Efficacy of Eltrombopag to Improve Thrombocytopenia of MYH9-related Disease

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-01-31

Study Completion Date

2010-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The term MYH9-related disease (MYH9RD) includes four genetic disorders: May-Hegglin anomaly, Sebastian syndrome, Fechtner syndrome, and Epstein syndrome. All these disorders derive from mutation of a unique gene, named MYH9, and they have been recognized as different clinical presentations of a single illness that was named MYH9RD. All patients affected by MYH9RD present since birth with thrombocytopenia, which can result in a variable degree of bleeding diathesis; some of them subsequently develop additional clinical manifestations, such as renal damage, sensorineural hearing loss, and/or presenile cataracts. Eltrombopag is an oral thrombopoietin receptor agonist that stimulates proliferation and differentiation of megakaryocytes, the bone marrow cells that produce blood platelets. This drug is effective in increasing platelet count in healthy volunteers, as well as in patients affected by some acquired thrombocytopenias, such as idiopathic thrombocytopenic purpura and HCV related thrombocytopenia. The purpose of this study is to determine if eltrombopag, administered orally at the dose of 50 or 75 mg/daily for up to 6 weeks, is effective in increasing platelet count of patients affected by MYH9RD. Further aims of this study are to test if eltrombopag is effective in reducing bleeding tendency of MYH9RD patients; to evaluate safety and tolerability of eltrombopag in patients with MYH9RD; to evaluate in vitro function of platelets produced during therapy in patients responding to this drug.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Eltrombopag for Inherited Thrombocytopenias

NCT02422394

Inherited Platelet Disorder

COMPLETED PHASE2

Study of Eltrombopag in Platelet Refractory Thrombocytopenia

NCT01194167

Thrombocytopenia

WITHDRAWN PHASE2

Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes

NCT02912208

Myelodysplastic Syndromes Thrombocytopenia

ACTIVE_NOT_RECRUITING PHASE2

A Study of Eltrombopag in Patients With CMML and Thrombocytopenia

NCT02323178

CMML Thrombocytopenia

COMPLETED PHASE1/PHASE2

A Study to Assess the Ability of Eltrombopag to Induce Sustained Response Off Treatment in Subjects With ITP

NCT03524612

Immune Thrombocytopenic Purpura (ITP)

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Blood Platelet Disorders

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

eltrombopag

Group Type EXPERIMENTAL

eltrombopag

Intervention Type DRUG

Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 will continue eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 will receive eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 will stop therapy.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

eltrombopag

Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 will continue eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 will receive eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 will stop therapy.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Revolade Promacta

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Age 16 years or more
* Confirmed diagnosis of MYH9-related disease
* Average platelet count for the previous year less than 50x10e9/L
* Written informed consent

Exclusion Criteria

* Diseases known to involve the risk of thromboembolic events (e.g. atrial fibrillation)
* History of thrombosis within 1 year
* Use of drugs that affect platelet function (including but not limited to, aspirin, clopidogrel or NSAIDS) or anti-coagulants
* Females who are pregnant or nursing (a negative pregnancy test in required before enrollment of fertile women)
* Formal refusal of any recommendation of a safe contraception
* Alcohol or drug addiction
* Altered renal function as defined by creatinine of 20 mg/L or more
* Any other disease or condition that by the advise of the responsible physician would make the treatment dangerous for the patient or would make the patient ineligible for the study, including physical, psychiatric, social and behavioral problems. HCV positivity and liver diseases will not be considered an exclusion criterion since a phase II study showed that eltrombopag was effective and safe in this patient population.

Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No