Clinical trial • Phase III • Cardiology

EPLONTERSEN for Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM)

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

Overview

Trial Therapeutic Area
Cardiology
Trial Disease
Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM)
Trial Stage
Phase III
Drug Modality
Oligonucleotide
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
22-08-2024
First CTIS Authorization Date
17-09-2024

Trial design

Randomised, ion-682884: subcutaneous injection once every 4 weeks; placebo injection, 0.8 ml subcutaneous once every 4 weeks-controlled, adaptive Phase III trial in Belgium, Portugal, Denmark and others.

Randomised
Yes
Comparator
ION-682884: subcutaneous injection once every 4 weeks; Placebo injection, 0.8 ml subcutaneous once every 4 weeks
Adaptive
True, protocol describes blinded assessments in advance of when the last patient randomized reaches Week 61 and every 12 weeks thereafter to project study power and to determine whether to stop the study early for final analysis (interim blinded assessments that may lead to early stop for final analysis).
Target Sample Size
910
Trial Duration For Participant
1120

Eligibility

Recruits 910 Vulnerable population selection is indicated (isVulnerablePopulationSelected: true). Local informed consent materials include LAR (legally authorised representative) forms (e.g. L1_DK_SIS-ICF_LAR_Danish) and survival/vital status addenda (e.g. Survival Consent Addendum, Vital Status ICF Addendum) indicating provisions for consent by legally authorised representatives where applicable. Subject information and consent forms are provided in multiple country-specific languages and with addenda for partner/pregnancy and vital status, showing tailored consent handling for vulnerable participants..

Pregnancy Exclusion
Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method
Vulnerable Population
Vulnerable population selection is indicated (isVulnerablePopulationSelected: true). Local informed consent materials include LAR (legally authorised representative) forms (e.g. L1_DK_SIS-ICF_LAR_Danish) and survival/vital status addenda (e.g. Survival Consent Addendum, Vital Status ICF Addendum) indicating provisions for consent by legally authorised representatives where applicable. Subject information and consent forms are provided in multiple country-specific languages and with addenda for partner/pregnancy and vital status, showing tailored consent handling for vulnerable participants.

Inclusion criteria

  • {"criterion_text":"- Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method"}
  • {"criterion_text":"- Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participant's non-pregnant female partner must be using a highly effective contraceptive method"}
  • {"criterion_text":"- Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy (99mTc -3,3- diphosphono-1,2- propanodicarboxylic acid [DPD-Tc], 99m Tc- pyrophosphate [PYP-Tc], or 99mTc-hydroxymethylene-diphosphonate [HMDP-Tc]) with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, centrally confirmed"}
  • {"criterion_text":"- End-diastolic interventricular septum thickness of > 12 mm on Screening echocardiogram"}
  • {"criterion_text":"- New York Heart Association (NYHA) class I-III"}

