Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruiting
Participants planned
1,254
Countries
12
Sites
38
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
To evaluate the safety and tolerability of extended dosing with eplontersen in subjects with ATTR-CM.
Key facts
- Sponsor
- Ionis Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Cardiovascular Diseases [C14]
- Trial duration
- 16 Jun 2023 → ongoing
- Decision date (initial)
- 2023-06-05
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Ionis Pharmaceuticals, Inc
External identifiers
- EU CT number
- 2022-502415-11-00
- WHO UTN
- U1111-1285-6834
- ClinicalTrials.gov
- NCT05667493
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
To evaluate the safety and tolerability of extended dosing with eplontersen in subjects with ATTR-CM.
Secondary objectives 1
- To evaluate parameters of efficacy following extended dosing with eplontersen in subjects with ATTR-CM within the overall population and within subgroups based on treatment allocation during the Index Study.
Conditions and MedDRA coding
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | LLT | 10002020 | Amyloid cardiomyopathy | 10007541 |
Regulatory references
- Scientific advice from competent authorities
- European Medicines Agency
- Plan to share IPD
- Yes
- IPD plan description
- Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-514434-20-00 | A Phase 3 Global, Double-Blind, Randomized, Placebo‑Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin‑Mediated Amyloid Cardiomyopathy (ATTR-CM) | Ionis Pharmaceuticals Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- Satisfactory completion of the Treatment Period and the End of Treatment Visit of the Index Study (ION‑682884-CS2) OR Diagnosis of ATTR-CM and satisfactory participation on ISIS 420915CS101 study, as judged by the Investigator and Sponsor
- Investigator is willing to treat the subject with open label ION-682884 (eplontersen)
- Willingness to adhere to vitamin A supplementation per protocol
Exclusion criteria 2
- Permanently discontinued Study Drug administration while participating in the Index Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study)
- Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the subject unsuitable for enrollment, or which could interfere with the subject participating in or completing the study, including need for treatment with medications disallowed in the Index Study
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Description of TEAEs (AE/SAE/AESI)
- Analysis of change in safety laboratory assessments of interest over time (Platelet count, eGFR, UPCR, LFTs)
Secondary endpoints 1
- Analysis of change in clinical and Biomarker assessment of interest and survival: •TTR serum levels •6MWT •Kansas City Cardiomyopathy Questionnaire (KCCQ) • CV-death •All-cause death
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
PRD7488479 · Product
- Active substance
- Eplontersen
- Other product name
- Transthyretin Antisense Oligonucleotide
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 45 mg milligram(s)
- Max total dose
- 3240 mg milligram(s)
- Max treatment duration
- 72 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- IONIS PHARMACEUTICALS, INC.
- Paediatric formulation
- No
- Orphan designation
- No
PRD10199021 · Product
- Active substance
- Eplontersen
- Pharmaceutical form
- INJECTION
- Route of administration
- SOLUTION FOR INJECTION IN PRE-FILLED SYRINGE
- Max daily dose
- 45 mg/g milligram(s)/gram
- Max total dose
- 3240 mg milligram(s)
- Max treatment duration
- 72 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- IONIS PHARMACEUTICALS, INC.
- Paediatric formulation
- No
- Orphan designation
- No
Auxiliary 1
PRD1171071 · Product
- Active substance
- Ergocalciferol Bp
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL USE
- Max daily dose
- 3000 IU international unit(s)
- Max total dose
- 6570000 IU international unit(s)
- Max treatment duration
- 72 Month(s)
- Authorisation status
- Authorised
- ATC code
- A11BA — MULTIVITAMINS, PLAIN
- Marketing authorisation
- PL00400/5009R
- MA holder
- WALLACE MANUFACTURING CHEMISTS LTD
- MA country
- XI
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Ionis Pharmaceuticals Inc.
- Sponsor organisation
- Ionis Pharmaceuticals Inc.
- Address
- 2855 Gazelle Court
- City
- Carlsbad
- Postcode
- 92010-6670
- Country
- United States
Scientific contact point
- Organisation
- Ionis Pharmaceuticals Inc.
