Romiplostim, Rituximab and Dexamethasone as Frontline Treatment for Immune Thrombocytopenia

Sponsor

David Gomez Almaguer (Other)

Overall Status

Recruiting

CT.gov ID

NCT04588194

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to determine the response rate and response duration with the combination of low-dose rituximab, romiplostim and high-dose dexamethasone.

Condition or Disease	Intervention/Treatment	Phase
Immune Thrombocytopenia Thrombotic Thrombocytopenic Purpura	Drug: Romiplostim Drug: Rituximab Drug: Dexamethasone	Phase 2

Detailed Description

Immune thrombocytopenia is an autoimmune disorder characterized by formation of autoantibodies against platelet antigens leading platelet destruction.

Corticosteroids increase the platelet count in about 80 percent of patients. However, many patients have a relapse when the dose of corticosteroid is reduced. Debilitating side effects are common in patients who require long-term corticosteroid therapy to maintain the platelet count. Romiplostim, it is a small molecule agonist of the c-mpl (TpoR) receptor, which is the physiological target of the hormone thrombopoietin, has been shown to be effectively raise the platelet count in adult patients (aged 18 years and over) who have had their spleen removed or where splenectomy is not an option and have received prior treatment with corticosteroids or immunoglobulins, and these medicines did not work (refractory ITP). There are a few case reports where romiplostim an option as first line treatment for IT.

The purpose of this study is to determine the response rate and response duration with the combination of rituximab (100 mg weekly four weeks), romiplostim (2mcg/Kg four weekly) and high-dose dexamethasone (40mg PO days 1-4) in untreated adult patients with <30*109/L platelet count diagnosed with immune thrombocytopenia.

A complete response is defined as an increase in platelet counts to >150×109/L on two consecutive occasions. A clinical response is defined as an increase in the platelet count between >30×109/L on two consecutive measures and no bleeding. Duration of response is considered from the day of the initial administration to the first time of relapse (platelet count <30×109/L) or to time of analysis Patients will be evaluated each week during 4 weeks and then every month for at least 6 months.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

12 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Romiplostim in Combination With Low-dose Rituximab and High-dose Dexamethasone as Frontline Treatment for Immune Thrombocytopenia

Anticipated Study Start Date :

Nov 1, 2020

Anticipated Primary Completion Date :

Nov 1, 2021

Anticipated Study Completion Date :

Nov 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Romiplostim, Rituximab, Dexamethasone Each patient will receive Rituximab 100 mg weekly days 1, 7, 14, 21, Romiplostim 2mcg/Kg subcutaneously weekly days 1, 7, 14, 21 and Dexamethasone 40 mg IV/PO days 1-4.	Drug: Romiplostim Romiplostim 2mcg/Kg subcutaneously weekly days 1, 7, 14, 21 Drug: Rituximab Rituximab 100 mg weekly days 1, 7, 14, 21 Drug: Dexamethasone 40 mg IV/PO days 1-4

Arm

Intervention/Treatment

Experimental: Romiplostim, Rituximab, Dexamethasone

Each patient will receive Rituximab 100 mg weekly days 1, 7, 14, 21, Romiplostim 2mcg/Kg subcutaneously weekly days 1, 7, 14, 21 and Dexamethasone 40 mg IV/PO days 1-4.

Drug: Romiplostim

Romiplostim 2mcg/Kg subcutaneously weekly days 1, 7, 14, 21

Drug: Rituximab

Rituximab 100 mg weekly days 1, 7, 14, 21

Drug: Dexamethasone

40 mg IV/PO days 1-4

Outcome Measures

Primary Outcome Measures

Clinical Response [28 days]
Platelet counts to >30×109/L on two consecutive occasions

Secondary Outcome Measures

Complete Response [28 days]
Platelet counts to >100×109/L on two consecutive occasions

Eligibility Criteria

Criteria

Ages Eligible for Study:

16 Years to 90 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Clinically confirmed immune thrombocytopenia (IT) Platelet count less than 30,000/mm3 on two occasions.
Subject ≥ 16 years
Subject has signed and dated written informed consent.

Exclusion Criteria:

Previous treatment (only corticosteroids at dose or prednisone equivalent of 300 mg)
Performance status above or equal to 2.
Pregnancy and lactation
Previous splenectomy
Connective tissue disease
Autoimmune hemolytic anemia
Relapse
Active infection, sepsis or fever
Positive for hepatitis B virus or hepatitis C virus or human immunodeficiency virus.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Servicio de Hematología, Hospital Universitario "Dr. José Eleuterio González", Universidad Autónoma de Nuevo León	Monterrey	Nuevo León	Mexico	64460