Romiplostim Plus Dexamethasone vs Dexamethasone in Patients With Newly Diagnosed Primary Immune Thrombocytopenia
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- Registration Number
- NCT05325593
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 126
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description romiplostim plus dexamethasone (ROM + DEX) romiplostim plus dexamethasone 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. Dexamethasone (DEX) Dexamethasone Dexamethasone 40 mg daily x 4 days for up to 3 cycles every 14 to 28 days
- Primary Outcome Measures
Name Time Method 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) 180 days after treatment withdrawal 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
- Secondary Outcome Measures
Name Time Method 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 with platelets higher or equal than 30x109/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.
Proportion of patients with complete response (CR) Day 180, Day 365, Day 545 Patients with platelet count ≥100x109/L and absence of bleeding symptoms.
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 platelets higher or equal than 50x109/L in the absence of any ITP treatment including any rescue treatment for at least 12 consecutive months (≥365 days) from treatment cessation and without World Health Organization grade 2 or more bleeding.
Maximum number of consecutive days with platelet complete response (CR) In every study visit, assessed up to 545 days - The maximum number of consecutive days with platelet count ≥100x109/L in the total sample and in the absence of any rescue treatment
Proportion of patients with initial response (IR) Day 30 Proportion of patients with platelet count higher or equal than 30x109/L
Proportion of patients with response (R) Day 180, Day 365, Day 545 Patients with platelet count between 100x109/L and 30x109/L and at least doubled from baseline and absence of bleeding symptoms.
Changes in patients' quality of life- Short Form-36 Health Survey Day 1, Week 8, Day 180, Day 365 and Day 545 For the assessment of the quality of life during the study will be used Short-Form-36 Health Survey: is a 36-item scale constructed to survey health-related 8 domains: limitations in physical activities due to health problems; limitations in social activities due to physical or emotional problems; limitations unusual role activities due to physical health problems; bodily pain; general mental health (psychological distress and well-being); limitations unusual role activities due to emotional problems; vitality (energy and fatigue); and general health perceptions.
Total number of days with platelet targeted range (TR) In every study visit, assessed up to 545 days The total number od days with platelet count between ≥30x109/L and ≤400x109/ L in the total sample and in the absence of any rescue treatment
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 with platelets higher or equal than 30x109/L in the absence of any ITP treatment including any rescue treatment for at least 12 consecutive months (≥365 days) from treatment cessation and without World Health Organization grade 2 or more bleeding.
Proportion of patients with early response (ER) Day 7 Proportion of patients with platelet count higher or equal than 30x109/L and at least double than baseline.
Proportion of patients with targeted range (TR) Day 180, Day 365, Day 545 Patients with platelet count between ≥30x109/L and ≤400x109/L.
Changes in patients bleeding Screning, Day 1, Week 8, Week 12, Moth 6, Moth 12 and End of study Visit For the assessment of the of the patients' bleeding will be used immune thrombocytopenia-bleeding assessment tool (ITP-BAT): bleeding signs/symptoms are grouped in three domains (skin, visible mucosae and organs) and is graded from 0 (No) to 4.
Loss of productivity In every study visit, assessed up to 545 days Number of days of absenteeism from school or work and associated cost
Total number of days with platelet complete response (CR) In every study visit, assessed up to 545 days The total number od days with platelet count ≥100x109/L in the total sample and in the absence of any rescue treatment
Proportion of patients requiring any rescue treatment In every study visit, assessed up to 545 days Proportion of patients who need rescue treatments in each arm and total patients
Proportion and time to treatment failures In every study visit, assessed up to 545 days Proportion of patients who need rescue treatments and And the number of days they needed treatment
Changes in patients' quality of life- FACIT-F Day 1, Week 8, Day 180, Day 365 and Day 545 For the assessment of the quality of life during the study will be used FACIT-F (Fatigue Scale): is a short scale, 13-item, easy to administer tool that measures an individual's level of fatigue during their usual daily activities over the past week.The level of fatigue is measured by recording item responses ona 4-point Likert scale ranging from 0 "not at all" to 4 "very much.
