Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
218
Countries
9
Sites
67
Graves’ Disease
To evaluate the efficacy of IMVT-1402 dose 1 versus placebo as assessed by T3 (Total T3 or FT3), FT4, TSH, and ATD dose at Week 26
Key facts
- Sponsor
- Immunovant Sciences GmbH
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20]
- Trial duration
- 21 Nov 2025 → ongoing
- Decision date (initial)
- 2025-10-22
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
External identifiers
- EU CT number
- 2025-521920-31-00
- ClinicalTrials.gov
- NCT07018323
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacogenetic, Therapy, Safety, Efficacy, Pharmacodynamic, Pharmacokinetic
To evaluate the efficacy of IMVT-1402 dose 1 versus placebo as assessed by T3 (Total T3 or FT3), FT4, TSH, and ATD dose at Week 26
Conditions and MedDRA coding
Graves’ Disease
Regulatory references
- Scientific advice from competent authorities
- European Medicines Agency
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- Participants with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures
- Male or female participants aged ≥ 18 years
- Participants with diagnosis of GD who are hyperthyroid despite ATD treatment
Exclusion criteria 3
- Have previously been successfully treated with RAI therapy or have undergone total thyroidectomy.
- Have an autoimmune disease other than GD requiring treatment that, in the Investigator’s judgment, puts the participant at undue risk
- Have moderate-to-severe active thyroid eye disease (TED) and are expected to require immediate surgical intervention and/or are planning corrective surgery/irradiation or medical therapy for TED during study participation
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Proportion of participants who are euthyroid and off ATD at Week 26
Secondary endpoints 9
- Proportion of participants who have T3 (Total T3 or FT3) and FT4 ≤ ULN and are off ATD at Week 26
- Proportion of participants who are euthyroid, off ATD, and seronegative at Week 26
- Proportion of participants who are euthyroid and off ATD at Week 26
- Proportion of participants who have T3 (Total T3 or FT3) and FT4 ≤ ULN and are off ATD at Week 26
- Proportion of participants who are euthyroid, off ATD, and seronegative at Week 26
- Proportion of participants who have T3 (Total T3 or FT3) and FT4 ≤ ULN at Week 4 in participants who have T3 (Total T3 or FT3) and/or FT4 > ULN at baseline
- Proportion of participants who have T3 (Total T3 or FT3) and FT4 ≤ ULN at Week 2 in participants who have T3 (Total T3 or FT3) and/or FT4 > ULN at baseline
- Proportion of participants who have T3 (Total T3 or FT3) and FT4 ≤ ULN at Week 4 in participants who have T3 (Total T3 or FT3) and/or FT4 > ULN at baseline
- Proportion of participants who have T3 (Total T3 or FT3) and FT4 ≤ ULN at Week 2 in participants who have T3 (Total T3 or FT3) and/or FT4 > ULN at baseline
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11127703 · Product
- Active substance
- Imeroprubart
- Substance synonyms
- Human IgG1 monoclonal antibody against FcRn receptor, IMVT-1402, HL161ANS
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 600 mg milligram(s)
- Max total dose
- 15600 mg milligram(s)
- Max treatment duration
- 26 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- IMMUNOVANT SCIENCES GMBH
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
Placebo is identical to IMP but with no active substance.
