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

NCT05325593 | Active Not Recruiting Phase 3

• 3 other identifiers: RODEX2024-514147-28-002021-006970-22
• Study Type: interventional
• Target: 129
• Locations: 3 countries
• Sites: 30

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

Conditions

Primary Immune Thrombocytopenia

Keywords

Romiplostim; Dexamethasone

Outcome Measures

Primary Outcomes (1)

• Proportion of patients achieving sustain response out of treatment with platelets higher or equal than 30x109/L for 6 months (Sustained Response Off any ITP Treatment)

  Proportion of patients with platelets higher or equal than 50x109/L in the absence of any ITP treatment including any rescue treatment for at least 6 consecutive months (≥180 days) from treatment cessation and without World Health Organization grade 2 or more bleeding

  180 days after treatment withdrawal

Secondary Outcomes (27)

• Proportion of patients achieving sustain response out of treatment with platelets higher or equal than 30x109/L for 6 months in the absence of any ITP treatment including any rescue treatment.

  180 days after treatment withdrawal

• Proportion of patients achieving sustain response out of treatment with platelets higher or equal than 30x109/L for 12 months in the absence of any ITP treatment including any rescue treatment.

  365 days after treatment withdrawal

• Proportion of patients achieving sustain response out of treatment with platelets higher or equal than 50x109/L for 12 months in the absence of any ITP treatment including any rescue treatment.

  365 days after treatment withdrawal

• Proportion of patients with early response (ER)

  Day 7

• Proportion of patients with initial response (IR)

  Day 30

Study Arms (2)

romiplostim plus dexamethasone (ROM + DEX)

EXPERIMENTAL

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.

Drug: romiplostim plus dexamethasone

Dexamethasone (DEX)

ACTIVE COMPARATOR

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

Drug: Dexamethasone

Interventions

romiplostim plus dexamethasone 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.

Also known as: ROM + DEX

romiplostim plus dexamethasone (ROM + DEX)

Dexamethasone 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.

Also known as: DEX

Dexamethasone (DEX)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years of age at the time of signing informed consent.
• Newly diagnosis of primary ITP according to the International Working Group assessment \[1\] and previously untreated for ITP.
• Platelet counts \<30x109/L or ITP with platelet counts \<50x109/L and concomitant bleeding symptoms.
• Serum creatinine concentration ≤1.5 mg/dL.

You may not qualify if:

• World Health Organization's performance status \>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.
• 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.
• Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study.
• Splenectomy within 3 months of the screening visit or planned splenectomy during study period.
• Abnormal renal function (serum creatinine \> 1.5 mg/dL).
• 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
• 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.
• Patients with known immunoglobulin M seropositive tests for cytomegalovirus and/or Epstein-Barr virus in the previous month.
• Patients with an active viral infection at screening with: Hepatitis B Virus, Hepatitis C Virus, detectable virus charge of HIV.
• Intolerance to dexamethasone.
• History of a bone marrow stem cell disorder.
• Active or prior malignancy except adequately treated (ie, complete surgical excision with negative margins) basal cell carcinoma.
• History of helicobacter pylori by urea breath test or stool antigen test within 6 months of enrollment, if available.
• History of myelodysplastic syndrome, systemic lupus erythematosus, or autoimmune cytopenia.

Sponsors & Collaborators

• Fundación Pública Andaluza para la gestión de la Investigación en Sevilla: lead

Study Sites (30)

IRCCS AOU di Bologna, Seràgnoli Institute of Hematology

Bologna, Italy

Location

ASST Fatebenefratelli Sacco - Ospedale L. Sacco

Milan, Italy

Location

Grande Ospedale Metropolitano Niguarda

Milan, Italy

Location

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

Rome, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Rome, Italy

Location

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, Italy

Location

Hospital del Mar

Barcelona, Barcelona, 08003, Spain

Location

Centre Sociosanitari Sant Jordi de la Vall D'Hebron

Barcelona, Barcelona, 08035, Spain

Location

Complejo Asistencial Universitario de Burgos

Burgos, Burgos, 09006, Spain

Location

Complejo Hospitalario Universitario A Coruña

A Coruña, Coruña, 15006, Spain

Location

Hospital Universitario Virgen de las Nieves

Granada, Granada, 18014, Spain

Location

Hospital General Universitario Gregorio Marañón

Madrid, Madrid, 28007, Spain

Location

Compejo Hospitalario La Paz

Madrid, Madrid, 28046, Spain

Location

Hospital Universitario Fundación Alcorcon

Madrid, Madrid, 28922, Spain

Location

Hospital Universitario Morales Meseguer

Murcia, Murcia, 3008, Spain

Location

Hospital Clínico Universitario Virgen de la Arrixaca

Murcia, Murcia, 30120, Spain

Location

Hospital Universitario Virgen de la Victoria

Málaga, Málaga, 29010, Spain

Location

Complejo Asistencial Universitario de Salamanca

Salamanca, Salamanca, 37007, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, Sevilla, 41013, Spain

Location

Hospital Universitario y Pilitécnico La Fe

Valencia, Valencia, 46026, Spain

Location

Complejo Asistencial Son Espases

Palma de Mallorca, 07120, Spain

Location

University Hospitals Birmingham NHS Foundation Trust

Birmingham, United Kingdom

Location

University Hospitals Bristol and Weston NHS Trust

Bristol, United Kingdom

Location

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

Canterbury, United Kingdom

Location

Greater Glasgow and Clyde Health Board

London, United Kingdom

Location

Haematology, Royal Victoria Infirmary

Newcastle, United Kingdom

Location

Norfolk & Norwich University Hospital Trust

Norwich, United Kingdom

Location

Oxford University Hospitals NHS Foundation Trust

Oxford, United Kingdom

Location

"Haematology, Derriford Hospital, University Hospitals Plymouth NHS Trust

Plymouth, United Kingdom

Location

Torbay and South Devon NHS Foundation Trust

Torquay, United Kingdom

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

romiplostim; Dexamethasone

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Study Officials

• Charlotte Bradbury

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Parallel Assignment Randomized 1:1

Study Record Dates

• First Submitted: March 7, 2022
• First Posted: April 13, 2022
• Study Start: December 2, 2022
• Primary Completion (Estimated): October 24, 2026
• Study Completion (Estimated): October 24, 2026
• Last Updated: June 17, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: Along the study
• Access Criteria: Direct collaborators within the study

Locations

GB (9); IT (6); ES (15)