Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
50
Countries
1
Sites
1
Immune thrombocytopenia
Evaluation of the effectiveness of treatment of a four-combination of evaluated medicinal products in patients with immune thrombocytopenia.
Key facts
- Sponsor
- Fakultni Nemocnice Hradec Kralove
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20], Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 11 Jun 2025 → ongoing
- Decision date (initial)
- 2023-06-22
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
- Funding sources
- Fakultní nemocnice Hradec Králové - Internal grant competion
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety, Others
Evaluation of the effectiveness of treatment of a four-combination of evaluated medicinal products in patients with immune thrombocytopenia.
Secondary objectives 4
- Evaluation of the safety of the treatment of the four-combination of evaluated medicinal products in patients with immune thrombocytopenia
- Comparison of patients' quality of life before, during and after treatment
- Determination of the level of biomarkers (kynurenine, neopterin, tryptophan) and their changes in connection with treatment
- Evaluation of possible predictive parameters of response to therapy
Conditions and MedDRA coding
Immune thrombocytopenia
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 23.0 | PT | 10083842 | Immune thrombocytopenia | 100000004851 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 8
- Age ≥ 18 years - 70 years
- Ability to understand the nature and course of the study and sign written informed consent to participate in the study.
- Ability and willingness to complete study visit and commute to study center.
- Diagnosis of primary immune thrombocytopenia.
- Platelets count below 80x109/l (after termination or on corticotherapy) and fulfillment of the indication for therapy (bleeding, need for anticoagulation, constitutional symptoms, documented refractoriness when stopping corticoids, etc.).
- Previous corticosteroid therapy lasting at least 14 days (pulse or continuous), after which, according to the doctor's decision, it is necessary/appropriate to continue ITP therapy.
- Women of childbearing potential must have a negative pregnancy test (from blood serum) and use effective methods of contraception before starting treatment.
- For women of childbearing potential: Willingness to prevent pregnancy and breast-feeding during treatment and for 12 months after the last dose of rituximab, using at least one of the highly reliable methods of contraception. For men: Willingness to prevent paternity during treatment and for the next 90 days after stopping mycophenolate treatment.
Exclusion criteria 19
- Severe bleeding (grade 2 or more according to CTCAE), which requires a more intensive approach (hospitalization, IVIG).
- Active cancer in the last 5 years
- Previous therapy: • Previous rituximab therapy in the last 12 months. • IVIG therapy or plasmapheresis in the last 28 days before the 1st study treatment administration (visit M1D1). • TPO-RA therapy, fostamatinib, immunosuppressants (MMF, CSA) in the last 28 days before the 1st study treatment administration (visit M1D1). • Cytostatic therapy in the last 28 days before the 1st study treatment administration (visit M1D1).
- Participation in a clinical study involving administration of investigational medicinal product within 3 months (or 5 half-terms, whichever is longer) prior to screening.
- Laboratory findings more than 1.5 x upper limit - ALT, AST, GMT, ALP, creatine, PT, aPTT.
- Uncontrolled arterial hypertension.
- Infectious disease in the last 14 days before starting the study treatment (visit M1D1).
- Planned surgical procedure in the next 6 months, or until visit M6.
- Splenectomy in the last 3 months.
- The presence of another significant disease limiting therapy (liver disease, GIT disease, psychiatric disease /suicide, depression/, etc.)
- Contraindications to administered drugs (simultaneous use of contraindicated drugs, HIV, hepatitis, liver disease, etc.)
- Breastfeeding, pregnancy, trying to get pregnant
- Myelodysplastic syndrome (MDS) or other abnormalities in the blood count, except for thrombocytopenia (a sternal puncture may be necessary to exclude MDS).
- History of Thrombocytopenic thrombotic purpura (TTP), Hemolytic-uremic syndrome (HUS), antiphospholipid syndrome (AFS) or acquired hemophilia
- Immunodeficiency
- History of anaphylaxis
- Vaccination in the last 28 days before the 1st administration of study treatment (visit M1D1).
- Neuropathy
- One other severe autoimmune disease (SLE, AFS, Crohn's disease, ulcerative colitis, ...) or a combination of 2 or more less severe autoimmune diseases
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Platelets count at visit M3, M7, M12 and M18,
- Number of patients in remission without the need for therapy at visit M7, M12, M18
Secondary endpoints 5
- The number of relapses within 12 months of the end of therapy
- The number of patients who complete the entire cycle
- The results of the standardized SF-36 quality of life questionnaire before starting, during and after treatment
- Biomarker levels (kynurenine, neopterin, tryptophan) and their changes in connection with treatment
- values of CD4+/CD8+ T lymphocytes and their cytokine production (IL-4, IL-17A, IFNγ), regulatory CD4+ T lymphocytes and anti-platelet antibodies as possible predictive parameters of response to therapy
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 11
Revolade 50 mg film-coated tablets
PRD3045769 · Product
- Active substance
- Eltrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 14 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BX05 — -
- Marketing authorisation
- EU/1/10/612/004
- MA holder
- NOVARTIS EUROPHARM LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Doptelet 20 mg film-coated tablets
PRD8435882 · Product
- Active substance
- Avatrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 20 mg milligram(s)
- Max total dose
- 20 mg milligram(s)
- Max treatment duration
- 14 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BX08 — -
- Marketing authorisation
- EU/1/19/1373/002
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Revolade 50 mg film-coated tablets
PRD3045770 · Product
- Active substance
- Eltrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 14 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BX05 — -
- Marketing authorisation
- EU/1/10/612/005
- MA holder
- NOVARTIS EUROPHARM LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Nplate 250 micrograms powder for solution for injection
PRD3613282 · Product
- Active substance
- Romiplostim
- Substance synonyms
- AMG 531
- Pharmaceutical form
- POWDER FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 35.7 µg microgram(s)
- Max total dose
- 250 µg microgram(s)
- Max treatment duration
- 14 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BX04 — -
- Marketing authorisation
- EU/1/08/497/003
- MA holder
- AMGEN EUROPE B.V.
