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

Sponsor
Fundación Pública Andaluza para la gestión de la Investigación en Sevilla (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT05325593
Collaborator
(none)
126
12
2
28.1
10.5
0.4

Study Details

Study Description

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

Condition or Disease Intervention/Treatment Phase
Phase 3

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 WHO 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.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
126 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
Parallel Assignment Randomized 1:1Parallel Assignment Randomized 1:1
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Multicentre, Randomized, Open-label Study of Romiplostim Plus Dexamethasone vs Dexamethasone in Patients With Newly Diagnosed Primary Immune Thrombocytopenia
Anticipated Study Start Date :
Jul 1, 2022
Anticipated Primary Completion Date :
Nov 1, 2023
Anticipated Study Completion Date :
Nov 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: 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

Drug: 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.
Other Names:
  • ROM + DEX
  • Active Comparator: Dexamethasone (DEX)

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

    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.
    Other Names:
  • DEX
  • Outcome Measures

    Primary Outcome Measures

    1. Proportion of patients achieving 6mSROT-50 (Sustained Response Off any ITP Treatment) [180 days after treatment withdrawal]

      6mSROT-50: 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 WHO grade 2 or more bleeding

    Secondary Outcome Measures

    1. Proportion of patients achieving 6mSROT-30, 12mSROT-30, 6mSROT-50 and 12mSROT-50 in the absence of any ITP treatment including any rescue treatment. [Day 180, Day 365]

      6mSROT-30: 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 WHO grade 2 or more bleeding. 12mSROT-30: 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 WHO grade 2 or more bleeding. 12mSROT-50: 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 WHO grade 2 or more bleeding.

    2. Proportion of patients with early response (ER) and initial response (IR) [ER (Day 7), IR (Day 30)]

      ER: proportion of patients with platelet count higher or equal than 30x109/L and at least double than baseline IR: proportion of patients with platelet count higher or equal than 30x109/L

    3. Proportion of patients with complete response (CR), response (R), global response (GR) and targeted range (TR). [Day 180, Day 365, Day 545]

      CR: platelet count ≥100x109/L and absence of bleeding symptoms. R: platelet count between 100x109/L and 30x109/L and at least doubled from baseline and absence of bleeding symptoms. TR: platelet count between ≥30x109/L and ≤400x109/L.

    4. Time to loss of response (LoR) in patients who achieved response in both arms. [day 1, week 1, week 2, week 3, week 4, week 5, week 6, week 7, week 8, day 180, day 365, day 545]

      LoR: 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

    5. Proportion of patients requiring any rescue treatment along the study period. [day 1, week 1, week 2, week 3, week 4, week 5, week 6, week 7, week 8, day 180, day 365, day 545]

      Proportion of patients who need rescue treatments and proportion and time to treatment failures.

    6. Proportion of patients with adverse events (AEs), including serious adverse events (SAEs) and laboratory safety parameters. [day 1, week 1, week 2, week 3, week 4, week 5, week 6, week 7, week 8, day 180, day 365, day 545]

      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

    7. Changes in patients' quality of life- ITP-BAT [Day +180, +365 and +545]

      For the assessment of the quality of life during the study will be used: -ITP (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.

    8. Changes in patients' quality of life- EQ-5D-5L [Day +180, +365 and +545]

      For the assessment of the quality of life during the study will be used: -EQ-5D-5L (European Quality of Life-5 Dimensions- 5 levels): descriptive system: with five dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression). Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions

    9. Changes in patients' quality of life- EQ VAS [Day +180, +365 and +545]

      For the assessment of the quality of life during the study will be used: -EQ visual analogue scale (EQ VAS): is a visual analogue scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).

    10. Differences between study arms in mean of healthcare resources use (HRU) [day 1, week 1, week 2, week 3, week 4, week 5, week 6, week 7, week 8, day 180, day 365, day 545]

      The data collected may be used to conduct exploratory economic analyses and may include: Hospitalization (yes or no) Duration of hospitalization (days) Duration of medical care visits (days) Number of Emergency room visits Number of diagnostic and therapeutic tests and procedures Number of medical care visits

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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. WHO (World Health Organization) performance status >2.

    2. Previous therapy with rituximab, corticosteroids, immunomodulating agents, hematopoietic analogs and fostamatinib for any other reason despite ITP.

    3. Previous use of romiplostim, PEG-recombinant human (rHu) megakaryocyte growth and development factor, eltrombopag, recombinant human anti-thrombopoietin (rHuTPO), or any platelet-producing agent

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Hospital General de Burgos Burgos Spain
    2 Hospital Universitario A Coruña Coruña Spain
    3 Hospital Clínico de Granada Granada Spain
    4 Hospital Gregorio Marañon Madrid Spain
    5 Hospital La Paz Madrid Spain
    6 Hospital Universitario Morales Meseguer Murcia Spain
    7 Hospital Virgen de la Arrixaca Murcia Spain
    8 Hospital Universitario Virgen de la Victoria Málaga Spain
    9 Hospital Son Espases Palma De Mallorca Spain
    10 Hospital Universitario de Salamanca Salamanca Spain
    11 Hospital Universitario Virgen del Rocío Sevilla Spain
    12 Hospital La Fe Valencia Spain

    Sponsors and Collaborators

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

    Investigators

    • Principal Investigator: Charlotte Bradbury, Centre for Trials Research College of Biomedical & Life Sciences Cardiff University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Fundación Pública Andaluza para la gestión de la Investigación en Sevilla
    ClinicalTrials.gov Identifier:
    NCT05325593
    Other Study ID Numbers:
    • RODEX
    • 2021-006970-22
    First Posted:
    Apr 13, 2022
    Last Update Posted:
    Apr 13, 2022
    Last Verified:
    Apr 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Fundación Pública Andaluza para la gestión de la Investigación en Sevilla
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Apr 13, 2022