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Immunovant Sciences GmbH
- Sponsor organisation
- Immunovant Sciences GmbH
- Address
- Viaduktstrasse 8
- City
- Basel Town
- Postcode
- 4051
- Country
- Switzerland
Scientific contact point
- Organisation
- Immunovant Sciences GmbH
- Contact name
- Immunovant Clinical Trials
Public contact point
- Organisation
- Immunovant Sciences GmbH
- Contact name
- Immunovant Clinical Trials
Third parties 18
| Organisation | City, country | Duties |
|---|---|---|
| Avance Biosciences Inc. ORG-100016282
|
Houston, United States | Laboratory analysis |
| Clariness GmbH ORG-100045306
|
Hamburg, Germany | Other |
| Voisin Consulting Life Sciences ORG-100009282
|
Boulogne Billancourt, France | Code 12 |
| Scout Clinical ORG-100042228
|
Dallas, United States | Other |
| Pharmaceutical Research Associates Group B.V. ORG-100006268
|
Assen, Netherlands | Laboratory analysis |
| Icon Clinical Research Limited ORG-100008322
|
Dublin 18, Ireland | Other, Code 5 |
| Mayo Collaborative Services LLC ORG-100046687
|
Rochester, United States | Laboratory analysis |
| Suvoda LLC ORG-100043523
|
Conshohocken, United States | Interactive response technologies (IRT) |
| Catalent Cts (Edinburgh) Limited ORG-100011832
|
Bathgate, United Kingdom | Code 14 |
| Catalent Pharma Solutions LLC ORG-100011506
|
Philadelphia, United States | Code 14 |
| Avantor Inc. ORG-100050885
|
Wayne, United States | Other |
| Certe Medische Diagnostiek en Advies Stichting ORG-100050554
|
Groningen, Netherlands | Laboratory analysis |
| Arup Laboratories Inc. ORG-100041750
|
Salt Lake City, United States | Laboratory analysis |
| Primevigilance USA Inc. ORG-100047266
|
Raleigh, United States | Code 8 |
| Icon Clinical Research Limited ORG-100008322
|
Dublin 18, Ireland | Laboratory analysis |
| PRA Hellas CRO A.E. ORG-100048208
|
Nea Ionia, Greece | On site monitoring, Code 5 |
| Clinical Ink Inc. ORG-100042433
|
Winston Salem, United States | Other, E-data capture |
| Syneos Health Clinique Inc. ORG-100028348
|
Quebec, Canada | Laboratory analysis |
Locations
9 EU/EEA countries · 67 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Bulgaria | Ongoing, recruiting | 22 | 14 |
| Czechia | Ongoing, recruiting | 10 | 5 |
| Germany | Authorised, recruitment pending | 10 | 2 |
| Greece | Ongoing, recruiting | 8 | 4 |
| Italy | Ongoing, recruiting | 54 | 16 |
| Latvia | Ongoing, recruiting | 15 | 6 |
| Netherlands | Ongoing, recruiting | 7 | 4 |
| Poland | Ongoing, recruiting | 24 | 12 |
| Spain | Authorised, recruitment pending | 10 | 4 |
| Rest of world
Brazil, Chile, Israel, Japan, Georgia, United Kingdom, Korea, Republic of, New Zealand, Australia, United States, Mexico
|
— | 58 | — |
Investigational sites
University Multiprofile Hospital For Active Treatment Sofiamed OOD
Clinic of Endocrinology and Metabolic Diseases, Ulitsa Dimitir Mollov 10, 1750, Sofiya
University Multiprofile Hospital For Active Treatment Pulmed Ltd.
Department of Endocrinology and Metabolic Diseases at Clinic of Internal Diseases, Ulitsa Perushtitsa 1a, 4002, Plovdiv
University Multiprofile Hospital For Active Treatment Kaspela EOOD
Clinic of Endocrinology and Metabolic Diseases, Zapaden District, Sofia Str 64, Plovdiv
Diagnostic Consultative Center Equita OOD
N/A, Bulevard Tsar Osvoboditel 5, 9000, Varna
Alexandrovska University Hospital
Clinic of Endocrinology and Metabolic Diseases, Georgy Sofiiski Str 1, 1431, Sofia
Medical Center Berbatov Ltd.
N/A, Ulitsa Beli Drin 9, 8600, Yambol
Multiprofile Hospital For Active Treatment Knyaginya Klementina Sofia EAD
Department of Endocrinology and Metabolic Diseases, Bulevard Gen. Stoletov No 67a, 1233, Sofia
Medical Center Smolyan Klinikal Research Ltd.
N/A, Ulitsa Han Asparuh 2, 4700, Smolyan
Medical Centre Pratia Clinic EOOD
N/A, Bulevard Republika 15, 9020, Varna
Medical Center Viva Phoenix OOD
N/A, Ulitsa Nezavisimost 2, 9300, Dobrich
Medcenter Nova Clinic Ltd.
N/A, Ulitsa Vyara 7, 9020, Varna
Kalimat Medical Center Ltd.
N/A, Ulitsa Yastrebets 11, 1680, Sofia
Multiprofile Hospital For Active Treatment Hadji Dimitar OOD
Department of Endocrinology and Metabolic Diseases, Ulitsa Dimitir Pehlivanov 5, 8800, Sliven
University Specialized Hospital For Active Treatment In Endocrinology Akad. Iv. Penchev EAD
Third Clinic of Endocrinology and Metabolic Diseases, Ulitsa Zdrave 2, 1431, Sofia
Edumed s.r.o.