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Nplate 250 micrograms powder for solution for injection
PRD3613280 · Product
- Active substance
- Romiplostim
- Substance synonyms
- AMG 531
- Pharmaceutical form
- POWDER FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 35.7 µg microgram(s)
- Max total dose
- 250 µg microgram(s)
- Max treatment duration
- 14 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BX04 — -
- Marketing authorisation
- EU/1/08/497/001
- MA holder
- AMGEN EUROPE B.V.
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Doptelet 20 mg film-coated tablets
PRD8668320 · Product
- Active substance
- Avatrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 20 mg milligram(s)
- Max total dose
- 20 mg milligram(s)
- Max treatment duration
- 14 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BX08 — -
- Marketing authorisation
- EU/1/19/1373/003
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Revolade 50 mg film-coated tablets
PRD3045771 · Product
- Active substance
- Eltrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 14 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BX05 — -
- Marketing authorisation
- EU/1/10/612/006
- MA holder
- NOVARTIS EUROPHARM LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Doptelet 20 mg film-coated tablets
PRD8435879 · Product
- Active substance
- Avatrombopag
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 20 mg milligram(s)
- Max total dose
- 20 mg milligram(s)
- Max treatment duration
- 14 Week(s)
- Authorisation status
- Authorised
- ATC code
- B02BX08 — -
- Marketing authorisation
- EU/1/19/1373/001
- MA holder
- SWEDISH ORPHAN BIOVITRUM AB (PUBL)
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB06250MIG · Substance
- Active substance
- Ciclosporin
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 200 mg milligram(s)
- Max total dose
- 200 mg milligram(s)
- Max treatment duration
- 7 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB12570MIG · Substance
- Active substance
- Rituximab
- Pharmaceutical form
- INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 100 mg/m2 milligram(s)/sq. meter
- Max total dose
- 100 mg/m2 milligram(s)/sq. meter
- Max treatment duration
- 2 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB03360MIG · Substance
- Active substance
- Mycophenolate Mofetil
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 1000 mg milligram(s)
- Max total dose
- 1000 mg milligram(s)
- Max treatment duration
- 7 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Fakultni Nemocnice Hradec Kralove
- Sponsor organisation
- Fakultni Nemocnice Hradec Kralove
- Address
- Sokolska 581
- City
- Novy Hradec Kralove
- Postcode
- 500 03
- Country
- Czechia
Scientific contact point
- Organisation
- Fakultni Nemocnice Hradec Kralove
- Contact name
- MUDr. Milan Košťál, Ph.D.
Public contact point
- Organisation
- Fakultni Nemocnice Hradec Kralove
- Contact name
- Mgr. Johana Krempova
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Czechia | Ongoing, recruiting | 50 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Fakultni Nemocnice Hradec Kralove
IV. interní hematologická klinika, Sokolska 581, 500 03, Novy Hradec Kralove
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 |
|---|---|---|---|---|---|
| Czechia | 2025-06-11 | 2025-06-11 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 13 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | TMEM protokol | 1.1 |
| Recruitment arrangements (for publication) | Sablona 1 Nabor | 1 |
| Subject information and informed consent form (for publication) | GDPR souhlas pro akademicka klinicka hodnoceni | 2.0 |
| Subject information and informed consent form (for publication) | TMEM Dotaznik SF36 | 1 |
| Subject information and informed consent form (for publication) | TMEM Informace pro pacienta a IS | 2.0 |
| Subject information and informed consent form (for publication) | TMEM Karticka pacienta Verze 1 10MAY2023 | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | Doptelet SmPC | 3 |
| Summary of Product Characteristics (SmPC) (for publication) | Equoral SmPC | 3 |
| Summary of Product Characteristics (SmPC) (for publication) | MabThera SmPC | 3 |
| Summary of Product Characteristics (SmPC) (for publication) | Mycophenolate mofetil Teva SmPC | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | Nplate SmPC | 3 |
| Summary of Product Characteristics (SmPC) (for publication) | Revolade SmPC | 3 |
| Synopsis of the protocol (for publication) | TMEM Synopse protokolu | 1.1 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-05-21 | Czechia | Acceptable 2023-06-22
|
2023-06-22 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-09-23 | Czechia | Acceptable 2024-11-22
|
2024-11-25 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-05-23 | Czechia | Acceptable 2025-05-28
|
2025-05-29 |