Evaluation of safety and efficacy of eltrombopag in increasing platelet count and reducing bleeding tendency of patients with different forms of inherited thrombocytopenia.

Phase 1

• Conditions: Inherited thrombocytopenias; MedDRA version: 17.0Level: HLTClassification code 10043555Term: ThrombocytopeniasSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2014-000608-90-IT
• Lead Sponsor: Fondazione IRCCS Policlinico San Matteo

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-10-03
• Last Update Posted: 14 March 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

Phase 1
Patients with the following forms of inherited thrombocytopenias will be considered for enrolment:
- MYH9-related disorders (OMIM 155100, 605249, 153640, 153650)
- Bernard-Soulier Syndrome (OMIM 231200) deriving from monoallelic mutations (at variance with biallelic classical BSS, monoallelic form does not present defective platelet function)
- Wiskott-Aldrich syndrome (OMIM 301000).
- X-linked thrombocytopenia (OMIM 313900).
- X-linked thrombocytopenia with thalassemia (OMIM 314050).
- Dyserythropoietic anemia with thrombocytopenia (OMIM 300367).
- ITGA2B/ITGB3-related thrombocytopenia (OMIM not available).
- ANKRD26-related thrombocytopenia (OMIM 188000).
- TUBB1-related thrombocytopenia (OMIM not available)
- ACTN1-related thrombocytopenia (OMIM not available)
- GFI1B-related thrombocytopenia (OMIM not available)
Patients will have to fulfill all the following criteria:
- Age = 16 years and = 70 years
- Average platelet count during the previous year less than 80 x109/L
- Written informed consent

Phase 2
Patients with clinically relevant chronic or recurrent bleedings at baseline (grade 2-4 of the WHO bleeding scale) who: (i) completed the phase 1 without severe side effects and (ii) obtained reduction or remission of bleeding by Eltrombopag administration.
Are the trial subjects under 18? yes
Number of subjects for this age range: 3
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 16
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

Phases 1 and 2
-Hypersensitivity to Eltrombopag or one of the excipients.
- History of thromboembolic events.
- Treatment with anti-platelet drugs or other drugs affecting platelet function and/or with anticoagulants.
- Concurrent diseases or conditions that significantly increase the risk of thromboembolic events, including: malignancies, congestive heart failure (NYHA Grade III/IV), atrial fibrillation or other arrhythmias that predispose to thromboembolic events, known hereditary factors predisposing to venous thromboembolism (factor V mutations, factor II mutations, deficiency of antithrombin III, or protein C, or protein S), antiphospholipid syndrome, hormone replacement therapy and systemic contraception containing estrogen, severe obesity, severe hypertension not controlled by treatment, heavy smoking, diabetes mellitus not controlled by therapy or complicated, severe dislipidemia, haemoglobinopathies increasing the risk of thrombotic events, such as sickle cell anemia or thalassemia major.
- Moderate to severe liver failure (Child-Pugh score = 5).
- Altered renal function as defined by creatinine = 2 mg/dL
- Previous or concurrent clonal disorders of the myeloid series (acute myeloid leukemias and myelodysplastic syndromes).
- Females who are pregnant or nursing (a negative pregnancy test is required before enrolment of fertile women).
- Formal refusal of any recommendations for a safe contraception.
- Alcohol or drug addiction.
- Any other disease or condition that by the advice of the responsible physician would make the treatment dangerous for the patient or would make the patient ineligible for this study, including physical, psychiatric, social and behavioral problems.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified