Romiplostim Plus Dexamethasone vs Dexamethasone in Patients With Newly Diagnosed Primary Immune Thrombocytopenia

NCT ID: NCT05325593

Last Updated: 2025-06-17

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 129 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-12-02
• Study Completion Date: 2026-10-24

Brief Summary

Phase III, open-labeled, randomized and multicenter clinical trial to evaluate the superiority of romiplostim plus dexamethasone vs dexamethasone alone in patients with newly diagnosed primary immune thrombocytopenia

Detailed Description

The main objective of the study is to evaluate the superiority of romiplostim plus dexamethasone versus dexamethasone alone in the treatment of primary immune thrombocytopenia, with sustained response to any ITP treatment and without World Health Organization grade 2 or higher bleeding, after six months from cessation of treatment.

Maximum time on treatment with romiplostim will be 12 months (365 days). Then, patients will be followed up for 6 additional months (180 days) after stopping romiplostim.

Clinical rules are included if romiplostim dose should be modified or finished. In case of dexamethasone, no dose adjustment is permitted.

The evaluation of romiplastim plus dexamethasone´s superiority in different periods and platelet count, proportion of patients with complete response (CR), global response (GR), early response (ER) and initial response (IR); time to loss of response (LoR), adverse events, quality of life and healthcare resources use are included as secondary objectives.

Conditions

Primary Immune Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

romiplostim plus dexamethasone (ROM + DEX)

Dexamethasone 40 mg daily x 4 days only in the first cycle and subcutaneous romiplostim weekly for up to 12 months

Romiplostim:

1. The starting dose should be 3 mcg/kg/week. It could be start during de 4 days of dexamethasone.

2. Patients will weekly receive dose increases of romiplostim in increments of 1 mcg/kg up to a maximum dose of 10 mcg/kg in an attempt to reach a target platelet count higher than 50x109/L.

3. Otherwise, if platelets are lower than 50x109/L treatment with romiplostim will go on until Day 365 since randomization.

Group Type: EXPERIMENTAL

romiplostim plus dexamethasone

Intervention Type: DRUG

Patients will be reviewed weekly for 8 weeks (56 days). After Week 8, patients will be reviewed every 2 weeks (14 days) for 8 additional weeks and then monthly until Week 52 (365 days) from randomization.

Dexamethasone (DEX)

Dexamethasone 40 mg daily x 4 days for up to 3 cycles every 14 to 28 days

Group Type: ACTIVE_COMPARATOR

Dexamethasone

Intervention Type: DRUG

Patients will be reviewed weekly until the completion of dexamethasone cycles and for a minimum of 8 weeks (56 days). After that, every 2 weeks (14 days) for 8 additional weeks and then monthly until Week 52 (365 days) from randomization.

Interventions

romiplostim plus dexamethasone

Patients will be reviewed weekly for 8 weeks (56 days). After Week 8, patients will be reviewed every 2 weeks (14 days) for 8 additional weeks and then monthly until Week 52 (365 days) from randomization.

Intervention Type: DRUG

Dexamethasone

Patients will be reviewed weekly until the completion of dexamethasone cycles and for a minimum of 8 weeks (56 days). After that, every 2 weeks (14 days) for 8 additional weeks and then monthly until Week 52 (365 days) from randomization.

Intervention Type: DRUG

Other Intervention Names

ROM + DEX; DEX

Eligibility Criteria

Inclusion Criteria

1. Age ≥ 18 years of age at the time of signing informed consent.

2. Newly diagnosis of primary ITP according to the International Working Group assessment [1] and previously untreated for ITP.

3. Platelet counts <30x109/L or ITP with platelet counts <50x109/L and concomitant bleeding symptoms.

4. Serum creatinine concentration ≤1.5 mg/dL.

Exclusion Criteria

1. World Health Organization's performance status >2.

2. Previous therapy with rituximab (within 3 months previous of study enrollment), corticosteroids or, therapy with other immunomodulating agents within 1 month before of enrolment;,prior use of hematopoietic analogs and or fostamatinib for any other reason despite ITP three months before enrolment.

3. Previous use of romiplostim, polyethylene glycol-recombinant human megakaryocyte growth and development factor, Eltrombopag, recombinant human anti-thrombopoietin, or any platelet-producing agent three months before enrolment.

4. Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study.

5. Splenectomy within 3 months of the screening visit or planned splenectomy during study period.

6. Abnormal renal function (serum creatinine > 1.5 mg/dL).

7. Active hepatic disease (evidenced by alanine aminotransferase [ALT] or aspartate aminotransferase [AST] levels >5 times the upper limit of normal (it will only be necessary to determine one of the two transaminases

8. Severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio >1.4, hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.

