Clinical trial NCT05369208: Evaluation of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic ITP
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Immune Thrombocytopenia
Keywords: ITP
Lead sponsor: Sobi, Inc.
Sponsor class: INDUSTRY
First posted: 2022-05-11
Enrollment: 19
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility criteria: 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 (β-hCG) test) or breastfeeding.
* Received treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) before Day 1/Baseline.

Primary outcomes:
measure: Cumulative Number of Weeks of Platelet Response
timeFrame: 26 weeks of active treatment
Secondary outcomes:
measure: Response Rate at Day 8
timeFrame: Day 8
Other outcomes:
Locations:
city: Toyohashi
country: Japan
facility: Sobi Site 105
state: Aichi
city: Toon City
country: Japan
facility: Sobi Site 110
state: Ehime
city: Iizuka-shi
country: Japan
facility: Sobi Site 118
state: Fukuoka
city: Kitakyushu City
country: Japan
facility: Sobi Site 116
state: Fukuoka
city: Kurume City
country: Japan
facility: Sobi Site 117
state: Fukuoka
city: Gifu City
country: Japan
facility: Sobi Site 114
state: Gifu
city: Fukuyama-shi
country: Japan
facility: Sobi Site 115
state: Hiroshima
city: Hiroshima City
country: Japan
facility: Sobi Site 109
state: Hiroshima
city: Kobe
country: Japan
facility: Sobi Site 108
state: Hyogo
city: Kanazawa
country: Japan
facility: Sobi Site 113
state: Ishikawa
city: Shiwa-gun
country: Japan
facility: Sobi Site 101
state: Iwata
city: Fujisawa City
country: Japan
facility: Sobi Site 111
state: Kanagawa
city: Kumamoto-shi
country: Japan
facility: Sobi Site 119
state: Kumamoto
city: Hirakata City
country: Japan
facility: Sobi Site 107
state: Osaka
city: Suita
country: Japan
facility: Sobi Site 106
state: Osaka
city: Hachiōji-shi
country: Japan
facility: Sobi Site 104
state: Tokyo
city: Bunkyō-Ku
country: Japan
facility: Sobi Site 103
state: Toyko
city: Chuo-shi
country: Japan
facility: Sobi Site 102
state: Yamanashi
city: Kōfu
country: Japan
facility: Sobi Site 112
state: Yamanashi
Study officials:
References: