Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruitment ended
Participants planned
147
Countries
11
Sites
35
Primary immune thrombocytopenia (ITP)
To demonstrate that the addition of ianalumab (either dose) to standard of care, eltrombopag, prolongs Time to Treatment Failure (TTF) compared to eltrombopag alone in participants with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids
Key facts
- Sponsor
- Novartis Pharma AG
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 20 Mar 2023 → ongoing
- Decision date (initial)
- 2024-08-12
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Novartis Pharma AG
External identifiers
- EU CT number
- 2024-512890-28-00
- EudraCT number
- 2022-001627-32
- ClinicalTrials.gov
- NCT05653219
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Pharmacodynamic, Others, Pharmacokinetic, Efficacy, Therapy
To demonstrate that the addition of ianalumab (either dose) to standard
of care, eltrombopag, prolongs Time to Treatment Failure (TTF) compared to eltrombopag alone in participants with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids
Secondary objectives 9
- To assess quality, time to and duration of response in each treatment arm
- To assess the rate of participants who successfully taper and discontinue eltrombopag in each treatment arm
- To assess the safety profile of ianalumab.
- To assess the incidence and severity of bleeding in each treatment arm
- To assess the need for rescue treatment in each treatment group.
- To evaluate treatment effects on ITP related symptoms, functioning, and health-related quality of life (HFRQoL)
- To assess B-Cell levels and immunoglobulin (lg) levels
- To assess ianalumab pharmacokinetics (PK)
- To assess the immunogenicity of ianalumab
Conditions and MedDRA coding
Primary immune thrombocytopenia (ITP)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 23.0 | LLT | 10083843 | Primary immune thrombocytopenia | 10005329 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Male or female patients aged 18 years and older on the day of signing the informed consent.
- A signed informed consent must be obtained prior to participation in the study
- A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy ± IVIG.
- Patients with platelet count <30 G/L for whom eltrombopag is clinically indicated as per physician’s discretion and with no contraindication to receive eltrombopag.
Exclusion criteria 9
- ITP patients who received second-line ITP treatments (other than corticosteroid therapy ± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonist (TPO-RAs) for a limited time (max. one week) before screening are eligible.
- Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia (patients with low grade anemia related to bleeding or iron deficiency are eligible).
- Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters.
- Patients with current or history of life-treating bleeding.
- Patients that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)- positive.
- Patients with known active or uncontrolled infection requiring systemic treatment during screening period.
- Patients with hepatic impairment with Child-Pugh score >5
- Patients with concurrent coagulation disorder and/or receiving anti-platelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (≤150 mg daily).
- Nursing (breast feeding) or pregnant women.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Time to treatment failure (TTF) defined as the time from randomization until: - Platelet counts <30 G/L or need for rescue treatment later than 8 weeks from randomization, - Start of new ITP treatment - Ineligibility to taper or inability to discontinue eltrombopag or - Death. TTF will be assessed in each treatment group and each of the 2 doses of ianalumab (ianalumab+ eltrombopag) will be compared to the control arm (placebo+ eltrombopag).
Secondary endpoints 9
- At each time point: - Complete Response (CR) rate (proportion of participants with any platelet count ≤100 G/L without rescue or new ITP treatment).Response rate (R) (proportion of participants with any platelet count ≤50 G/L without rescue or new ITP treatment). Best response rate over all time points(proportion of participants with a best response of either R or CR). Time from randomization to R & CR. Duration of R & CR. Stable response at 6 months & at 1 year.
- Probability to be treatment failure-free (as defined for the primary efficacy endpoint) at the end of the planned treatment period (end of Week 24)
- Frequency of adverse events and other safety parameters. Number of severe infections and proportion of participants with severe infection.
- Proportion of participants with bleeding events according to World Health Organization (WHO)Bleeding Scale.
- Number and proportion of participants receiving rescue treatment.
- Change from baseline on total score of the Patient- Reported Outcomes Measurement Information System (PROMIS)Short Form (SF) v1.0 Fatigue 13a. Change from baseline in ITP Patient Assessment Questionnaire (PAQ) domain scores of Symptoms, Fatigue, Bother, Activity.