- Contact name
- Global Regulatory Affairs
Public contact point
- Organisation
- Ionis Pharmaceuticals Inc.
- Contact name
- Global Regulatory Affairs
Third parties 14
| Organisation | City, country | Duties |
|---|---|---|
| Charles River Laboratories Montreal ULC ORG-100041009
|
Senneville, Canada | Other |
| Scout Clinical ORG-100042228
|
Dallas, United States | Other |
| Inceptua GmbH ORG-100033479
|
Berlin, Germany | Other |
| Versiti Wisconsin Inc. ORG-100044223
|
Milwaukee, United States | Other |
| Brigham and Women's Hospital ORL-000000453
|
Boston, United States | Other |
| PPD Development LP ORG-100011560
|
Richmond, United States | Other |
| Icon Clinical Research Limited ORG-100008322
|
Dublin 18, Ireland | On site monitoring, Code 12, Other, Code 2, Data management, E-data capture, Code 8 |
| Mycardium AI Limited ORG-100049567
|
Liverpool, United Kingdom | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| Immunologix ORL-000000464
|
Tampa, United States | Other, Laboratory analysis |
| Almac Clinical Services LLC ORG-100041692
|
Durham, United States | Code 14 |
| BioClinica GmbH ORG-100032790
|
Munich, Germany | Other |
| MEDPACE LABORATORIES ORG-100042942
|
Leuven, Belgium | Laboratory analysis |
| PRA Hellas CRO A.E. ORG-100048208
|
Nea Ionia, Greece | On site monitoring, Code 12, Other, Code 2, Data management, E-data capture, Code 8 |
Locations
12 EU/EEA countries · 38 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ongoing, recruiting | 24 | 3 |
| Belgium | Ongoing, recruitment ended | 21 | 2 |
| Czechia | Ongoing, recruiting | 21 | 3 |
| Denmark | Ongoing, recruiting | 11 | 1 |
| France | Ongoing, recruiting | 41 | 6 |
| Germany | Ongoing, recruiting | 50 | 5 |
| Greece | Ongoing, recruiting | 13 | 1 |
| Italy | Ongoing, recruiting | 47 | 6 |
| Poland | Ongoing, recruiting | 8 | 1 |
| Portugal | Ongoing, recruiting | 23 | 4 |
| Spain | Ongoing, recruiting | 59 | 4 |
| Sweden | Ongoing, recruiting | 21 | 2 |
| Rest of world
Japan, Australia, Israel, United Kingdom, Brazil, Argentina, United States, Canada
|
— | 915 | — |
Investigational sites
Medizinische Universitaet Innsbruck
Universitätsklinik für Innere Medizin III - Kardiologie und Angiologie, Anichstrasse 35, 6020, Innsbruck
Medical University Of Graz
Department of Cardiology, Neue Stiftingtalstrasse 6, 8010, Graz
Medical University Of Vienna
Clinical Department for Cardiology, Waehringer Guertel 18-20, Alsergrund, Vienna
Az St-Jan Brugge-Oostende A.V.
