Thalassemia in ChildrenNCT07003269
Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT
An observational study of Thalassemia in Children, run by Haikou Affiliated Hospital of Central South University Xiangya School of Medicine at 1 site in China.
Trial summary
The objective of this observational study is to investigate the long-term effects of Hetrombopag in promoting platelet engraftment during haploidentical hematopoietic stem cell transplantation (HSCT) in children with thalassemia, with a specific focus on a 28-day time window post-transplantation. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Thalassemia in Children
- What is being tested?
- Hetrombopag
- Type of treatment
- Pill or oral medicine, Injection or infusion
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2024to 30 Apr 2026
- Phase
- Observational
- 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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: The Department of Hematology of Haikou People's Hospital accepts children with severe thalassemia aged 2 - 17 years old for haploidentical hematopoietic stem cell transplantation.
Who may be able to join
- Diagnosed with severe thalassemia via thalassemia gene testing, transfusion history, and complete blood count (CBC).
- Pediatric patients aged 2-17 years.
- Consented to haploidentical transplantation and evaluated by the transplant team as having no transplant contraindications.
Who cannot join
- Presence of a fully matched donor with refusal of haploidentical transplantation.
- Donor or recipient with transaminase levels >2× the upper limit of normal (ULN).
- Positive hepatitis B DNA test result.
- Active infection at the time of enrollment.
- Donor-specific antibodies (DSA) >5,000 and unable to decrease below 3,000 after antibody therapy.
- Presence of transplant contraindications as assessed by the transplant team.
Trial locations and contacts
1 site in 1 country.
China1 site
Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
Haikou, Hainan, 570208
About this study
Official title: An Observational Study on the Promotion of Platelet Recovery by Hetrombopag During Haploidentical Hematopoietic Stem Cell Transplantation
The objective of this observational study is to investigate the long-term effects of Hetrombopag in promoting platelet engraftment during haploidentical hematopoietic stem cell transplantation (HSCT) in children with thalassemia, with a specific focus on a 28-day time window post-transplantation. The core question to be addressed is: Is Hetrombopag safe and effective for platelet engraftment in children with thalassemia undergoing haploidentical HSCT within a 28-day post-transplant period? Subjects who received Hetrombopag as part of routine care for haploidentical HSCT in children with thalassemia will be required to complete a 28-day online survey on platelet engraftment outcomes.
Read the detailed description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains a critical and often the sole curative modality for diverse hematological malignancies and disorders. Post-transplant thrombocytopenia (platelet count <20×10⁹/L) significantly compromises long-term patient survival, with an incidence of 5-20% in allo-HSCT recipients, thereby escalating treatment morbidity and costs. Current research on platelet engraftment promotion in pediatric thalassemia patients undergoing allo-HSCT is limited. Repeated platelet transfusions are associated with substantial complications, including transfusion reactions, platelet alloimmunization, and transfusion-transmitted viral infections. Eltrombopag, a thrombopoietin receptor agonist (TPO-RA), carries a black-box warning for hepatotoxicity, with a real-world incidence of 11.8%. Transplant recipients frequently experience diarrhea, which impairs the absorption of oral thrombopoietic agents, while daily subcutaneous injections exacerbate pediatric patient discomfort and reduce treatment adherence. In contrast, long-acting TPO-RAs administered once post-transplant have demonstrated favorable tolerability and promising efficacy in thalassemia transplant pediatric populations. To date, clinical data on Hetrombopag use for platelet recovery in haploidentical HSCT for pediatric thalassemia are lacking. Therefore, this observational study aims to evaluate the efficacy and safety of Hetrombopag in facilitating platelet engraftment during haploidentical HSCT in children with thalassemia.
- Conditions
- Thalassemia in Children
- Being tested
- Hetrombopag
- Keywords
- Thalassemia, HSCT, Hetrombopag
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HetrombopagDrug
Also called Hetrombopag Olamine
In allogeneic hematopoietic stem cell transplantation, hetrombopag is initiated at 3 µg/kg subcutaneously on day +6 post-transplant. The dose is increased by 2 µg/kg weekly up to a maximum of 10 µg/kg. Treatment is discontinued when platelet counts rise to 100×10⁹/L. If platelet counts remain ≤20×10⁹/L on day +20, hetrombopag is combined with eltrombopag 25 mg orally once daily. Fresh apheresis platelet suspensions (1 therapeutic dose, containing >2.5×10¹¹ platelets) are administered when platelet counts are ≤20×10⁹/L or when counts are between 21-50×10⁹/L with active bleeding. If engraftment has not occurred by day +28 post-transplant, re-transplantation is required, and hetrombopag is considered ineffective.