- B-cell levels: - Change from baseline in the frequency (% within the CD45) and absolute number of CD19+ B-cell counts - Time to first occurrence of B-cell recovery, defined as ≥80% of baseline or ≥50 cells/µL Immunoglobulins: - Change from baseline in immunoglobulin level
- Ianalumab concentration in serum and PK parameters after the first and last dose in a subset of participants
- Incidence and titer of anti-ianalumab antibodies in serum (anti-drug antibodies (ADA) assay) over time
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 5
PRD10266757 · Product
- Active substance
- Ianalumab
- Pharmaceutical form
- CONCENTRATE FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 9 mg/Kg milligram(s)/kilogram
- Max total dose
- 9 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 16 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- NOVARTIS PHARMA AG
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/25/3036 (ITP)
SUB30140 · Substance
- Active substance
- Eltrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 75 mg milligram(s)
- Max total dose
- 126 g gram(s)
- Max treatment duration
- 24 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB30140 · Substance
- Active substance
- Eltrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 75 mg milligram(s)
- Max total dose
- 126 g gram(s)
- Max treatment duration
- 24 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB30140 · Substance
- Active substance
- Eltrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 75 mg milligram(s)
- Max total dose
- 126 g gram(s)
- Max treatment duration
- 24 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB30140 · Substance
- Active substance
- Eltrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 75 mg milligram(s)
- Max total dose
- 126 g gram(s)
- Max treatment duration
- 24 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Placebo 1
Placebo to ianalumab 150 mg/1 mL concentrate for solution for infusion
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
Auxiliary 6
-
H02AB · Product
- Pharmaceutical form
- PHF00231MIG
- Route of administration
- UNKNOWN USE
- Max daily dose
- 0 DF dosage form
- Max total dose
- 0 DF dosage form
- Max treatment duration
- 39 Month(s)
- Authorisation status
- Authorised
- ATC code
- H02AB — GLUCOCORTICOIDS
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SCP25844199 · ATC
- Active substance
- Entecavir
- Substance synonyms
- 2-amino-9-((1S,3R,4S)-4-hydroxy-3-(hydroxymethyl)-2-methylenecyclopentyl)-1,9-dihydro-6H-purin-6-one
- Route of administration
- UNKNOWN USE
- Max daily dose
- 0 DF dosage form
- Max total dose
- 0 DF dosage form
- Max treatment duration
- 15 Month(s)
- Authorisation status
- Authorised
- ATC code
- J05AF10 — ENTECAVIR
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
-
J06BA · Product
- Pharmaceutical form
- PHF00230MIG
- Route of administration
- UNKNOWN USE
- Max daily dose
- 0 DF dosage form
- Max total dose
- 0 DF dosage form
- Max treatment duration
- 39 Month(s)
- Authorisation status
- Authorised
- ATC code
- J06BA — IMMUNOGLOBULINS, NORMAL HUMAN
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
-
R06A · Product
- Pharmaceutical form
- -
- Route of administration
- UNKNOWN USE
- Max daily dose
- 0 DF dosage form
- Max total dose
- 0 DF dosage form
- Max treatment duration
- 39 Month(s)
- Authorisation status
- Authorised
- ATC code
- R06A — ANTIHISTAMINES FOR SYSTEMIC USE
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
-
N02BE · Product
- Pharmaceutical form
- PHF00006MIG
- Route of administration
- UNKNOWN USE
- Max daily dose
- 0 DF dosage form
- Max total dose
- 0 DF dosage form
- Max treatment duration
- 39 Month(s)
- Authorisation status
- Authorised
- ATC code
- N02BE — ANILIDES
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
A02BA · Product
- Active substance
- H2-receptor antagonists
- Pharmaceutical form
- -
- Route of administration
- UNKNOWN USE
- Max daily dose
- 0 DF dosage form
- Max total dose
- 0 DF dosage form
- Max treatment duration
- 39 Week(s)
- Authorisation status
- Authorised
- ATC code
- A02BA — H2-receptor antagonists
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Novartis Pharma AG
- Sponsor organisation
- Novartis Pharma AG
- Address
- Lichtstrasse 35
- City
- Basel
- Postcode
- 4056
- Country
- Switzerland
Scientific contact point
- Organisation
- Novartis Pharma AG
- Contact name
- Novartis Pharma Arzneimittel GmbH
Public contact point
- Organisation
- Novartis Pharma AG
- Contact name
- Novartis Pharma Arzneimittel GmbH
Third parties 21
| Organisation | City, country | Duties |
