Immune ThrombocytopeniaPhase 3NCT04737850
Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia
A Phase 3 study testing Hetrombopag in people with Immune Thrombocytopenia, run by Jiangsu HengRui Medicine Co., Ltd. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Hetrombopag
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- 29 Mar 2021to 17 Jun 2025
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥ 6 years old and ≤ 17 years old,both sexes.
- Part A:Confirmed diagnosis of ITP ≥6months; Part B: Confirmed diagnosis of ITP ≥12months;Platelets <30×10^9/L twice in a row,and platelets <30×10^9/L before taking the medicine.
- Subjects who are refractory or have relapsed after at least one prior ITP therapy.
- Birth control during and 28 days after the trial.
- Written informed consent must be obtained from the patient's guardian and accompanying informed assent from the patient (for children over 8 years old).
Who cannot join
- No evidence of other causes of thrombocytopenia.
- Diagnosis as Evans or Wiskott-Aldrich comprehensive.
- Patients with any prior history of arterial or venous thrombosis, or diagnosis as thrombophilia.
- Suffering from serious, progressive, uncontrolled kidney, liver, gastrointestinal, endocrine, lung, heart, nervous system, brain, or mental illness.
- ALT, AST, or ALP> 1.5 x upper limit of normal (ULN), DBLI, or Scr > 1.2 x upper limit of normal (ULN).
- Active HIV or HCV-Ab positive,HBsAg positive.
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- PT result exceeds normal by more than ±3s, APTT result exceeds normal by more than ±10s
- Participated in clinical trials of other drugs (received experimental drugs) within 3 months prior to medication.
- The inestigators determined that other conditions were inappropriate for participation in this clinical trial.
Trial locations and contacts
1 site in 1 country.
China1 site
Beijing children's hospital .Capital medical university
Beijing, Beijing Municipality, 100032
About this study
Official title: Safety and Efficacy of Hetrombopag in Children and Adolescents With Chronic Primary Immune Thrombocytopenia：a Randomized, Multicenter, Placebo-controlled Trial
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.
Read the 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.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Hetrombopag, Placebo
- Keywords
- ITP
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HetrombopagDrug
Thrombopoietin receptor agonist
PlaceboDrug
Placebo with no active pharmaceutical ingredient
| Study group | Role | What they receive |
|---|---|---|
| PartA, open-label | Experimental | Hetrombopag plus standard of care Receives: Drug: Hetrombopag |
| PartB, double-blind treatment group | Experimental | Hetrombopag plus standard of care Receives: Drug: Hetrombopag |
| Placebo Comparator | Placebo comparator | Placebo plus standard of care Part B, double-blind treatment group Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
the main parameters in population PK/PD modeling in Part A
Peak Plasma Concentration (Cmax)
Measured: from baseline to Week 2
the proportion of patients with a platelet count ≥50×10^9/L at week 10.
efficacy in part B
Measured: from baseline to Week 10
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2021
- Study start29 Mar 2021
- Primary completion31 Dec 2024
- Study completion17 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Jiangsu HengRui Medicine Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Parallel assignment
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jiangsu HengRui Medicine Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04737850
- Sponsor's protocol ID
- HR-TPO-ITP-III-PED
- Status last verified
- Jul 2025
- First submitted
- 17 Jan 2021
- Last updated
- 18 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Wu R, Gao J, Liu Y, Yu J, Hu S, Liang H, Jiang H, Zhai X, Zhou M, Hao W, Mei H, Li F, Chen Y, Liu W, Yan M, Li R, Xiong J, Zhu Z, Cheng X, Wang T. Efficacy and safety of hetrombopag, a novel thrombopoietin receptor agonist, in children and adolescents with immune thrombocytopenia: results from a randomized, multicenter, placebo-controlled phase 3 trial. J Hematol Oncol. 2026 Apr 8;19(1):24. doi: 10.1186/s13045-026-01790-x.
PubMed 41952212