Private Practice, Mlynska 189, 547 01, Nachod
Endokrinologie Klanovice s.r.o.
N/A, Axmanova 131, Klanovice, Prague
ResTrial s.r.o.
Klinika ResTrial, Zhorelecka 514/2, Bohnice, Prague 8
Milan Kvapil s.r.o.
Diabetologická ambulance, Michnova 1622/4, Chodov, Prague
Vseobecna Fakultni Nemocnice V Praze
Oddelenf endokrinologie a metabolismu, U Nemocnice 504/1 Nove Mesto, 128 00, Prague
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
Department of Medicine III, Devision of Endocrinology, Diabetes and Bone deseases, Fetscherstrasse 74, Johannstadt-Nord, Dresden
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department of Medicine I, Langenbeckstrasse 1, Oberstadt, Mainz
Alexandra Hospital
Endocrine Department and Diabetical Center, Vassilissas Sofias Avenue 80, 115 28, Athens
Evaggelismos Hospital
Department of Endocrinology "D.IKKOS", Ipsiladou 45-47, 106 76, Athens
Laiko General Hospital Of Athens
1st department of internal medecine, Endocrinology Unit, Agiou Thoma (goudi) 17, 115 27, Athens
Ippokratio General Hospital Of Thessaloniki
Department of Endocrinology, Diabetes and Metabolism, Konstadinoupoleos 49, 546 42, Thessaloniki
Azienda Sanitaria Universitaria Friuli Centrale
Oncology Area Department, Via Pozzuolo 330, 33100, Udine
Hospital Santa Maria Della Misericordia
Medicine and Surgery, Piazzale Giorgio Menghini 1, 06129, Perugia
Ospedale San Raffaele S.r.l.
Endocrinology Department, Via Olgettina 60, 20132, Milan
Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii
SC Malattie Endocrine-Diabetologia, Piazza Oms 1, 24127, Bergamo
Azienda Ospedaliero-Universitaria Policlinico Umberto I
Department of Experimental Medicine, Sapienza University of Rome, Viale Del Policlinico 155, 00161, Rome
Universita Humanitas
Department of Biomedical Sciences, Via Rita Levi Montalcini 4, 20072, Pieve Emanuele
Istituti Clinici Scientifici Maugeri S.p.A. Societa' Benefit In Forma Abbreviata Istituti Clinici Scientifici Maugeri S.p.A. Sb O Anche Ics Maugeri S.p.A. Sb O Maugeri S.p.A. Sb
Unit of Endocrinology and Metabolic Diseases, Via Salvatore Maugeri 10, 27100, Pavia
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Endocrinologia, Largo Francesco Vito 1, 00168, Rome
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Dipartimento di Medicinina Generale e Specilistica, Corso Bramante 88, 10126, Turin
Azienda Ospedaliera Santa Croce E Carle
Endocrinology, Via Michele Coppino 26, 12100, Cuneo
Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata Fon
Osteo-metabolic and Thyroid Disorders, Via Alvaro Del Portillo N 200, 00128, Rome
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
UOC Endocrinologia, Via Antonio Cardarelli 9, 80131, Naples
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
PROMISE / DAI Medico / UOC Malattie Endocrine, del ricambio e della nutrizione, Via Del Vespro 129, 90127, Palermo
Azienda Unita' Sanitaria Locale Toscana Nord Ovest
U.O. Aziendale di Endocrinologia, Viale Vittorio Alfieri 36, 57124, Leghorn
Azienda Ospedaliera Ordine Mauriziano Di Torino
SC Endocrinologia, Via Ferdinando Magellano 1, 10128, Turin
Ente Ecclesiastico Ospedale Generale Regionale Miulli
Division of Endocrinology and Metabolic Diseases, Strada Provinciale 127 Acquaviva Santeramo 4/100, 70021, Acquaviva Delle Fonti
Veselibas un socialas aprupes klinika APS SIA
Endocrinology, Lastadijas Iela 40, 1050, Riga
Siguldas Slimnica
Outpatient Department, Ziedu Iela 5, 2150, Siguldas Pagasts
Daces Teterovskas arsta prakse endokrinologija SIA
N/A, Brivibas Street 22, 5001, Ogre
Ziemelkurzemes regionala slimnica SIA
Ophthalmology, Inzenieru Iela 60, 3601, Ventspils
Riga East University Hospital
Clinical Centre "Bikernieki", Ophthalmology clinic, Lielvardes Street 68, LV-1006, Riga
Pauls Stradins Clinical University Hospital
Centre of Endocrinology, Pilsonu Iela 13, 1002, Riga
Ikazia Ziekenhuis
Internal medicine, Montessoriweg 1, 3083 AN, Rotterdam
Sint Franciscus Vlietland Groep Stichting
Internal Medicine, Kleiweg 500, 3045 PM, Rotterdam
Albert Schweitzer Ziekenhuis
Internal Medicine, Albert Schweitzerplaats 25, 3318 AT, Dordrecht
Maasstad Ziekenhuis Stichting
Internal Medicine, Maasstadweg 21, 3079 DZ, Rotterdam
Uniwersytecki Szpital Kliniczny W Bialymstoku
Klinika Endokrynologii, Diabetologii i Chorób Wewnętrznych, Ul. Marii Curie-Sklodowskiej 24a, 15-276, Bialystok
Swietokrzyskie Centrum Onkologii Samodzielny Publiczny Zaklad Opieki Zdrowotnej W Kielcach
Klinika Endokrynologii, Ul. Prezydenta Stefana Artwinskiego 3, 25-734, Kielce
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Klinika Endokrynologii i Chorób Wewnętrznych, Ul. Wybrzeze Ludwika Pasteura 4, 50-367, Wroclaw
Trialmed Sp. z o.o.
Clinical Trials, Solipska 27/ Lu 3, 02-482, Warsaw
Centrum Medyczne Intercor Sp. z o.o.
N/A, Ul. Kasztanowa 57, 85-605, Bydgoszcz
Med Polonia Sp. z o.o.
Dział badań klinicznych, Obornicka 262, 60-693, Poznan
Reumed Sp. z o.o.
Poradnia endokrynologiczna i diabetologiczna, Ul. Konrada Wallenroda 2f/4, 20-607, Lublin
Uniwersytecki Szpital Kliniczny Nr 1 Im. Prof. Tadeusza Sokolowskiego Pum W Szczecinie
Centrum Wsparcia Badań Klinicznych Pomorskiego Uniwersytetu Medycznego, Ul. Unii Lubelskiej 1, 71-252, Szczecin
Diab Serwis Popenda Sp. j.
N/A, Ul. Jozefa Ryszki 51, 41-516, Chorzow
Etyka Osrodek Badan Klinicznych Tomasz Pesta S.K.A.
N/A, Ul. 1 Maja 13 C, 10-117, Olsztyn
Panstwowy Instytut Medyczny Ministerstwa Spraw Wewnetrznych I Administracji
Cetrum Diabetologiczne Kiliniki Chorób Wewnętrznych, Endokrynologii i Diabetologii, Ul. Woloska 137, 02-507, Warsaw
Clinicly Sp. z o.o.
Clinicly Sp. z o.o. Centrum Medyczne Clinicly, Ul. Ludwika Warynskiego 30, 45-047, Opole
Hospital Universitario De La Princesa
Endocrinology, Calle De Diego De Leon 62, 28006, Madrid
Hospital Universitario Virgen De La Macarena
Ophthalmology, Avenida Del Doctor Fedriani 3, 41009, Sevilla
Hospital Clinico San Carlos
Endocrinology, Calle Del Profesor Martin Lagos Sn, 28040, Madrid
Hospital Germans Trias I Pujol
Endocrinology, Carretera Canyet 1a Planta, 08916, Badalona
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 |