|---|---|---|
| Syneos Health Inc. ORG-100008382
|
Morrisville, United States | On site monitoring |
| Icon Clinical Research Limited ORG-100008322
|
Dublin 18, Ireland | On site monitoring, Code 2, E-data capture |
| Iqvia Laboratories Limited ORG-100042527
|
Livingston, United Kingdom | Laboratory analysis |
| Icon Laboratory Services Inc. ORG-100037135
|
Farmingdale, United States | Laboratory analysis |
| Labcorp Early Development Laboratories Limited ORG-100011365
|
Harrogate, United Kingdom | Laboratory analysis |
| Labcorp Pharmaceutical Research And Development (Shanghai) Co. Ltd. ORG-100043119
|
Shanghai, China | Laboratory analysis |
| Veeda Clinical Research Limited ORG-100012827
|
Ahmedabad, India | Laboratory analysis |
| ICON Bioanalytical Laboratory ORL-000007500
|
Assen, Netherlands | Code 13, Laboratory analysis |
| Illingworth Research Group Limited ORG-100042356
|
Macclesfield, United Kingdom | Other |
| Mag. Andreas Raffeiner GmbH ORG-100043223
|
Walding, Austria | Code 8 |
| RWS Life Sciences Inc. ORG-100042348
|
East Hartford, United States | Other |
| Reify Health Inc. ORG-100049669
|
Boston, United States | Code 13 |
| Parexel International (IRL) Limited ORG-100022780
|
Dublin 2, Ireland | Code 12 |
| Iqvia Rds Inc. ORG-100043858
|
Durham, United States | Other, Interactive response technologies (IRT) |
| IQVIA Limited ORG-100008655
|
Reading, United Kingdom | On site monitoring |
| Laboratory Corporation Of America ORG-100050465
|
Burlington, United States | Laboratory analysis |
| Movianto Ceska republika s.r.o. ORG-100012787
|
Podoli, Czechia | Other |
| Jumo Health USA Inc. ORG-100044054
|
New Haven, United States | Other |
| Mag. Andreas Raffeiner GmbH ORG-100043223
|
Walding, Austria | Code 8 |
| DATAMAP-Gesellschaft fuer Datenmanagement Datenanalyse und Datenpraesentation mbH ORG-100042869
|
Freiburg Im Breisgau, Germany | Code 10, Other |
| Movianto Ceska republika s.r.o. ORG-100012787
|
Podoli, Czechia | Other |
Locations
11 EU/EEA countries · 35 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ended | 3 | 2 |
| Belgium | Ongoing, recruitment ended | 3 | 2 |
| Czechia | Ongoing, recruitment ended | 4 | 3 |
| France | Ongoing, recruitment ended | 1 | 3 |
| Germany | Ongoing, recruitment ended | 13 | 7 |
| Hungary | Ongoing, recruitment ended | 3 | 2 |
| Italy | Ongoing, recruitment ended | 6 | 5 |
| Netherlands | Ongoing, recruitment ended | 3 | 2 |
| Norway | Ended | 2 | 1 |
| Romania | Ended | 5 | 2 |
| Spain | Ongoing, recruitment ended | 5 | 6 |
| Rest of world
India, Turkey, Taiwan, China, Argentina, Japan, Thailand, Australia, Philippines, Malaysia, Singapore, Korea, Republic of, Mexico, United States, United Kingdom
|
— | 99 | — |
Investigational sites
Medical University Of Vienna
3050: Department of Hematology, Waehringer Guertel 18-20, Alsergrund, Vienna
Klinikum Wels-Grieskirchen GmbH
3052: Department of Hematology, Grieskirchner Strasse 42, 4600, Wels
Algemeen Ziekenhuis Delta
#3101: Hematology, Deltalaan 1, 8800, Roeselare
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur
#3102: Hematology, Avenue Docteur Gaston Therasse 1, 5530, Yvoir
Fakultni Nemocnice Brno
#3250: Interni hematologicka a onkologicka klinika, Jihlavska 340/20, Bohunice, Brno
Vseobecna Fakultni Nemocnice V Praze
#3252: Hematologie, U Nemocnice 499/2, Nove Mesto, Prague
Fakultni Nemocnice Kralovske Vinohrady
#3251: Hematologicka klinika, Srobarova 1150/50, Vinohrady, Prague
CHRU De Nancy
#3352: Médecine interne, Rue Du Morvan, 54500, Vandoeuvre Les Nancy
Clinique Victor Hugo
#3350: Hématologie, Centre De Cancerologie De La Sarthe, 66 Rue De Degre, Le Mans
Centre Hospitalier Blois Simone Veil
#3354: Médecine interne, Mail Pierre Charlot, 41016, Blois Cedex
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
#3456: Medizinische Klinik und Poliklinik 1, Fetscherstrasse 74, Johannstadt-Nord, Dresden
Universitaetsklinikum Aachen AöR
3457_CiO_A:Uniklinik RWTH Aachen Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzellt, Pauwelsstrasse 30, 52074, Aachen
University Hospital Cologne AöR
#3457: Department I of Internal Medicine, Kerpener Strasse 62, Lindenthal, Cologne
Universitaetsklinikum Jena KöR
3453:Klinik für Innere Medizin II Hämatologie und internistische Onkologie, Am Klinikum 1, Lobeda, Jena
Universitaetsklinikum Duesseldorf AöR
3457_CIO_D:Klinik für Hämatologie, Onkologie und klinische Immunologie, Moorenstrasse 5, Bilk, Duesseldorf
Gemeinschaftspraxis Haematologie Onkologie
#3450: Private Practice, Arnoldstrasse 18, Johannstadt-Nord, Dresden
Universitaetsklinikum Bonn AöR