Exclusion criteria

  • {"criterion_text":"- Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening"}
  • {"criterion_text":"- Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease"}
  • {"criterion_text":"- Monoclonal gammopathy of undetermined significance (MGUS) and/or alterations in immunoglobulin free light chain (FLC) ratio, unless fat, bone marrow, or heart biopsy confirming the absence of light chain and the presence of TTR protein by mass spectrometry or immunoelectron microscopy. For patients with CKD and without presence of monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26-2.25. Results different from that may be discussed with local hematologist, Investigator and Medical Monitor if the risks associated with the biopsy outweigh the benefits"}
  • {"criterion_text":"- Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization"}
  • {"criterion_text":"- Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or other oligonucleotide or RNA therapeutic (including siRNA; does not apply to COVID-19 mRNA vaccinations)"}
  • {"criterion_text":"- Current treatment with diflunisal, doxycycline, with or without ursodeoxycholic acid, and/or non-dihydropyridine calcium-channel blocker (e.g.,verapamil, diltiazem). Patients receiving any of these agents must respect a Wash-out Period of 14 days before randomization."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Composite endpoint of CV-death and recurrent CV clinical events comparing the 2 study arms up to Week 140. Cardiovascular clinical events include: 1. Hospitalization for myocardial infarction (MI)","definition_or_measurement_approach":"Composite assessed up to Week 140; Andersen-Gill method mentioned for composite; centrally adjudicated cardiovascular clinical events (includes hospitalization for MI) comparing ION-682884 vs placebo."}
  • {"endpoint_text":"- Hospitalization for HF","definition_or_measurement_approach":"Hospitalization events for heart failure counted as part of composite endpoint up to Week 140."}
  • {"endpoint_text":"- Hospitalization for arrhythmia","definition_or_measurement_approach":"Hospitalization events for arrhythmia counted as part of composite endpoint up to Week 140."}
  • {"endpoint_text":"- Hospitalization for stroke/Transient Ischemic Attack TIA","definition_or_measurement_approach":"Hospitalization events for stroke/TIA counted as part of composite endpoint up to Week 140."}
  • {"endpoint_text":"- HF urgent visits to ED/ER or HF clinics requiring administration of intravenous (IV) diuretics for improvement.","definition_or_measurement_approach":"Urgent/unplanned heart-failure-related visits requiring IV diuretics are counted as CV clinical events in the composite up to Week 140."}

Secondary endpoints

  • {"endpoint_text":"- Change From Baseline in the 6-minute Walk Test (6MWT) Distance at Week 140","definition_or_measurement_approach":"Change from baseline in 6MWT distance measured at Week 140."}
  • {"endpoint_text":"- Change From Baseline in the Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores at Week 140","definition_or_measurement_approach":"Change from baseline in KCCQ scores measured at Week 140."}
  • {"endpoint_text":"- Recurrent CV Clinical Events up to Week 140","definition_or_measurement_approach":"Counting recurrent cardiovascular clinical events up to Week 140 (components consistent with primary composite)."}
  • {"endpoint_text":"- All-Cause Mortality up to Week 160","definition_or_measurement_approach":"All-cause mortality assessed up to Week 160."}
  • {"endpoint_text":"- All-Cause Mortality up to Week 140","definition_or_measurement_approach":"All-cause mortality assessed up to Week 140."}
  • {"endpoint_text":"- Primary endpoint in subgroup of patients treated with tafamidis at Baseline","definition_or_measurement_approach":"Primary composite endpoint evaluated within the subgroup of patients on tafamidis at baseline."}
  • {"endpoint_text":"- CV Mortality up to Week 160","definition_or_measurement_approach":"Cardiovascular cause mortality assessed up to Week 160."}
  • {"endpoint_text":"- CV Mortality up to Week 140","definition_or_measurement_approach":"Cardiovascular cause mortality assessed up to Week 140."}

Recruitment

Registry Or Advocacy Recruitment
True, Fondazione Toscana Gabriele Monasterio
Digital Remote Recruitment
True, use of Scout Clinical email communications and digital patient contact/agreements (Scout Email Communication, Scout Patient Agreement) and use of patient locator service (Omnitrace) to identify/communicate with potential participants
Planned Sample Size
910
Recruitment Window Months
77
Consent Approach
Informed consent is obtained via country- and language-specific subject information and informed consent forms (multiple L1_... SIS-ICF documents in Dutch, French, Portuguese, Danish, Polish, Greek, Spanish, German, Czech, Italian, Swedish etc.). Addenda are provided for partner/pregnancy and vital status, and survival consent addenda are available. Legal/legally authorised representative (LAR) consent forms are available in some countries (e.g. L1_DK_SIS-ICF_LAR_Danish), indicating provision for consent by LAR where applicable. ICFs and relevant addenda are provided per site/country language versions.