|---|---|---|---|---|---|
| Bulgaria | 2025-11-24 | 2025-11-26 | |||
| Czechia | 2025-11-21 | 2025-12-04 | |||
| Greece | 2025-12-19 | 2026-02-03 | |||
| Italy | 2026-02-24 | 2026-04-22 | |||
| Latvia | 2025-11-26 | 2025-12-15 | |||
| Netherlands | 2026-01-23 | 2026-04-14 | |||
| Poland | 2025-11-21 | 2025-12-11 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 214 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Justification for Placebo Use_2025-521920-31-00_redacted | N/A |
| Protocol (for publication) | D1_PI facing_Questionnaire 1_EN_redacted | N/A |
| Protocol (for publication) | D1_PI facing_Questionnaire 2_EN_redacted | N/A |
| Protocol (for publication) | D1_PI facing_Questionnaire 3_EN_redacted | N/A |
| Protocol (for publication) | D1_Protocol_2025-521920-31-00_GR_redacted | 4.0 |
| Protocol (for publication) | D1_Protocol_2025-521920-31-00_redacted | 4.0 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_BG | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_CZ | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_DE | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_EN | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_ES | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_GR | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_IT | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_LV | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_LV-RU | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Dosing Diary_PL | 2.1 |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_BG_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_CZ_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_DE_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_EN_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_ES_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_GR_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_IT_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_LV_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_LV-RU_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 1_PL_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_BG_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_CZ_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_DE_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_EN_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_ES_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_GR_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_IT_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_LV_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_LV-RU_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 2_PL_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_BG_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_CZ_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_DE_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_EN_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_ES_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_GR_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_IT_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_LV_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_LV-RU_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 3_PL_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_BG_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_CZ_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_DE_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_EN_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_ES_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_GR_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_IT_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_LV_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_LV-RU_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_Questionnaire 4_PL_redacted | N/A |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_BG | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_CZ | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_DE | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_EN | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_ES | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_GR | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_IT | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_LV | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_LV-RU | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Post_PL | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_BG | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_CZ | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_DE | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_EN | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_ES | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_GR | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_IT | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_LV | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_LV-RU | 2.2 |
| Protocol (for publication) | D4_Patient facing document_SIAQ-Pre_PL | 2.2 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 3.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | N/A |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_LV_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Physician Referral Letter_redacted | 3.0 |
