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

NCT05369208 | Completed Phase 3 Results FDA Drug

• 1 other identifier: AVA-ITP-307
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 19

Brief Summary

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

Conditions

Immune Thrombocytopenia

Keywords

ITP

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Response Rate at Day 8

  Day 8

Study Arms (1)

Avatrombopag

OTHER

Avatrombopag 20 mg oral tablet

Drug: Avatrombopag Oral Tablet

Interventions

Avatrombopag Oral Tablet DRUG

Avatrombopag 20 mg given once daily (initial dose). Dosage adjustments were determined by the physician to maintain a platelet count between 50 x 10\^9 to 200 x 10\^9 as defined in the protocol and in accordance with overseas labeling.

Also known as: Doptelet

Avatrombopag

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Sobi, Inc.: lead

Study Sites (19)

Sobi Site 105

Toyohashi, Aichi-ken, 441-8570, Japan

Location

Sobi Site 110

Tōon, Ehime, 791-0295, Japan

Location

Sobi Site 118

Iizuka-shi, Fukuoka, 820-8505, Japan

Location

Sobi Site 116

Kitakyushu, Fukuoka, 802-8555, Japan

Location

Sobi Site 117

Kurume, Fukuoka, 830-8543, Japan

Location

Sobi Site 114

Gifu, Gifu, 500-8513, Japan

Location

Sobi Site 115

Fukuyama-shi, Hiroshima, 720-2121, Japan

Location

Sobi Site 109

Hiroshima, Hiroshima, 730-0052, Japan

Location

Sobi Site 108

Kobe, Hyōgo, 650-0047, Japan

Location

Sobi Site 113

Kanazawa, Ishikawa-ken, 920-8650, Japan

Location

Sobi Site 101

Shiwa-gun, Iwata, 028-3695, Japan

Location

Sobi Site 111

Fujisawa, Kanagawa, 251-8550, Japan

Location

Sobi Site 119

Kumamoto, Kumamoto, 862-8655, Japan

Location

Sobi Site 107

Hirakata, Osaka, 573-1191, Japan

Location

Sobi Site 106

Suita, Osaka, 565-0871, Japan

Location

Sobi Site 104

Hachiōji-shi, Tokyo, 192-0032, Japan

Location

Sobi Site 103

Bunkyō City, Toyko, 113-8603, Japan

Location

Sobi Site 102

Chuo-shi, Yamanashi, 409-3898, Japan

Location

Sobi Site 112

Kofu, Yamanashi, 400-8506, Japan

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Medicine Development Leader
• Organization: Sobi Inc.

medinfo.us@sobi.com

1-781-786-7370

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 21, 2022
• First Posted: May 11, 2022
• Study Start: June 15, 2022
• Primary Completion: January 17, 2024
• Study Completion: October 29, 2025
• Last Updated: April 21, 2026
• Results First Posted: February 11, 2025

Record last verified: 2026-03

Locations

JP (19)