Methods

  • Site-based recruitment at participating hospitals/clinics listed in Part II submissions (site contact details available per country)
  • Use of a Patient Locator Service (Omnitrace Corp.) as documented in sponsor third-party duties
  • Scout Clinical patient agreements and email communications (documents: Scout Clinical Patient Agreement, Scout Email Communication) to support participant contact and logistics
  • Country-specific 'Recruitment Arrangements' documents submitted for publication (K_* Recruitment Arrangements placeholders present per Member State)

Geography

Total Number Of Sites
40
Total Number Of Participants
413

Belgium

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
26-05-2025
Processing Time Days
264
Number Of Sites
2
Number Of Participants
20

Sites

Site Name
Az St-Jan Brugge-Oostende A.V.
Department Name
Valvular & structural heart disease, cardiomyopathies
Contact Person Name
Philippe Debonnaire
Site Name
Ziekenhuis Oost Limburg
Department Name
Department of Cardiology
Contact Person Name
Matthias Dupont
Contact Person Email
matthias.dupont@zol.be

Portugal

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
06-03-2026
Processing Time Days
548
Number Of Sites
4
Number Of Participants
40

Sites

Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Cardiology
Contact Person Name
André Luz
Site Name
Unidade Local De Saude De Sao Jose E.P.E.
Department Name
Cardiology
Contact Person Name
Sílvia Rosa
Contact Person Email
silvia.rosa@chlc.min-saude.pt
Site Name
Unidade Local de Saude de Sao Joao E.P.E.
Department Name
Cardiology
Contact Person Name
Elisabete Martins
Site Name
Unidade Local De Saude Do Alto Ave E.P.E.
Department Name
Cardiology
Contact Person Name
Olga Azevedo

Denmark

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
03-03-2026
Processing Time Days
545
Number Of Sites
1
Number Of Participants
20

Sites

Site Name
Rigshospitalet
Department Name
Department of Cardiology
Contact Person Name
Kasper Rossing
Contact Person Email
kasper.rossing@regionh.dk

Poland

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
10-03-2026
Processing Time Days
552
Number Of Sites
2
Number Of Participants
20

Sites

Site Name
Krakowski Szpital Specjalistyczny Im. Sw. Jana Pawla II
Department Name
Oddzial Kliniczny Chorob Serca i Naczyn z Pododdzialem Intensywnego Nadzoru Kardilogicznego
Contact Person Name
Piotr Podolec
Contact Person Email
p.podolec@szpitaljp2.krakow.pl
Site Name
Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego Panstwowy Instytut Badawczy
Department Name
Oddział Kardiomiopatii
Contact Person Name
Jacek Grzybowski
Contact Person Email
jgrzybowski@ikard.pl

France

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
03-03-2026
Processing Time Days
545
Number Of Sites
6
Number Of Participants
75

Sites

Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Centres d'Investigation Clinique à thématique Cardio-Vasculaire
Contact Person Name
Patricia Reant
Contact Person Email
patricia.reant@chu-bordeaux.fr
Site Name
Institut De Cancerologie De L Ouest
Department Name
Centre d'investigation Clinique Thorax (équipe Cardiovasculaire
Contact Person Name
Nicolas Piriou
Contact Person Email
nicolas.piriou@chu-nantes.fr
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Cardiologie
Contact Person Name
Gilbert Habib
Contact Person Email
gilbert.habib@ap-hm.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Cardiology
Contact Person Name
Eve Cariou
Contact Person Email
cariou.e@chu-toulouse.fr
Site Name
Centre Hospitalier Departemental Vendee
Department Name
Cardiologie, Chirurgie digestive et Hépato-gastro-entérologie
Contact Person Name
Fabien Miette
Contact Person Email
fabien.miette@ght85.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Cardiologie
Contact Person Name
Thibaud Damy
Contact Person Email
thibaud.damy@aphp.fr

Greece

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
09-03-2026
Processing Time Days
551
Number Of Sites
1
Number Of Participants
23

Sites

Site Name
Alexandra Hospital
Department Name
Clinical Therapeutics Department
Contact Person Name
Efstathios Kastritis
Contact Person Email
ekastritis@gmail.com

Spain

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
06-03-2026
Processing Time Days
548
Number Of Sites
4
Number Of Participants
10

