoltanimre@gmail.com

Portugal

Earliest CTIS Part Ii Submission Date
01-10-2024
Latest Decision Or Authorization Date
18-11-2024
Processing Time Days
48
Number Of Sites
3
Number Of Participants
40

Sites

Site Name
Unidade Local De Saude Do Alto Ave E.P.E.
Department Name
Cardiology Deparment
Contact Person Name
Olga Azevedo
Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Cardiology Deparment
Contact Person Name
Patricia Rodrigues
Contact Person Email
pfdrodrigues@gmail.com
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Cardiology Deparment
Contact Person Name
Dulce Brito
Contact Person Email
dulcebrito59@gmail.com

Norway

Earliest CTIS Part Ii Submission Date
27-09-2024
Latest Decision Or Authorization Date
22-11-2024
Processing Time Days
56
Number Of Sites
1
Number Of Participants
20

Sites

Site Name
Oslo University Hospital HF
Department Name
Oslo Universitetssykehus HF Rikshospitalet, Kardiologisk Forskning
Contact Person Name
Kaspar Broch
Contact Person Email
sbbrok@ous-hf.no

Czechia

Earliest CTIS Part Ii Submission Date
26-09-2024
Latest Decision Or Authorization Date
20-11-2024
Processing Time Days
55
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
University Hospital Olomouc
Department Name
I. interní klinika - kardiologická
Contact Person Name
Renáta Aiglová
Contact Person Email
renata.aiglova@fnol.cz
Site Name
Synexus Czech s.r.o.
Contact Person Name
Iva Škarpová
Contact Person Email
iva.skarpova@synexus.com

France

Earliest CTIS Part Ii Submission Date
25-09-2024
Latest Decision Or Authorization Date
19-11-2024
Processing Time Days
55
Number Of Sites
4
Number Of Participants
36

Sites

Site Name
Assistance Publique Hopitaux De Paris (Creteil)
Department Name
Cardiology
Contact Person Name
Thibaud DAMY
Contact Person Email
thibaud.damy@aphp.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Cardiology
Contact Person Name
Pauline Fournier
Contact Person Email
fournier.p@chu-toulouse.fr
Site Name
Assistance Publique Hopitaux De Paris (Paris)
Department Name
Cardiology
Contact Person Name
Vincent Algalarrondo
Contact Person Email
vincent.algalarrondo@aphp.fr
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Cardiology
Contact Person Name
Gilbert Habib
Contact Person Email
gilbert.habib@ap-hm.fr

Sponsor

Primary sponsor

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

Contract research organisations

Name
PPD Development LP
Responsibilities
Multiple operational roles (codes:1,12,2,5,8,9)
Name
Syneos Health Inc.
Responsibilities
Operational support (code:8)
Name
IQVIA Biotech LLC
Responsibilities
Operational support / services (code:10)

Third parties

  • {"country":"United Kingdom","full_name":"Medical Research Network Limited","duties_or_roles":"Homecare Nursing Services","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"The Brigham And Women’s Hospital Inc.","duties_or_roles":"Cardiac Imaging Core Lab (ECHO), Central ECHO Analysis","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Omnitrace Corp.","duties_or_roles":"Support collection of survival status information for subjects (primary endpoint)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"Patient Travel & Reimbursement Services","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Fisher Clinical Services Inc.","duties_or_roles":"IP Packaging, Labeling, Storage and Distribution","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"EPL Pathology Archives LLC","duties_or_roles":"Long-term Genetic Sample Storage","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Multiple operational roles (codes:1,12,2,5,8,9)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central ECG Reading, Central ECG Analysis","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Quanterix Corp.","duties_or_roles":"neurofilament analysis","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"code:8","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"IRT (IXRS)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Cisys Inc.","duties_or_roles":"electronic Adjudication System","organisation_type":"Pharmaceutical company"}
  • {"country":"Canada","full_name":"Charles River Laboratories Montreal ULC","duties_or_roles":"Labs analysis TTR and ADA; code:4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Pyxant Labs Inc.","duties_or_roles":"PK Analysis","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Colorado Prevention Center","duties_or_roles":"6-MWT Oversight","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Iqvia Biotech LLC","duties_or_roles":"code:10","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"SGS Belgium","duties_or_roles":"Randomization List; codes:6,7","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Amvuttra 25 mg solution for injection in pre-filled syringe
Active Substance
Vutrisiran
Modality
Oligonucleotide
Routes Of Administration
Subcutaneous
Route
Subcutaneous
Authorisation Status
Authorised (EU marketing authorisation EU/1/22/1681/001)
Orphan Designation
Yes
Starting Dose
25 mg
Dose Levels
25 mg
Frequency
Every 12 weeks (once every 3 months)
Maximum Dose
550 mg (maxTotalDoseAmount 550 mg)
Investigational Product Name
0.9% Sodium chloride
Modality
Other
Routes Of Administration
Subcutaneous (placebo administered SC)
Route
Subcutaneous
Authorisation Status
Not applicable
Frequency
Every 12 weeks (placebo dosing matches active arm schedule)

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