Cardiology, Ruddershove 10, 8000, Brugge
Ziekenhuis Oost Limburg
Cardiology, Synaps Park 1, 3600, Genk
Fakultni Nemocnice U Sv Anny V Brne
I. Internal cardioangiological clinic, Pekarska 53, Stare Brno, Brno-Stred
Vseobecna Fakultni Nemocnice V Praze
II. Department of internal Medicine – Department of Cardiology and Angiology, U Nemocnice 499/2, Nove Mesto, Prague 2
Institute For Clinical And Experimental Medicine
Cardiology, Videnska 1958/9 Krc, 140 00, Prague
Rigshospitalet
Department of Cardiology, Blegdamsvej 9, 2100, Copenhagen Oe
Assistance Publique Hopitaux De Paris
Cardiology, 51 Av Du Mal De Lattre De Tassigny, 94000, Creteil
Centre Hospitalier Universitaire De Nantes
Cardiology, Boulevard Du Professeur Jacques Monod, 44800, Saint Herblain
Assistance Publique Hopitaux De Marseille
Cardiology and Heart failure, 264 Rue Saint Pierre, 13005, Marseille
Centre Hospitalier Departemental Vendee
Cardiology, Boulevard Stephane Moreau, 85925, La Roche Sur Yon Cedex 9
Centre Hospitalier Universitaire De Bordeaux
UMCV, Avenue De Magellan, 33600, Pessac
Centre Hospitalier Universitaire De Toulouse
Cardiology, 1 Avenue Du Professeur Jean Poulhes, Tsa 50032, Toulouse Cedex 9
Westfaelische Wilhelms Universitaet Muenster
Department of Cardiology, Division of Cardiovascular Imaging, Von-Esmarch-Strasse 48, 48149, Munster
University Of Saarland
Department of Medicine III, Kirrberger Strasse, 66421, Homburg
Universitaetsklinikum Heidelberg AöR
Clinic for Cardiology, Angiology and Pneumology, Im Neuenheimer Feld 410, Neuenheim, Heidelberg
Universitatsklinikum Wurzburg AöR
Department of Clinical Research and Epidemiology, Comprehensive Heart Failure Center, Am Schwarzenberg 15, Lindleinsmuehle, Wuerzburg
University Hospital Cologne AöR
Clinic of Internal Medicine III, Kerpener Strasse 62, Lindenthal, Cologne
Alexandra Hospital
Department of Clinical TherapeuticsPlasma Cell Dyscrasias Unit, Vassilissas Sofias Avenue 80, 115 28, Athens
Azienda Ospedale-Universita Padova
Cardio-Toraco-Vascolare e Sanità Pubblica, Via Nicolo' Giustiniani 2, 35128, Padova
Careggi University Hospital
Health and vessels department, Largo Giovanni Alessandro Brambilla 3, 50134, Florence
Fondazione IRCCS Policlinico San Matteo
U.O.C. Medicina Gen2 Centro Amiloidosi Sistemiche e Malattie ad Alta Complessità, Viale Camillo Golgi 19, 27100, Pavia
Azienda Ospedaliero-Universitaria Sant Andre
Clinical and Molecolare Medicine, Via Di Grottarossa 1035-1039, 00189, Rome
Azienda Ospedaliero Universitaria Ospedali Riuniti Ancona
Dipartimento di Scienze Cardiovascolari, SOD Cadiologia Ospedaliera e UTIC, Via Conca 71, 60126, Ancona
Fondazione Toscana Gabriele Monasterio
U.O.C. Cardiologia e Medicina Cardiovascolare, Via Trieste 41, 56126, Pisa
Krakowski Szpital Specjalistyczny Im. Sw. Jana Pawla II
Oddział Kliniczny Chorób Serca i Naczyń z Pododdziałem Intensywnego adzoru Kardiologicznego, Ul. Pradnicka 80, 31-202, Cracow
Centro Hospitalar Universitario De Santo Antonio E.P.E.
Cardiology, Largo Professor Abel Salazar, 4050-011, Porto
Centro Hospitalar Universitario Lisboa Central E.P.E.
Cardiology, Rua de Santa Marta 50, 1169-024, Lisbon
Hospital Da Senhora Da Oliveira Guimaraes E.P.E.