Sites

Site Name
University Clinical Hospital Virgen De La Arrixaca
Department Name
Cardiology
Contact Person Name
Domingo Pascual-Figal
Contact Person Email
dpascual@um.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Cardiology
Contact Person Name
Jose Fernando Rodriguez Palomares
Contact Person Email
jfrodriguezpalomares@gmail.com
Site Name
Hospital Universitario Puerta De Hierro De Majadahonda
Department Name
Cardiology
Contact Person Name
Pablo Garcia Pavia
Contact Person Email
pablogpavia@cnic.es
Site Name
Complexo Hospitalario Universitario De Santiago
Department Name
Cardiology
Contact Person Name
Alfonso Varela Román
Contact Person Email
alfonso.varela.roman@sergas.es

Germany

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
09-03-2026
Processing Time Days
551
Number Of Sites
5
Number Of Participants
35

Sites

Site Name
Universitaetsklinikum Muenster AöR
Department Name
Herz-MRT-Zentrum Klinik für Kardiologie I
Contact Person Name
Ali Yilmaz
Contact Person Email
ali.yilmaz@ukmuenster.de
Site Name
Universitaetsklinikum des Saarlandes AöR
Department Name
Innere Medizin III
Contact Person Name
Ingrid Kindermann
Contact Person Email
Ingrid.Kindermann@uks.eu
Site Name
University Hospital Cologne AöR
Department Name
Department III für Innere Medizin
Contact Person Name
Henrik ten Freyhaus
Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Deutsches Zentrum für Herzinsuffizienz
Contact Person Name
Stefan Störk
Contact Person Email
stoerk_s@ukw.de
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Internal Medicine III
Contact Person Name
Fabian aus dem Siepen

Czechia

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
09-03-2026
Processing Time Days
551
Number Of Sites
3
Number Of Participants
30

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
II. Internal Clinic - Clinic of Cardiology and Angiology
Contact Person Name
Tomáš Paleček
Contact Person Email
tpalec@lf1.cuni.cz
Site Name
Fakultni Nemocnice U Sv Anny V Brne
Department Name
I. Internal Clinic of Cardiology
Contact Person Name
Jan Krejčí
Contact Person Email
jan.krejci@fnusa.cz
Site Name
Institute For Clinical And Experimental Medicine
Department Name
Clinic of Cardiology
Contact Person Name
Miloš Kubánek
Contact Person Email
milos.kubanek@ikem.cz

Austria

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
11-03-2026
Processing Time Days
553
Number Of Sites
3
Number Of Participants
50

Sites

Site Name
Medizinische Universitaet Innsbruck
Department Name
Universitätsklinik für Innere Medizin III – Kardiologie und Angiology
Contact Person Name
Gerhard Pölzl
Site Name
Medical University Of Graz
Department Name
Department of Internal Medicine, Division of Cardiology
Contact Person Name
Klemens Ablasser
Contact Person Email
klemens.ablasser@medunigraz.at
Site Name
Medical University Of Vienna
Department Name
Clinical Department for Cardiology
Contact Person Name
Johannes Kastner

Sweden

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
05-03-2026
Processing Time Days
547
Number Of Sites
2
Number Of Participants
30

Sites

Site Name
Region Vaesterbotten
Department Name
Dept. of Internal Medicine
Contact Person Name
Kurt Boman
Site Name
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department Name
Department of Cardiology
Contact Person Name
Entela Bollano
Contact Person Email
entela.bollano@vgregion.se

Italy

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
11-03-2026
Processing Time Days
553
Number Of Sites
7
Number Of Participants
60