3457_CIO_B:Medizinische Klinik III, Venusberg-Campus 1, Venusberg, Bonn
Semmelweis University
#3552: Belgyogyaszati es Hematologiai Klin, Szentkiralyi Utca 46, VIII Kerulet, Budapest VIII
University Of Debrecen
#3551: Hematologia Osztaly, Nagyerdei Korut 98, 4032, Debrecen
Azienda Sanitaria Universitaria Giuliano Isontina
#3651 - U.C.O. Ematologia - Dipartimento di Oncologia, Via Costantino Costantinides 2, 34128, Trieste
Azienda Ospedaliera Policlinico Universitario Tor Vergata
#3652 - U.O.S.D. Patologie Mieloproliferative Dipartimento Oncoematologia, Viale Oxford 81, 00133, Rome
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
#3650 - U.O.C. Servizio e DH di Ematologia, Largo Francesco Vito 1, 00168, Rome
Azienda Unita Sanitaria Locale Di Bologna
#3655 - U.O.C. Ematologia, Via Giuseppe Massarenti 9, 40138, Bologna
Azienda Unita Locale Socio Sanitaria N 8 Berica
#3653 - Presidio Ospedaliero San Bortolo, Viale Ferdinando Rodolfi 37, 36100, Vicenza
Universitair Medisch Centrum Utrecht
#3750: Hematology, Heidelberglaan 100, 3584 CX, Utrecht
Leids Universitair Medisch Centrum (LUMC)
#3751: Hematology, Albinusdreef 2, 2333 ZA, Leiden
Sykehuset Oestfold HF Kalnes
#3150 - Kreft og hematologisk avdeling, trombose poliklinikk, Kalnesveien 300, 1714, Graalum
Spitalul Clinic Municipal De Urgenta Timisoara
#7055 - Hematology, Strada Dima Gheorghe Nr.5, 300079, Timisoara
Spitalul Clinic Municipal Filantropia Craiova
#7051 - Hematology, Strada Filantropiei No 1, 200143, Craiova
Complexo Hospitalario Universitario De Santiago
#3852: Hematología, Calle Choupana Da S/n, 15706, Santiago De Compostela
Hospital General Universitario Morales Meseguer
#3854: Hematología, Avenida Del Marques De Los Velez S/n, 30008, Murcia
Hospital Universitario De Salamanca
#3855: Hematología, Paseo De San Vicente 58-182, 37007, Salamanca
Hospital Universitario La Paz
#3853: Hematología, Paseo De La Castellana 261, 28046, Madrid
Hospital Del Mar
#3850: Hematología, Passeig Maritim De La Barceloneta 25-29, 08003, Barcelona
Hospital Universitario 12 De Octubre
#3851: Hematología, Bloque D, Avenida De Cordoba Sn, Madrid
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 | 2023-10-16 | 2025-02-27 | 2023-10-16 | 2025-02-27 | |
| Belgium | 2023-07-31 | 2023-07-31 | 2025-02-27 | ||
| Czechia | 2023-11-08 | 2023-11-08 | 2025-02-27 | ||
| France | 2024-07-23 | 2024-07-23 | 2025-02-27 | ||
| Germany | 2023-04-06 | 2023-04-06 | 2025-02-27 | ||
| Hungary | 2023-03-20 | 2023-03-20 | 2025-02-27 | ||
| Italy | 2024-09-23 | 2024-09-23 | 2025-02-27 | ||
| Netherlands | 2023-10-23 | 2023-10-23 | 2025-02-27 | ||
| Romania | 2023-07-24 | 2026-01-30 | 2023-07-24 | 2025-02-27 | |
| Spain | 2024-07-01 | 2024-07-01 | 2025-02-27 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 173 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol - Signature Page_2024-512890-28-00_1_English_Red | v05 |
| Protocol (for publication) | D1_Protocol_2024-512890-28-00_1_English_Red | v5.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_Czech_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_Dutch_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_English_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_French_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_German_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_Hungarian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_Italian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_Romanian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_1_Spanish_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_Czech_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_Dutch_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_English_NonRed | 20Aug2023 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_French_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_German_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_Hungarian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_Italian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_Romanian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_2_Spanish_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_Czech_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_Dutch_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_English_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_French_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_German_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_Hungarian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_Italian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_Romanian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_3_Spanish_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_Czech_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_Dutch_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_English_NonRed | 20Aug2023 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_French_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_German_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_Hungarian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_Italian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_Romanian_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_4_Spanish_NonRed | 1.0 |