| Study group | Role | What they receive |
|---|---|---|
| Experimental group | In allogeneic hematopoietic stem cell transplantation, hetrombopag is initiated at 3 µg/kg subcutaneously on day +6 post-transplant. The dose is increased by 2 µg/kg weekly up to a maximum of 10 µg/kg. Treatment is discontinued when platelet counts rise to 100×10⁹/L. If platelet counts remain ≤20×10⁹/L on day +20, hetrombopag is combined with eltrombopag 25 mg orally once daily. Fresh apheresis platelet suspensions (1 therapeutic dose, containing >2.5×10¹¹ platelets) are administered when platelet counts are ≤20×10⁹/L or when counts are between 21-50×10⁹/L with active bleeding. If engraftment has not occurred by day +28 post-transplant, re-transplantation is required, and hetrombopag is considered ineffective. Receives: Drug: Hetrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet recovery time
The time at nodes such as platelet >20×10^9/L, 50×10^9/L and 100×10^9/L
Measured: From enrollment to 28 days after transplantation
Platelet transfusion volume
Required dosage of platelet suspension
Measured: From enrollment to 28 days after transplantation
Secondary outcomes (8)
The rate of adverse drug reactions
The rate of adverse drug reactions occurring during the follow-up period
Measured: From enrollment to 28 days after transplantation
Bleeding incidence rate
The incidence rate of bleeding during the follow-up period
Measured: From enrollment to 28 days after transplantation
Thrombosis incidence rate
The incidence rate of thrombosis during the follow-up period
Measured: From enrollment to 28 days after transplantation
Survival rate
Measured: From enrollment to 28 days after transplantation
Relapse-free survival rate
Measured: From enrollment to 28 days after transplantation
Transplant-related mortality rate
Measured: From enrollment to 28 days after transplantation
Major transplant-related complications
Measured: From enrollment to 28 days after transplantation
Cause of death
Measured: From enrollment to 28 days after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2025
- Study start1 Sep 2024
- Primary completion30 Apr 2026
- Study completion30 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia in ChildrenStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Xiaoyang Yang, MD, Principal investigator, Department of Hematology, Haikou People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07003269
- Sponsor's protocol ID
- SC20240561
- Status last verified
- Jul 2026
- First submitted
- 15 May 2025
- Last updated
- 28 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: The IPD encompasses highly sensitive and confidential data that was collected through significant investment of our resources, both in terms of time and funding. This data is integral to our ongoing research initiatives, which are at a crucial stage of development. Premature sharing could disrupt our research timelines and strategic plans. Additionally, we have not yet established comprehensive safeguards to ensure that the data will be used appropriately by external researchers. Without proper protocols in place, there is a risk of misuse or misinterpretation of the data, which could lead to inaccurate research outcomes and potential reputational damage to our institution. For these reasons, we have decided not to share the IPD at this time.
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Study on the Promotion of Platelet Recovery by Hetrombopag During Haploidentical Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07003269
- Acronym / sponsor ID
- SC20240561
- Conditions
- Thalassemia in Children
- Keywords
- Thalassemia, HSCT, Hetrombopag
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Haikou Affiliated Hospital of Central South University Xiangya School of MedicineUniversity, hospital or other
- Study start
- 1 Sep 2024 (actual)
- Primary completion
- 30 Apr 2026 (actual)
- Study completion
- 30 Apr 2026 (actual)
- First posted
- 4 Jun 2025 (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 objective of this observational study is to investigate the long-term effects of Hetrombopag in promoting platelet engraftment during haploidentical hematopoietic stem cell transplantation (HSCT) in children with thalassemia, with a specific focus on a 28-day time window post-transplantation. The core question to be addressed is: Is Hetrombopag safe and effective for platelet engraftment in children with thalassemia undergoing haploidentical HSCT within a 28-day post-transplant period? Subjects who received Hetrombopag as part of routine care for haploidentical HSCT in children with thalassemia will be required to complete a 28-day online survey on platelet engraftment outcomes.
