Clinical trial NCT02403440: A Study of Hetrombopag Olamine in Chronic Idiopathic Thrombocytopenic Purpura (ITP) Patients
Official title: A Safety, Pharmacokinetics and Pharmacodynamics Study of Hetrombopag Olamine in Chronic Idiopathic Thrombocytopenic Purpura
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Purpura, Thrombocytopenic, Idiopathic
Lead sponsor: Jiangsu HengRui Medicine Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2015-03-31
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to obtain information on efficacy, safety and Pharmacokinetics (PK)/Pharmacodynamics (PD) of Hetrombopag over 14 days in Chinese patients with chronic ITP.


Eligibility criteria: Inclusion Criteria:

1. Chronical ITP patients.
2. The subjects were diagnosed as ITP with bone marrow aspiration within 3 months before enrollment or in the screening period. And secondary immune thrombocytopenia (e.g., myelodysplastic syndrome, systemic lupus erythematosus, aplastic anemia) was excluded.
3. Patients had a mean platelet count of less than 30,000/µL in the screening period.
4. Patients receiving chronic maintenance steroid therapy must have received a stable dose for at least 1 month.
5. Patients receiving danazol, mycophenolate mofetil or cyclosporine A must have received a stable dose for at least 12 weeks.
6. Normal PT/INR and APTT.

Exclusion Criteria:

1. Any prior history of arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism), AND ≥ two of the following risk factors: hormone replacement therapy, systemic contraception (containing estrogen), smoking, diabetes, hypercholesterolemia, medication for hypertension, cancer, hereditary thrombophilic disorders (e.g., Factor V Leiden, ATIII deficiency, etc), or any other family history of arterial or venous thrombosis.
2. Pre-existing cardiovascular disease (congestive heart failure, New York Heart Association \[NYHA\] Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. atrial fibrillation).
3. Malignant disease
4. Cancer treatment with cytotoxic chemotherapy and/or radiotherapy.
5. Patients with one of the following conditions should be excluded:

   * Treatment with immunoglobulins within 1 week preceding the first dose of study medication.
   * Treatment with splenectomy or rituximab within 12 weeks preceding the first dose of study medication.
   * Treatment with eltrombopag or Nplate within 4 weeks preceding the first dose of study medication.
   * Treatment with cyclophosphamide or vinca alkaloids within 4 weeks preceding the first dose of study medication.
6. ALT\>2×ULN，AST\>2×ULN，Total Bilirubin\>1.5×ULN，serum creatinine \>1.2×ULN，Total albumin \<0.9×LLN

Primary outcomes:
measure: The number of volunteers with adverse events as a measure of safety and tolerability
timeFrame: up to Day 28
Secondary outcomes:
measure: Plasma pharmacokinetic (PK) parameters of Hetrombopag after multiple dose from day 1 to day 14, composite including AUC, Cmax, Tmax, and t1/2
timeFrame: day 1 and day 14
measure: The proportion of patients with platelet counts ≥50,000/µL after treatment
timeFrame: up to Day 28
Other outcomes:
Locations:
city: Chengdu
country: China
facility: West Hospital, Sichuan University
Study officials:
References:
citation: Wang Z, Chen L, Zhang F, Lu H, Chen X, Wen A, Luo J, Hu Y, Wang Y, Niu T, Zheng L. First-in-patient study of hetrombopag in patients with chronic idiopathic thrombocytopenic purpura. J Thromb Haemost. 2020 Nov;18(11):3053-3060. doi: 10.1111/jth.15078. Epub 2020 Oct 1.