Cardiology, Rua Dos Cuteleiros De Guimaraes, 4835-044, Guimaraes
Sao Joao University Hospital Center
Cardiology, Alameda Professor Hernani Monteiro, 4200-319, Porto
Hospital Universitario Puerta De Hierro De Majadahonda
Cardiology, Calle De Manuel De Falla 1, 28222, Majadahonda
University Clinical Hospital Virgen De La Arrixaca
Cardiology, Carretera De Cartagena S/n, El Palmar, Murcia
Hospital Universitari Vall D Hebron
Cardiology, Edificio Materno-Infantil, Passeig De La Vall D'hebron 119-129, Barcelona
Complexo Hospitalario Universitario De Santiago
Cardiology, Calle Choupana Da S/n, 15706, Santiago De Compostela
Region Vasterbotten
Medicinsk geriatrisk klinik, Skellefteå lasarett, Forskningsmott Plan 2 Lasarettet, 931 86, Skellefteå
Västra Götalandregionen
Sahlgrenska Universitetssjukhu aet Kardiologen, Blå stråket 5, Kardiologens Forskningsenhet, Göteborg
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Austria | 2023-08-01 | 2023-09-01 | |||
| Belgium | 2023-07-12 | 2023-08-22 | 2025-05-07 | ||
| Czechia | 2024-02-23 | 2024-07-09 | |||
| Denmark | 2024-03-04 | 2024-05-23 | |||
| France | 2024-02-08 | 2024-04-11 | |||
| Germany | 2023-08-01 | 2023-09-18 | |||
| Greece | 2023-08-29 | 2023-11-20 | |||
| Italy | 2023-07-26 | 2023-09-21 | |||
| Poland | 2025-04-14 | 2025-04-18 | |||
| Portugal | 2023-06-16 | 2023-07-27 | |||
| Spain | 2023-11-23 | 2024-02-19 | |||
| Sweden | 2023-08-16 | 2023-11-02 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 116 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol Clarification Letter_2022-502415-11_Greek_Redacted | 1.0 |
| Protocol (for publication) | D1_Protocol Clarification Letter_2022-502415-11_Redacted | 1.0 |
| Protocol (for publication) | D1_Protocol_2022-502415-11-00_EL_Greek_Redacted_PA2-EU | PA2-EU |
| Protocol (for publication) | D1_Protocol_2022-502415-11-00_Greek_redacted | 4.0 |
| Protocol (for publication) | D1_Protocol_2022-502415-11-00_redacted | 4.0 |
| Protocol (for publication) | D1_Protocol_2022-502415-11-00_Redacted_PA2-EU | PA2-EU |
| Recruitment arrangements (for publication) | K1_AT_Recruitment Procedure | 1 |
| Recruitment arrangements (for publication) | K1_BE_Recruitment Procedure | 1 |
| Recruitment arrangements (for publication) | K1_CZ_Recruitment Procedure_Bilingual | 1 |
| Recruitment arrangements (for publication) | K1_DE_Recruitment Procedure | 1.0 |
| Recruitment arrangements (for publication) | K1_DK_Recruitment procedure | 2 |
| Recruitment arrangements (for publication) | K1_EL_Recruitment procedure | 1 |
| Recruitment arrangements (for publication) | K1_ES_Recruitment Procedure | 1.0 |
| Recruitment arrangements (for publication) | K1_FR_Recruitment Procedure | 1.0 |
| Recruitment arrangements (for publication) | K1_IT_ Recruitment Procedure | 1 |
| Recruitment arrangements (for publication) | K1_PL_Recruitment Procedure_Polish | 1 |
| Recruitment arrangements (for publication) | K1_PT_Recruitment Procedure | 1 |
| Recruitment arrangements (for publication) | K1_SE_Recruitment Procedure_Swedish | 1 |
| Recruitment arrangements (for publication) | K2_PT_Recruitment Material_GP Letter_Portuguese | 2 |
| Subject information and informed consent form (for publication) | L1_AT_SIS ICF_Pregnancy_German_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_AT_SIS-ICF_Main_German_redacted | 6.1 |
| Subject information and informed consent form (for publication) | L1_AT_SIS-ICF_Site Info_German | 2 |
| Subject information and informed consent form (for publication) | L1_AT_SIS-ICF_Third-Party-Follow-up_German | 1.2 |
| Subject information and informed consent form (for publication) | L1_BE_SIS-ICF_Main_Dutch_redacted | 6.0 |
| Subject information and informed consent form (for publication) | L1_BE_SIS-ICF_Main_French_redacted | 6.0 |
| Subject information and informed consent form (for publication) | L1_BE_SIS-ICF_Main_Memo_Discontinuation of English-Language ICFs | 3 |
| Subject information and informed consent form (for publication) | L1_BE_SIS-ICF_Pregnancy ICF_Dutch | 2 |
| Subject information and informed consent form (for publication) | L1_BE_SIS-ICF_Pregnancy ICF_English | 2 |
| Subject information and informed consent form (for publication) | L1_BE_SIS-ICF_Pregnancy ICF_French | 2 |
| Subject information and informed consent form (for publication) | L1_BE_SIS-ICF_Recruitment Procedure | 1 |
| Subject information and informed consent form (for publication) | L1_CZ_SIS-ICF_Addendum Vital Status_Czech | 1.1 |
| Subject information and informed consent form (for publication) | L1_CZ_SIS-ICF_Data Privacy Notice ICF_Czech | 5.0 |
