Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia
Study Details
Study Description
Brief Summary
The purpose of this study is to investigate the efficacy, safety of Hetrombopag in children with previously treated chronic immune thrombocytopenia who are between 6 and 17 years of age. This is a 2 part study. In part A, patients will receive Hetrombopag for 8 weeks. In part B, all patients will receive Hetrombopag for 24 weeks.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Detailed Description
This is a two-part, double-blind, randomized, placebo-controlled, and open-label Phase III study to investigate the efficacy, safety of Hetrombopag in pediatric patients with previously treated chronic ITP. In Part A, patients will receive Hetrombopag for 8 weeks. After completing Part A, patients will begin Part B, in which they will be randomized to receive Hetrombopag or placebo in a 12 week double-blind, placebo-controlled treatment period, following an open-label 12 week treatment period.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: PartA, open-label Hetrombopag plus standard of care |
Drug: Hetrombopag
Thrombopoietin receptor agonist
|
| Experimental: PartB, double-blind treatment group Hetrombopag plus standard of care |
Drug: Hetrombopag
Thrombopoietin receptor agonist
|
| Placebo Comparator: Placebo Comparator Placebo plus standard of care Part B, double-blind treatment group |
Drug: Placebo
Placebo with no active pharmaceutical ingredient
|
Outcome Measures
Primary Outcome Measures
- the main parameters in population PK/PD modeling in Part A [from baseline to Week 8]
Peak Plasma Concentration (Cmax)
- the main parameters in population PK/PD modeling in Part A [from baseline to Week 8]
Area under the plasma concentration versus time curve (AUC)
- the proportion of patients with a platelet count ≥50×10^9/L at week 10. [from baseline to Week 10]
efficacy in part B
- Incidence of Treatment-Emergent Adverse Events, Serious Adverse Event. [form baseline to 28 days after the last dose]
safety in Part B
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Confirmed diagnosis of ITP ≥12 months; Platelets <30×109/L.
-
Subjects who are refractory or have relapsed after at least one prior ITP therapy.
-
Previous therapy for ITP including rescue must have been completed at least 21 days prior to randomization.
-
Subjects treated with maintenance therapy(such as Corticosteroids, Azathioprine, and Azathioprine) must be receiving a dose that has been stable for at least 14 days,30days, and 60 days respectively.
-
Written informed consent must be obtained from the patient's guardian and accompanying informed assent from the patient (for children over 8 years old).
Exclusion Criteria:
-
No evidence of other causes of thrombocytopenia.
-
Patients with any prior history of arterial or venous thrombosis, or diagnosis as thrombophilia.
-
Subjects who have previously received eltrombopag or any other thrombopoietin receptor agonist within 30 days.
-
ALT, AST, or ALP> 1.5 x upper limit of normal (ULN), DBLI, or Scr > 1.2 x upper limit of normal (ULN).
-
PT result exceeds normal by more than ±3s, APTT result exceeds normal by more than ±10s
-
Suffering from serious, progressive, uncontrolled kidney, liver, gastrointestinal, endocrine, lung, heart, nervous system, brain, or mental illness.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Beijing children's hospital .Capital medical university | Beijing | Beijing | China | 100032 |
Sponsors and Collaborators
- Jiangsu HengRui Medicine Co., Ltd.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- HR-TPO-ITP-III-PED