Evaluation of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic ITP

Immune Thrombocytopenia Clinical Trial

Official title:

An Open-label Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic Immune Thrombocytopenia

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05369208
• Other study ID #: AVA-ITP-307
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: June 15, 2022
• Est. completion date: August 31, 2025

Study information

• Verified date: January 2024
• Source: Sobi, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Evaluate the efficacy, safety, and PK of avatrombopag given for 26 weeks in Japanese adults with chronic immune thrombocytopenia (ITP).

Description:

This Phase 3, multicenter, open label study will evaluate the efficacy, safety, and population pharmacokinetics (PK) of avatrombopag in Japanese adults with chronic ITP. At least 19 subjects will be enrolled. The study will consist of 3 phases: Pre-enrollment, Primary Investigation, and Extension Phase until market authorization in Japan.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 19
• Est. completion date: August 31, 2025
• Est. primary completion date: January 17, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Subject has a confirmed diagnosis of chronic immune thrombocytopenia (ITP) (=12 months duration) and has had an insufficient response to a previous ITP treatment, in the opinion of the Investigator.

• Subject has an average of 2 platelet counts <30×10^9/L (no single count can be >35×10^9/L). The 2 samples must be obtained =48 hours and =2 weeks apart.

Exclusion Criteria:

• Subjects with known secondary immune thrombocytopenia (e.g., with known Helicobacter pylori-induced ITP, subjects infected with known human immunodeficiency virus (HIV) or hepatitis C virus (HCV) or subjects with known systemic lupus erythematosus).

• Subjects with known inherited thrombocytopenia (e.g., Myosin Heavy Chain 9 (MYH-9) disorders) or hereditary thrombophilic disorders (e.g., Factor V Leiden, antithrombin III deficiency).

• History of myelodysplastic syndrome (MDS).

• History of arterial or venous thrombosis.

• Subjects with a history of significant cardiovascular disease (e.g., congestive heart failure (CHF) New York Heart Association Grade III/IV, arrhythmia known to increase the risk of thromboembolic events [e.g., atrial fibrillation], angina, coronary artery stent placement, angioplasty, coronary artery bypass grafting).

• Subjects with a history of cirrhosis, portal hypertension, or chronic active hepatitis.

• Subjects with concurrent malignant disease or receiving cytotoxic chemotherapy for a reason other than ITP treatment.

• Use of immunoglobulins (IVIg and anti-D) or corticosteroid rescue therapy within 1 week of Day 1/Baseline.

• Splenectomy or use of rituximab within 12 weeks of Day 1/Baseline.

• Use of romiplostim or eltrombopag within 1 week of Day 1/Baseline.

• Use of chronic corticosteroid treatment or azathioprine within 4 weeks of Day 1/Baseline, unless receiving a stable dose for at least 4 weeks.

• Use of mycophenolate mofetil, cyclosporin A, or danazol within 4 weeks of Day 1/Baseline, unless receiving a stable dose for at least 12 weeks.

• Use of cyclophosphamide or vinca alkaloid regimens within 4 weeks of Baseline Visit.

• Currently receiving moderate or strong dual inhibitors/inducers of CYP2C9 and CYP3A4.

• Serum creatinine =1.5× the upper limit of normal (ULN).

• Serum bilirubin =2×ULN.

• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) =3×ULN.

• Females who are pregnant (positive beta-human chorionic gonadotropin (ß-hCG) test) or breastfeeding.

• Received treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) before Day 1/Baseline.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Avatrombopag Oral Tablet
Avatrombopag 20 mg given once daily (initial dose). Dose adjustments will be determined by the physician and in accordance with the overseas Doptelet prescribing information.

Locations

Country	Name	City	State
Japan	Sobi Site 103	Bunkyo-Ku	Toyko
Japan	Sobi Site 102	Chuo-shi	Yamanashi
Japan	Sobi Site 111	Fujisawa City	Kanagawa
Japan	Sobi Site 115	Fukuyama-shi	Hiroshima
Japan	Sobi Site 114	Gifu City	Gifu
Japan	Sobi Site 104	Hachioji-shi	Tokyo
Japan	Sobi Site 107	Hirakata City	Osaka
Japan	Sobi Site 109	Hiroshima City	Hiroshima
Japan	Sobi Site 118	Iizuka-shi	Fukuoka
Japan	Sobi Site 113	Kanazawa	Ishikawa
Japan	Sobi Site 116	Kitakyushu City	Fukuoka
Japan	Sobi Site 108	Kobe	Hyogo
Japan	Sobi Site 112	Kofu	Yamanashi
Japan	Sobi Site 119	Kumamoto-shi	Kumamoto
Japan	Sobi Site 117	Kurume City	Fukuoka
Japan	Sobi Site 101	Shiwa-gun	Iwata
Japan	Sobi Site 106	Suita	Osaka
Japan	Sobi Site 110	Toon City	Ehime
Japan	Sobi Site 105	Toyohashi	Aichi

Sponsors (1)

Lead Sponsor	Collaborator
Sobi, Inc.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cumulative Number of Weeks of Platelet Response	Cumulative number of weeks in which the platelet count is =50×10^9/L during 26 weeks of treatment in the absence of rescue therapy.	26 weeks of active treatment
Secondary	Response Rate at Day 8	Proportion of subjects with a platelet response =50×10^9/L at Day 8 in the absence of rescue therapy	Day 8