Romiplostim combined with dexamethasone for treating newly diagnosed primary immune thrombocytopenia

A Multicentre, Randomized, Open-label Study of Romiplostim Plus Dexamethasone vs Dexamethasone in Patients With Newly Diagnosed Primary Immune Thrombocytopenia

This study is testing if adding romiplostim to dexamethasone can help people recently diagnosed with primary immune thrombocytopenia have better results without bleeding compared to just using dexamethasone alone.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 126 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Fundación Pública Andaluza para la gestión de la Investigación en Sevilla (Academic / other)
• Drugs / interventions: rituximab, fostamatinib
• Locations: 15 sites (Barcelona and 14 other locations)
• Trial ID: NCT05325593 on ClinicalTrials.gov

What this trial studies

This Phase III clinical trial aims to evaluate the effectiveness of romiplostim in combination with dexamethasone compared to dexamethasone alone in patients who have recently been diagnosed with primary immune thrombocytopenia (ITP). The study will assess the superiority of the combination treatment in achieving sustained response without significant bleeding after six months of treatment cessation. Participants will receive romiplostim for up to 12 months, followed by a six-month follow-up period. Secondary objectives include evaluating platelet counts, response rates, adverse events, quality of life, and healthcare resource utilization.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older who have newly diagnosed primary immune thrombocytopenia and have not received prior treatment for this condition.

Not a fit: Patients with a performance status greater than 2 or those who have previously received certain treatments for ITP may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a more effective management option for patients with primary immune thrombocytopenia, potentially leading to better outcomes and quality of life.

How similar studies have performed: Previous studies have shown promising results with similar treatment approaches, indicating potential for success in this trial.

Eligibility criteria

Main 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.

Main 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.

Where this trial is running

Barcelona and 14 other locations

• Hospital del Mar — Barcelona, Spain (Recruiting)
• Centre Sociosanitari Sant Jordi de la Vall D'Hebron — Barcelona, Spain (Not_yet_recruiting)
• Complejo Asistencial Universitario de Burgos — Burgos, Spain (Recruiting)
• Complejo Hospitalario Universitario A Coruña — Coruña, Spain (Not_yet_recruiting)
• Hospital Universitario Virgen de las Nieves — Granada, Spain (Not_yet_recruiting)
• Hospital General Universitario Gregorio Marañón — Madrid, Spain (Not_yet_recruiting)
• Compejo Hospitalario La Paz — Madrid, Spain (Not_yet_recruiting)
• Hospital Universitario Fundación Alcorcon — Madrid, Spain (Not_yet_recruiting)
• Hospital Universitario Morales Meseguer — Murcia, Spain (Recruiting)
• Hospital Clínico Universitario Virgen de la Arrixaca — Murcia, Spain (Not_yet_recruiting)
• Hospital Universitario Virgen de la Victoria — Málaga, Spain (Recruiting)
• Complejo Asistencial Son Espases — Palma De Mallorca, Spain (Recruiting)
• Complejo Asistencial Universitario de Salamanca — Salamanca, Spain (Not_yet_recruiting)
• Hospital Universitario Virgen del Rocío — Sevilla, Spain (Recruiting)
• Hospital Universitario y Pilitécnico La Fe — Valencia, Spain (Not_yet_recruiting)

Study contacts

• Principal investigator: Charlotte Bradbury — Centre for Trials Research College of Biomedical & Life Sciences Cardiff University
• Study coordinator: Clara M Rosso Fernández, MD-PhD

Conditions Primary Immune Thrombocytopenia; Romiplostim; Dexamethasone