| Protocol (for publication) | D4_Patient-facing document - PRO_6_Note to Assessor_English_Red | 27Oct2024 |
| Protocol (for publication) | D4_Patient-facing document - PRO_7_Note to Assessor_English_Red | 27Oct2024 |
| Protocol (for publication) | D4_Patient-facing document - PRO_8_Note to Assessor_English_Red | 27Oct2024 |
| Protocol (for publication) | D4_Patient-facing document - PRO_9_Note to Assessor_English_Red | 27Oct2024 |
| Protocol (for publication) | D4_Patient-facing document -PRO_5_Note to Assessor_English_Red | 27Oct2024 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_AT_English_NonRed | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_BE_English_Red | 14Dec2022 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_CZ_Czech_Red | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_DE_English_NonRed | 01 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_DE_German_Red | 30Mar2023 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_ES_Spanish_NonRed | v5.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_FR_NonRed | 01 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_HU_English_NonRed | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_NL_English_NonRed | V00 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_NO_English_NonRed | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_1_RO_Romanian_NonRed | 28Aug2024 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_2_CZ_Czech_Red | 30Mar2023 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_2_DE_German_NonRed | 01Mar2024 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_3_CZ_Czech_Red | 30Mar2023 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_4_CZ_Czech_NonRed | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_5_CZ_Czech_NonRed | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_6_CZ_Czech_Red | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements - Country_7_CZ_Czech_NonRed | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements-Country _1_IT_Italian_NonRed | 1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_1_AT_German_Red | V2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_1_BE_Dutch_NonRed | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_1_BE_English_NonRed | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_1_BE_French_NonRed | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_1_ES_Spanish_NonRed | 15Oct2024 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_1_FR_French_Red | 01 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_1_HU_Hungarian_Red | 1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_1_NL_Dutch_Red | V1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_2_BE_Dutch_Red | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_2_BE_French_Red | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_2_ES_Spanish_Red | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_2_FR_French_Red | 01 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_2_HU_Hungarian_Red | 1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_3_AT_German_NonRed | V1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_3_BE_Dutch_Red | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_3_BE_French_Red | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_3_ES_Spanish_Red | v2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_3_FR_French_Red | 2.1 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_4_AT_German_Red | V1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_4_BE_Dutch_Red | v2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_4_BE_English_Red | v2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_4_BE_French_Red | v2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_4_ES_Spanish_Red | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_4_FR_French_Red | 02 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_5_BE_Dutch_Red | v2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_5_BE_English_Red | v2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_5_BE_French_Red | v2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_5_ES_Spanish_Red | v2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_5_FR_French_NonRed | 01 |