Sites

Site Name
Careggi University Hospital
Department Name
Cardio Toraco Vascolare - Interventistica Cardiologica Strutturale
Contact Person Name
Francesco Cappelli
Contact Person Email
cappellifrancesco@inwind.it
Site Name
Azienda Ospedaliera di Padova
Department Name
Cardiologia
Contact Person Name
Alberto Cipriani
Contact Person Email
alberto.cipriani@unipd.it
Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
Cardiologia - UTIC
Contact Person Name
Beatrice Musumeci
Contact Person Email
beatrice.musumeci@uniroma1.it
Site Name
Azienda Ospedaliero Universitaria Delle Marche
Department Name
SOD Cardiologia Emodinamica e UTIC
Contact Person Name
Marini Marco
Site Name
Fondazione Toscana Gabriele Monasterio
Department Name
Cardiotoracico
Contact Person Name
Michele Emdin
Contact Person Email
emdin@ftgm.it
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scienti
Department Name
Cardiologia
Contact Person Name
Galiè Nazzareno
Contact Person Email
nazzareno.galie@unibo.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
SC Medicina Generale 2- Centro Amiloidosi Sistemiche e Malattie ad Alta Complessitá
Contact Person Name
Laura Obici
Contact Person Email
l.obici@smatteo.pv.it

Sponsor

Primary sponsor

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

Contract research organisations

Name
PRA Hellas CRO A.E.
Responsibilities
Medical monitoring, Managing vendors, Non-IP Clin.Supplies, PV and other trial support activities
Name
Icon Clinical Research Limited
Responsibilities
Medical monitoring, Managing vendors, Non-IP Clin.Supplies, PV
Name
Parexel International Co. Ltd.
Responsibilities
CRO services (duties code 10 listed)

Third parties

  • {"country":"United States","full_name":"Versiti Wisconsin Inc.","duties_or_roles":"Platelet Autoantibodies","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Medical image analysis/ review - X-ray, MRI, ultrasound, etc.","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Germany","full_name":"Inceptua GmbH","duties_or_roles":"Vitamin A procurement","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PRA Hellas CRO A.E.","duties_or_roles":"Medical monitoring, Managing vendors, Non-IP Clin.Supplies, PV (and other listed duties)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"The Brigham And Women’s Hospital Inc.","duties_or_roles":"Medical image analysis/ review - X-ray, MRI, ultrasound, etc.","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Omnitrace Corp.","duties_or_roles":"Patient Locator Service","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Mycardium AI Limited","duties_or_roles":"Image central reader","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"Home Healthcare","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"BioClinica GmbH","duties_or_roles":"Image central reader","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"MEDPACE LABORATORIES","duties_or_roles":"HIV (Quest), Platelet Testing (ARUP), PK samples management","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"Duties code 14 (unspecified in data)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Biologics Development Services LLC","duties_or_roles":"testing TTR as part of PD panel","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Medical monitoring, Managing vendors, Non-IP Clin.Supplies, PV","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Travel Reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Canada","full_name":"Charles River Laboratories Montreal ULC","duties_or_roles":"Specialty lab","organisation_type":"Pharmaceutical company"}
  • {"country":"Taiwan","full_name":"Parexel International Co. Ltd.","duties_or_roles":"Duties code 10 (unspecified in data)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"Duties code 3 (unspecified in data)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Duke Clinical Research Institute","duties_or_roles":"Adjudication","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Duke Clinical And Translational Science Institute","duties_or_roles":"Primary/ surrogate endpoint test; Cardiovascular Event Adjudication Committee","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United Kingdom","full_name":"Medical Research Network Limited","duties_or_roles":"Home Healthcare","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Duties code 7 (unspecified in data)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"PK","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
ION 682884
Active Substance
EPLONTERSEN
Modality
Oligonucleotide
Routes Of Administration
SUBCUTANEOUS USE
Route
Subcutaneous
Authorisation Status
prodAuthStatus: 1
Orphan Designation
Yes
Frequency
Once every 4 weeks (SC every 4 weeks)
Maximum Dose
1575 mg (maxTotalDoseAmount: 1575 mg)
Investigational Product Name
Placebo injection, 0.8 ml (is a sterile, preservative-free, parenteral solution of sodium chloride and riboflavin in Water for Injection) for subcutaneous administration
Modality
Other
Routes Of Administration
Subcutaneous
Route
Subcutaneous
Frequency
Once every 4 weeks (matching ION-682884 schedule)

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