9. Patients with known immunoglobulin M seropositive tests for cytomegalovirus and/or Epstein-Barr virus in the previous month.

10. Patients with an active viral infection at screening with: Hepatitis B Virus, Hepatitis C Virus, detectable virus charge of HIV.

11. Intolerance to dexamethasone.

12. History of a bone marrow stem cell disorder.

13. Active or prior malignancy except adequately treated (ie, complete surgical excision with negative margins) basal cell carcinoma.

14. History of helicobacter pylori by urea breath test or stool antigen test within 6 months of enrollment, if available.

15. History of myelodysplastic syndrome, systemic lupus erythematosus, or autoimmune cytopenia.

16. History of antiphospholipid antibody syndrome.

17. History of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura.

18. History of deep or superficial venous thromboembolism in the last 12 months or stroke, acute ischaemic heart disease or acute peripheral vascular disease in the last 6 months.

19. Hypersensitivity to any recombinant Escherichia coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune) or known sensitivity to any of the products to be administered during dosing

20. Currently enrolled in another investigational device or drug study or < 30 days since ending another investigational device or drug studies, or receiving other investigational agents.

21. Will have any other investigational procedures performed while enrolled in this clinical study.

22. Pregnant or breastfeeding, or planning to become pregnant or breastfeed during treatment or within 1 month after the end of treatment.

23. Female subject of childbearing potential is not willing to use, in combination with her partner, an acceptable method of effective contraception during treatment and for 1 month after the end of treatment. Females of childbearing potential should only be included after a negative, pregnancy test.

24. Will not be available for protocol-required study visits, to the best of the subject's and investigator's knowledge.

25. Any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures.

26. Other serious comorbidities at investigator criteria.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fundación Pública Andaluza para la gestión de la Investigación en Sevilla

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Charlotte Bradbury

Role: PRINCIPAL_INVESTIGATOR

Centre for Trials Research College of Biomedical & Life Sciences Cardiff University

Locations

IRCCS AOU di Bologna, Seràgnoli Institute of Hematology

Bologna, , Italy

ASST Fatebenefratelli Sacco - Ospedale L. Sacco

Milan, , Italy

Grande Ospedale Metropolitano Niguarda

Milan, , Italy

Azienda Ospedaliero-Universitaria Policlinico Umberto I / SAPIENZA Universitá di Roma

Rome, , Italy

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Rome, , Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, , Italy

Hospital del Mar

Barcelona, Barcelona, Spain

Centre Sociosanitari Sant Jordi de la Vall D'Hebron

Barcelona, Barcelona, Spain

Complejo Asistencial Universitario de Burgos

Burgos, Burgos, Spain

Complejo Hospitalario Universitario A Coruña

A Coruña, Coruña, Spain

Hospital Universitario Virgen de las Nieves

Granada, Granada, Spain

Hospital General Universitario Gregorio Marañón

Madrid, Madrid, Spain

Compejo Hospitalario La Paz

Madrid, Madrid, Spain

Hospital Universitario Fundación Alcorcon

Madrid, Madrid, Spain

Hospital Universitario Morales Meseguer

Murcia, Murcia, Spain

Hospital Clínico Universitario Virgen de la Arrixaca

Murcia, Murcia, Spain

Hospital Universitario Virgen de la Victoria

Málaga, Málaga, Spain

Complejo Asistencial Universitario de Salamanca

Salamanca, Salamanca, Spain

Hospital Universitario Virgen del Rocío

Seville, Sevilla, Spain

Hospital Universitario y Pilitécnico La Fe

Valencia, Valencia, Spain

Complejo Asistencial Son Espases

Palma de Mallorca, , Spain

University Hospitals Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

University Hospitals Bristol and Weston NHS Trust

Bristol, , United Kingdom

Haemophilia and Thrombosis Centre, Kent & Canterbury Hospital, Kent & Canterbury Hospital, Ethelbert Road, Canterbury, CT1 3NG, England, UK

Canterbury, , United Kingdom

Greater Glasgow and Clyde Health Board

London, , United Kingdom

Haematology, Royal Victoria Infirmary

Newcastle, , United Kingdom

Norfolk & Norwich University Hospital Trust

Norwich, , United Kingdom

Oxford University Hospitals NHS Foundation Trust

Oxford, , United Kingdom

"Haematology, Derriford Hospital, University Hospitals Plymouth NHS Trust

Plymouth, , United Kingdom

Torbay and South Devon NHS Foundation Trust

Torquay, , United Kingdom

Countries

Italy; Spain; United Kingdom

Other Identifiers

2024-514147-28-00

Identifier Type: CTIS

Identifier Source: secondary_id

2021-006970-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

RODEX

Identifier Type: -

Identifier Source: org_study_id