| Recruitment arrangements (for publication) | K2_Advertisements - Country_6_ES_Spanish_NonRed | v1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements- Country_2_AT_German_Red | V2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements-Country_1_IT_Italian_Red | 2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements-Country_2_IT_Italian_Red | 2.0 |
| Recruitment arrangements (for publication) | K2_Advertisements-Country_3_IT_Italian_Red | 28Aug2023 |
| Recruitment arrangements (for publication) | K2_Advertisements-Country_4_IT_Italian_Red | 28Aug2023 |
| Recruitment arrangements (for publication) | K2_Advertisements-Country_5_IT_Italian_NonRed | 1.0 |
| Recruitment arrangements (for publication) | K2_Advertisements-Country_6_IT_Italian_NonRed | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF - Additional Biomarkers_1_DE_German_NonRed | 04.03.01 |
| Subject information and informed consent form (for publication) | L1_ICF - Child Assent_1_FR_French_NonRed | 00.00.00 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_AT_German_NonRed | 00.00.02 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_CZ_Czech_NonRed | 00.00.01 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_DE_German_NonRed | 00.00.00 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_ES_Spain_NonRed | v00.00.00 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_FR_French_NonRed | 00.00.00 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_HU_Hungarian_NonRed | 00.00.01 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_NL_Dutch_NonRed | V00000001 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_NO_Norwegian_NonRed | 00.00.03 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_1_RO_Romanian_Red | V00.00.01 |
| Subject information and informed consent form (for publication) | L1_ICF - Follow up for pregnant participant_2_HU_Hungarian_NonRed | v00.00.00 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_AT_German_Red | 04.03.00 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_BE_Dutch_Red | v04.03.07 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_BE_English_Red | v04.03.07 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_BE_French_Red | v04.03.07 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_CZ_Czech_Red | 04.03.04 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_DE_German_Red | 04.03.05 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_ES_Spain_Red | v04.03.03 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_FR_French_Red | 04.03.02 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_HU_Hungarian_Red | v04.03.04 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_IT_Italian_Red | 04.03.03 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_NL_Dutch_Red | V04030200 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_NO_Norwegian_Red | 04.03.06 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_1_RO_Romanian_Red | V04.03.04 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_2_CZ_Czech_Red | 04.03.04 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_2_FR_French_Red | 04.03.02 |
| Subject information and informed consent form (for publication) | L1_ICF - Main ICF - Adult_2_HU_Hungarian_NonRed | v04.03.03 |
| Subject information and informed consent form (for publication) | L1_ICF - Optional Assessment_1_CZ_Czech_NonRed | 00.00.00 |
| Subject information and informed consent form (for publication) | L1_ICF - Optional Assessment_1_DE_German_NonRed | 00.00.01 |
| Subject information and informed consent form (for publication) | L1_ICF - Optional Assessment_1_IT_Italian_NonRed | 04.03.03 |
| Subject information and informed consent form (for publication) | L1_ICF - Optional Assessment_1_NL_Dutch_NonRed | V02010001 |
| Subject information and informed consent form (for publication) | L1_ICF - Optional Assessment_2_CZ_Czech_NonRed | 00.00.00 |
| Subject information and informed consent form (for publication) | L1_ICF - Optional1_1_IT_Italian_NonRed | 00.00.01 |
| Subject information and informed consent form (for publication) | L1_ICF - Separate Data Protection Consent_1_CZ_Czech_NonRed | 04.03.02 |
| Subject information and informed consent form (for publication) | L1_ICF - Separate Data Protection Consent_1_ES_Spanish_NonRed | v3 |
| Subject information and informed consent form (for publication) | L1_ICF - Separate Data Protection Consent_2_CZ_Czech_NonRed | 04.03.02 |