Changes in patients' quality of life- ITP-Patient Assessment Questionnaire Day 1, Week 8, Day 180, Day 365 and Day 545 For the assessment of the quality of life during the study will be used ITP-Patient Assessment Questionnaire): is a disease-specific instrument that was designed to measure the QoL of adult patients with immune thrombocytopenia. The instrument comprises 38 items completed by male respondents and 44 items completed by female respondents.
Healthcare resources use (HRU) In every study visit, assessed up to 545 days The data collected may be used to conduct exploratory economic analyses and may include:
* Number of outpatient visits,
* Number of home health care
* Number of Hospitalization
* Duration of medical care visits (days)
* Number of Emergency room visits
* Number of diagnostic procedures
* Number of medical care visitsMaximum number of consecutive days with platelet response In every study visit, assessed up to 545 days - The maximum number of consecutive days with platelet count between 100x109/L and 30x109/L in the total sample and in the absence of any rescue treatment
Total number of days with platelet response In every study visit, assessed up to 545 days The total number od days with platelet count ≥100x109/L in the total sample and in the absence of any rescue treatment
Maximum number of consecutive days with platelet global response (GR) In every study visit, assessed up to 545 days The maximum number of consecutive days with platelet count ≥100x109/L or platelet count between 100x109/L and 30x109/L in the total sample and in the absence of any rescue treatment
Proportion of patients with global response (GR) Day 180, Day 365, Day 545 Patients with platelet count ≥100x109/L and absence of bleeding symptoms or platelet count between 100x109/L and 30x109/L and at least doubled from baseline and absence of bleeding symptoms.
Time to loss of response (LoR) in patients who achieved response in both arms. In every study visit, assessed up to 545 days Number of days from the first time the patient achieved a platelet count ≥30x109/L until platelet count dropped below 30x109/L measured on 2 occasions with more than 1 day apart or presence of bleeding
Proportion of patients with adverse events (AEs), including serious adverse events (SAEs) and laboratory safety parameters. In every study visit, assessed up to 545 days AEs will be graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Bleeding events will be carefully monitored
Maximum number of consecutive days with platelet targeted range (TR) In every study visit, assessed up to 545 days The maximum number of consecutive days with platelet count between ≥30x109/L and ≤400x109/ L in the total sample and in the absence of any rescue treatment
Total number of days with platelet global response (GR In every study visit, assessed up to 545 days The total number od days with platelet count ≥100x109/L or platelet count between 100x109/L and 30x109/L in the total sample and in the absence of any rescue treatment
Trial Locations
- Locations (15)
Hospital del Mar
🇪🇸Barcelona, Spain
Centre Sociosanitari Sant Jordi de la Vall D'Hebron
🇪🇸Barcelona, Spain
Complejo Asistencial Universitario de Burgos
🇪🇸Burgos, Spain
Complejo Hospitalario Universitario A Coruña
🇪🇸Coruña, Spain
Hospital General Universitario Gregorio Marañón
🇪🇸Madrid, Spain
Compejo Hospitalario La Paz
🇪🇸Madrid, Spain
Hospital Universitario Morales Meseguer
🇪🇸Murcia, Spain
Hospital Universitario Virgen de las Nieves
🇪🇸Granada, Spain
Hospital Clínico Universitario Virgen de la Arrixaca
🇪🇸Murcia, Spain
Hospital Universitario Virgen de la Victoria
🇪🇸Málaga, Spain
Hospital Universitario Fundación Alcorcon
🇪🇸Madrid, Spain
Complejo Asistencial Son Espases
🇪🇸Palma De Mallorca, Spain
Complejo Asistencial Universitario de Salamanca
🇪🇸Salamanca, Spain
Hospital Universitario y Pilitécnico La Fe
🇪🇸Valencia, Spain
Hospital Universitario Virgen del Rocío
🇪🇸Sevilla, Spain