| Subject information and informed consent form (for publication) | L1_CZ_SIS-ICF_Main_Czech | 6.0 |
| Subject information and informed consent form (for publication) | L1_CZ_SIS-ICF_Partner Pregnancy ICF_Czech | 2.1 |
| Subject information and informed consent form (for publication) | L1_CZ_SIS-ICF_Pregnant Participant ICF_Czech | 2.1 |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Main_German_redacted | 6.1 |
| Subject information and informed consent form (for publication) | L1_DE_SIS-ICF_Pregnancy_German_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_DK_SIS-ICF_Main_Danish_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_DK_SIS-ICF_Non-Knowledge_Danish | 1.0 |
| Subject information and informed consent form (for publication) | L1_DK_SIS-ICF_Partner Pregnancy_Danish_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_EL_SIS-ICF_Addendum for collection of health status_Greek | 1.0 |
| Subject information and informed consent form (for publication) | L1_EL_SIS-ICF_Main_Greek_redacted | 6.0 |
| Subject information and informed consent form (for publication) | L1_EL_SIS-ICF_Partner Pregnancy_Greek_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Main_Spanish | 6.2 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_MRI_Spanish | 2.0 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Pregnant Partner_Spanish_redacted | 2.2 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Scintigraphy_Spanish | 1.1 |
| Subject information and informed consent form (for publication) | L1_ES_SIS-ICF_Vital Status_Spanish | 1.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Adults_french_redacted | 6.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Partner Pregnancy follow-up_french | 2.0 |
| Subject information and informed consent form (for publication) | L1_FR_SIS-ICF_Scout Participant Agreement_french | 1.3 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_Adult Main_Italian | 6.0 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_Confidentiality and Data Protection_Italian | 5.0 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_MRI substudy ICF_Italian | 2.0 |
| Subject information and informed consent form (for publication) | L1_IT_SIS-ICF_Pregnancy_Italian | 2.1 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_Main_Polish_redacted | 6.0 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_Pregnancy_Polish | 2.0 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_Scout_Polish | 3.0 |
| Subject information and informed consent form (for publication) | L1_PL_SIS-ICF_Third-Party Follow-up_Polish | 1.0 |
| Subject information and informed consent form (for publication) | L1_PT_SIS-ICF_Main_Portuguese_redacted | 6.0 |
| Subject information and informed consent form (for publication) | L1_PT_SIS-ICF_Partner Pregnancy_Portuguese | 2.1 |
| Subject information and informed consent form (for publication) | L1_PT_SIS-ICF_Subject Pregnancy_Portuguese | 2.1 |
| Subject information and informed consent form (for publication) | L1_SE_Other subject material_Email Comm_ERR-TR_Swedish | 1.0 |
| Subject information and informed consent form (for publication) | L1_SE_Other subject material_ScoutPass Reloadable_EUR_Swedish | 1.0 |
| Subject information and informed consent form (for publication) | L1_SE_Other subject material_ScoutPass_Swedish | 1 |
| Subject information and informed consent form (for publication) | L1_SE_SIS-ICF_Main_Swedish_redacted | 5.1 |
| Subject information and informed consent form (for publication) | L1_SE_SIS-ICF_Partner Pregnancy_Swedish | 2.0 |
| Subject information and informed consent form (for publication) | L1_SE_SIS-ICF_Scout_Swedish | 1.0 |
| Subject information and informed consent form (for publication) | L1_SE_SIS-ICF_Vital Status_Swedish | 1.0 |
| Subject information and informed consent form (for publication) | L2_AT_Other subject material_Scout Agreement_German_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_BE_Other Subject Material_Scout Agreement_Dutch_redacted | 1.3 |
| Subject information and informed consent form (for publication) | L2_BE_Other Subject Material_Scout Agreement_English_redacted | 1.3 |
| Subject information and informed consent form (for publication) | L2_BE_Other Subject Material_Scout Agreement_French_redacted | 1.3 |
| Subject information and informed consent form (for publication) | L2_CZ_Other Subject Material_Scout Agreement_Czech | 1.3 |
| Subject information and informed consent form (for publication) | L2_DE_Other subject material_Scout Agreement_German_redacted | 1.3 |