| Subject information and informed consent form (for publication) | L1_ICF - Separate Data Protection Consent_2_ES_Spanish_NonRed | v3 |
| Subject information and informed consent form (for publication) | L1_List of submitted documents_1_HU_Hungarian_NonRed | 1.0 |
| Subject information and informed consent form (for publication) | L1_Patient Card_1_German_NonRed | V1.0 |
| Subject information and informed consent form (for publication) | L1_Patient Card_1_Hungarian_Red | 00.00. |
| Subject information and informed consent form (for publication) | L1_Patient Card_1_Italian_NonRed | 1.0 |
| Subject information and informed consent form (for publication) | L1_Patient Card_2_German_NonRed | V1.0 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_1_AT_German_Red | 2.1 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_1_FR_French_NonRed | 01 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_1_IT_Italian_NonRed | 1.0 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_1_RO_Romanian_NonRed | 1 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_2_AT_German_Red | V3.1 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_2_FR_French_NonRed | 01 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_2_IT_Italian_NonRed | 00.00 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_2_RO_Romanian_NonRed | 1 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_3_IT_Italian_NonRed | 00.00 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_3_Ro_Romanian_Red | 2 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_4_Ro_Romanian_Red | 2 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_5_Ro_Romanian_Red | 1 |
| Subject information and informed consent form (for publication) | L1_Subject Info Sheet or Other Info_6_Ro_Romanian_Red | 1 |
| Subject information and informed consent form (for publication) | L2_3751_ICF Procedure_1_NL_Dutch_NonRed | V00 |
| Subject information and informed consent form (for publication) | L2_ICF Procedure_1_BE_English_Red | 1.0 |
| Subject information and informed consent form (for publication) | L2_ICF Procedure_1_DE_English_NonRed | 01 |
| Subject information and informed consent form (for publication) | L2_ICF Procedure_1_ES_Spanish_NonRed | 13Sep2024 |
| Subject information and informed consent form (for publication) | L2_ICF Procedure_1_NO_English_NonRed | 1 |
| Subject information and informed consent form (for publication) | L2_Subject Info Sheet or Other Info_1_NL_Dutch_Red | V2.0 |
| Subject information and informed consent form (for publication) | L2_Subject Info Sheet or Other Info_2_NL_Dutch_Red | V2.0 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Local SmPC_1_Eltrombopag_RO_Romanian_NonRed | 06Nov2025 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_Reference SmPC_1_Eltrombopag_English_NonRed | 06Nov2025 |
| Synopsis of the protocol (for publication) | D1_Protocol - Protocol Summary in Technical Language_2024-512890-28-00_1_Czech_Red | 04 |
| Synopsis of the protocol (for publication) | D1_Protocol - Protocol Summary in Technical Language_2024-512890-28-00_1_French_Red | 03 |
| Synopsis of the protocol (for publication) | D1_Protocol - Protocol Summary in Technical Language_2024-512890-28-00_1_German_Red | V05 |
| Synopsis of the protocol (for publication) | D1_Protocol - Protocol Summary in Technical Language_2024-512890-28-00_1_Hungarian_Red | 05.01 |
| Synopsis of the protocol (for publication) | D1_Protocol - Protocol Summary in Technical Language_2024-512890-28-00_1_Italian_Red | 05.00 |
| Synopsis of the protocol (for publication) | D1_Protocol - Protocol Summary in Technical Language_2024-512890-28-00_1_Romanian_Red | 05 |
| Synopsis of the protocol (for publication) | D1_Protocol - Protocol Summary in Technical Language_2024-512890-28-00_1_Spanish_Red | 05 |
| Synopsis of the protocol (for publication) | D1_Protocol Summary in Lay Language_2024-512890-28-00_1_Dutch_Red | V00 |
| Synopsis of the protocol (for publication) | D1_Protocol Summary in Lay Language_2024-512890-28-00_1_Norwegian_Red | 01 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-07-10 | Germany | Acceptable with conditions 2024-08-07
|
2024-08-08 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-11-12 | Germany | Acceptable 2025-03-03
|
2025-03-03 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-04-16 | Germany | Acceptable 2025-07-11
|
2025-07-11 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-10-24 | Acceptable 2025-07-11
|
2025-10-24 | |
| 5 | SUBSTANTIAL MODIFICATION | SM-3 | 2026-01-14 | Germany | Acceptable 2026-03-16
|
2026-03-16 |