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
- Are there results?
- None posted in the registry
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
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Hetrombopag in Children and Adolescents With Chronic Primary Immune Thrombocytopenia：a Randomized, Multicenter, Placebo-controlled Trial
- ClinicalTrials.gov ID
- NCT04737850
- Acronym / sponsor ID
- HR-TPO-ITP-III-PED
- Conditions
- Immune Thrombocytopenia
- Keywords
- ITP
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Jiangsu HengRui Medicine Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 29 Mar 2021 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 17 Jun 2025 (actual)
- First posted
- 4 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Parallel assignment
| Arm | Type | Receives |
|---|---|---|
| PartA, open-label | Experimental | Drug: HetrombopagHetrombopag plus standard of care |
| PartB, double-blind treatment group | Experimental | Drug: HetrombopagHetrombopag plus standard of care |
| Placebo Comparator | Placebo comparator | Drug: PlaceboPlacebo plus standard of care Part B, double-blind treatment group |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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).
Key exclusion criteria (9 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HetrombopagThrombopoietin receptor agonist | Drug | PartA, open-label, PartB, double-blind treatment group |
| PlaceboPlacebo with no active pharmaceutical ingredient | Drug | Placebo Comparator |
Primary outcome
the main parameters in population PK/PD modeling in Part A
Time frame: from baseline to Week 2
Peak Plasma Concentration (Cmax)
the proportion of patients with a platelet count ≥50×10^9/L at week 10.
Time frame: from baseline to Week 10
efficacy in part B
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2025.
- Derived from this study: PubMed 41952212Wu 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Hetrombopag in people with Immune Thrombocytopenia, run by Jiangsu HengRui Medicine Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
the main parameters in population PK/PD modeling in Part A (time frame: from baseline to Week 2). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04737850, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jul 2025.