| Subject information and informed consent form (for publication) | L2_EL_Other subject material_Scout agreement_Greek_redacted | 1.3 |
| Subject information and informed consent form (for publication) | L2_IT_Other subject material_Scout participant agreement_Italian | 2.0 |
| Synopsis of the protocol (for publication) | D1_ Protocol synopsis_ES_2022-502415-11_Redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_ Protocol synopsis_ES_2022-502415-11_Redacted_PA2-EU | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_German_redacted | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_SE_Swedish_redacted | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis sub-study MRI_2022-502415-11-00_Italian_redacted | PA3 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_AT_German_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_AT_German_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_AT_German_redacted_PA2-EU | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_Dutch_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_Dutch_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_Dutch_redacted_PA2-EU | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_French_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_French_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_French_redacted_PA2-EU | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_German_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_BE_German_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_CZ_Czech_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_Czech_redacted_v2 | 2 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_DE_German_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_DE_German_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_DE_German_redacted_PA2-EU | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_EL_Greek_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_EL_Greek_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_EL_Greek_redacted_PA2-EU | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_ES_Spanish_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_FR_French_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_french_redacted_v2 | 2 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_IT_Italian_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_IT_Italian_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_IT_Italian_redacted_PA2-EU | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_PL_Polish_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_PT_Portuguese_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_PT_Portuguese_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_2022-502415-11-00_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_redacted_PA2-EU | PA2-EU |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_SE_Swedish_redacted | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2022-502415-11-00_SE_Swedish_redacted_PA1 | PA1 |
| Synopsis of the protocol (for publication) | D4_Memo to File_BE_Patient Facing Document_6MWT Worksheet_Dutch | 2 |
Application history
15 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-02-17 | Belgium | Acceptable 2023-05-30
|
2023-05-30 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2023-06-14 | Acceptable | 2023-07-31 | |
| 3 | SUBSEQUENT ADDITION OF MSC | APP-3 | 2023-06-30 | 2023-09-21 | ||
| 4 | SUBSEQUENT ADDITION OF MSC | APP-4 | 2023-06-30 | Acceptable 2023-05-30
|
2023-08-29 | |
| 5 | SUBSEQUENT ADDITION OF MSC | APP-5 | 2023-07-07 | Acceptable 2023-05-30
|
2023-08-25 | |
| 6 | SUBSTANTIAL MODIFICATION | SM-2 | 2023-07-07 | Acceptable | 2023-07-20 | |
| 7 | SUBSTANTIAL MODIFICATION | SM-3 | 2023-09-25 | Acceptable | 2023-11-14 | |
| 8 | SUBSTANTIAL MODIFICATION | SM-4 | 2024-01-09 | Belgium | No conclusion 2024-03-11
|
2024-03-14 |
| 9 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-10-07 | Belgium | No conclusion 2024-03-11
|
2024-10-07 |
| 10 | SUBSEQUENT ADDITION OF MSC | APP-10 | 2024-11-18 | 2025-03-02 | ||
| 11 | SUBSTANTIAL MODIFICATION | SM-5 | 2024-12-20 | No conclusion | 2025-02-28 | |
| 12 | SUBSTANTIAL MODIFICATION | SM-6 | 2025-01-23 | No conclusion | 2025-03-14 | |
| 13 | SUBSTANTIAL MODIFICATION | SM-7 | 2025-02-27 | No conclusion | 2025-03-28 | |
| 14 | SUBSTANTIAL MODIFICATION | SM-8 | 2025-07-10 | Belgium | Acceptable 2025-10-17
|
2025-10-17 |
| 15 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-10-30 | Belgium | Acceptable 2025-10-17
|
2025-10-30 |