Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome

NCT04371939 | Unknown Phase 2

• 1 other identifier: NCPHOI-2020-03
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective, open-label, randomized, two-arm clinical trial conducted to evaluate the safety and efficacy of romiplostim in comparison with eltrombopag in the treatment of thrombocytopenia in patients with Wiskott-Aldrich syndrome

Conditions

Wiskott-Aldrich Syndrome

Keywords

eltrombopag; romiplostim; thrombocytopenia; platelets

Outcome Measures

Primary Outcomes (1)

• Percentage of patients with overall platelet response (complete response + partial response) for romiplostim and eltrombopag group

  A complete response will be defined as a platelet count \>100 x 109/L, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L.

  1 month (30 day +/- 7 days)

Secondary Outcomes (3)

• Number of participants with bleeding events and severity of bleeding in romiplostim and eltrombopag group

  until discontinuation, from at least one month to one year

• Number of participants with drug related adverse events in each treatment group

  until discontinuation, from at least one month to one year

• Percentage of patients with overall platelet response (complete response + partial response) after switching from one TPO-RA to another one.

  until discontinuation, from at least one month to one year

Study Arms (2)

I (Romiplostim)

EXPERIMENTAL

Participants will receive romiplostim at an initial dose of 9 µg/kg subcutaneously per week for at least 1 month depending on their response to study drug. Patients failing to achieve a complete platelet response cross over to arm II.

Drug: Romiplostim

II (Eltrombopag)

EXPERIMENTAL

Participants will receive eltrombopag at a dose of 2-3mg/kg daily (ages 0 to 5 years) and 75 mg/daily (\>6 years) for at least 1 month depending on their response to study drug. Patients failing to achieve a complete platelet response switch to arm I.

Drug: Eltrombopag

Interventions

Romiplostim DRUG

Participants will receive romiplostim at an initial dose of 9 µg/kg subcutaneously per week for at least 1 month depending on their response to study drug. Patients failing to achieve a complete platelet response cross over to arm II.

Also known as: Nplate

I (Romiplostim)

Eltrombopag DRUG

Participants will receive eltrombopag at a dose of 2-3mg/kg daily (ages 0 to 5 years) and 75 mg/daily (\>6 years) for at least 1 month depending on their response to study drug. Patients failing to achieve a complete platelet response switch to arm I.

II (Eltrombopag)

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Genetically verified Wiskott-Aldrich syndrome
• Thrombocytopenia (platelet count \< 70 x 109/L)
• Age: under 18 years
• Subject/legal representative has signed written informed consent.

You may not qualify if:

• Any prior history of arterial or venous thrombosis within the past year.
• Arm II (eltrombopag):
• abnormal hepatic function -elevated AST/ALT \> 1.5 times upper limit of normal within 4 weeks prior to enrollment
• Active colitis

Sponsors & Collaborators

• Federal Research Institute of Pediatric Hematology, Oncology and Immunology: lead

Study Sites (1)

Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology

Moscow, 117997, Russia

RECRUITING

MeSH Terms

Conditions

Wiskott-Aldrich Syndrome; Thrombocytopenia

Interventions

romiplostim; eltrombopag

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphopenia; Leukopenia; Cytopenia; Hemorrhagic Disorders; Leukocyte Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Blood Platelet Disorders

Central Study Contacts

Anna Shcherbina, MD, PhD

CONTACT

+7(495)2876570; shcher26@hotmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 29, 2020
• First Posted: May 1, 2020
• Study Start: November 5, 2019
• Primary Completion: December 1, 2022
• Study Completion: July 30, 2023
• Last Updated: May 6, 2020

Record last verified: 2020-05

Locations

RU (1)