| Recruitment arrangements (for publication) | K2_Recruitment material_EC Document Recruitment Master | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment material_EC Document Recruitment Master | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment material_EC Document Recruitment Master | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment material_EC Document Recruitment Master | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Patient Brochure_LV_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Patient Brochure_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Patient Brochure_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Patient Brochure_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Patient Brochure_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Patient Brochure_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Patient Brochure_redacted | 03 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster_LV | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Poster_TC | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment Animation_redacted | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment Animation_redacted | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment Animation_redacted | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment Animation_redacted | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment Animation_redacted | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment Animation_redacted | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment_Banner Ads | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment_Banner Ads | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment_Banner Ads | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment_Banner Ads | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment_Banner Ads | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment_Banner Ads | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Recruitment_Banner Ads | 01 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_LV_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_LV-RU_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_redacted | 02 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_redacted | 03ES |
| Recruitment arrangements (for publication) | K2_Recruitment material_Study Guide_redacted | 02 |
| Subject information and informed consent form (for publication) | L1_Dosing Diary | 2.1 |
| Subject information and informed consent form (for publication) | L1_Emergency Patient Card | 1.0 |
| Subject information and informed consent form (for publication) | L1_Home Dosing Worksheet_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_Instruction for Use_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_Instruction for Use_Video Script_redacted | N/A |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver_BG | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver_EN | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver_LV | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver_LV-RU | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Caregiver_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Consent Aid_BG_redacted | 02 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Consent Aid_EN_redacted | 02 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Consent Aid_redacted | 02 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Consent Aid_redacted | 02 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Consent Aid_redacted | 02 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Consent Aid_redacted | 02 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Data Privacy | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Data Privacy_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Home HealthCare | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_BG_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_EN_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_LV_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_LV-RU_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_redacted | 3.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_redacted | 3.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Genetic Research_BG_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Genetic Research_EN_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Genetic Research_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Genetic Research_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Genetic Research_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Optional Genetic Research_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Patient Navigator Consent | 01 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Patient Navigator Consent | 01 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Patient Navigator Consent_BG | 01 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Patient Navigator Consent_EN | 01 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_BG_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_EN_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_LV_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_LV-RU_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_redacted | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout_BG | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout_EN | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout_LV | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout_LV-RU | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Scout_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Supporting person_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L2_Letter from Investigator to GP_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_SIS and ICF_Consent Aid_redacted | 02 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_BG_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_CZ_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_EN_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_ES_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_GR_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_IT_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_LV_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_LV-RU_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_NL_Redacted | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Language synopsis_2025-521920-31-00_PL_Redacted | 1.0 |
Application history
4 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-06-27 | Italy | Acceptable 2025-10-17
|
2025-10-17 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-10-29 | Acceptable 2025-10-17
|
2025-10-29 | |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2025-11-05 | Acceptable 2025-10-17
|
2025-11-05 | |
| 4 | SUBSTANTIAL MODIFICATION | SM-1 | 2026-02-20 | Italy | Acceptable 2026-04-22
|
2026-04-22 |