Detailed description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains a critical and often the sole curative modality for diverse hematological malignancies and disorders. Post-transplant thrombocytopenia (platelet count <20×10⁹/L) significantly compromises long-term patient survival, with an incidence of 5-20% in allo-HSCT recipients, thereby escalating treatment morbidity and costs. Current research on platelet engraftment promotion in pediatric thalassemia patients undergoing allo-HSCT is limited. Repeated platelet transfusions are associated with substantial complications, including transfusion reactions, platelet alloimmunization, and transfusion-transmitted viral infections. Eltrombopag, a thrombopoietin receptor agonist (TPO-RA), carries a black-box warning for hepatotoxicity, with a real-world incidence of 11.8%. Transplant recipients frequently experience diarrhea, which impairs the absorption of oral thrombopoietic agents, while daily subcutaneous injections exacerbate pediatric patient discomfort and reduce treatment adherence. In contrast, long-acting TPO-RAs administered once post-transplant have demonstrated favorable tolerability and promising efficacy in thalassemia transplant pediatric populations. To date, clinical data on Hetrombopag use for platelet recovery in haploidentical HSCT for pediatric thalassemia are lacking. Therefore, this observational study aims to evaluate the efficacy and safety of Hetrombopag in facilitating platelet engraftment during haploidentical HSCT in children with thalassemia.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental group | – | Drug: HetrombopagIn allogeneic hematopoietic stem cell transplantation, hetrombopag is initiated at 3 µg/kg subcutaneously on day +6 post-transplant. The dose is increased by 2 µg/kg weekly up to a maximum of 10 µg/kg. Treatment is discontinued when platelet counts rise to 100×10⁹/L. If platelet counts remain ≤20×10⁹/L on day +20, hetrombopag is combined with eltrombopag 25 mg orally once daily. Fresh apheresis platelet suspensions (1 therapeutic dose, containing >2.5×10¹¹ platelets) are administered when platelet counts are ≤20×10⁹/L or when counts are between 21-50×10⁹/L with active bleeding. If engraftment has not occurred by day +28 post-transplant, re-transplantation is required, and hetrombopag is considered ineffective. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- The Department of Hematology of Haikou People's Hospital accepts children with severe thalassemia aged 2 - 17 years old for haploidentical hematopoietic stem cell transplantation.
Key inclusion criteria (3 listed)
- Diagnosed with severe thalassemia via thalassemia gene testing, transfusion history, and complete blood count (CBC).
- Pediatric patients aged 2-17 years.
- Consented to haploidentical transplantation and evaluated by the transplant team as having no transplant contraindications.
Key exclusion criteria (6 listed)
- Presence of a fully matched donor with refusal of haploidentical transplantation.
- Donor or recipient with transaminase levels >2× the upper limit of normal (ULN).
- Positive hepatitis B DNA test result.
- Active infection at the time of enrollment.
- Donor-specific antibodies (DSA) >5,000 and unable to decrease below 3,000 after antibody therapy.
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 |
|---|---|---|
| HetrombopagAlso: Hetrombopag OlamineIn allogeneic hematopoietic stem cell transplantation, hetrombopag is initiated at 3 µg/kg subcutaneously on day +6 post-transplant. The dose is increased by 2 µg/kg weekly up to a maximum of 10 µg/kg. Treatment is discontinued when platelet counts rise to 100×10⁹/L. If platelet counts remain ≤20×10⁹/L on day +20, hetrombopag is combined with eltrombopag 25 mg orally once daily. Fresh apheresis platelet suspensions (1 therapeutic dose, containing >2.5×10¹¹ platelets) are administered when platelet counts are ≤20×10⁹/L or when counts are between 21-50×10⁹/L with active bleeding. If engraftment has not occurred by day +28 post-transplant, re-transplantation is required, and hetrombopag is considered ineffective. | Drug | Experimental group |
Primary outcome
Platelet recovery time
Time frame: From enrollment to 28 days after transplantation
The time at nodes such as platelet >20×10^9/L, 50×10^9/L and 100×10^9/L
Platelet transfusion volume
Time frame: From enrollment to 28 days after transplantation
Required dosage of platelet suspension
8 secondary outcomes
The rate of adverse drug reactions
Time frame: From enrollment to 28 days after transplantation
Bleeding incidence rate
Time frame: From enrollment to 28 days after transplantation
Thrombosis incidence rate
Time frame: From enrollment to 28 days after transplantation
Survival rate
Time frame: From enrollment to 28 days after transplantation
Relapse-free survival rate
Time frame: From enrollment to 28 days after transplantation
Transplant-related mortality rate
Time frame: From enrollment to 28 days after transplantation
Major transplant-related complications
Time frame: From enrollment to 28 days after transplantation
Cause of death
Time frame: From enrollment to 28 days after transplantation
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 28 Jul 2026.
- Individual participant dataSharing planned: No. The IPD encompasses highly sensitive and confidential data that was collected through significant investment of our resources, both in terms of time and funding. This data is integral to our ongoing research initiatives, which are at a crucial stage of development. Premature sharing could disrupt our research timelines and strategic plans. Additionally, we have not yet established comprehensive safeguards to ensure that the data will be used appropriately by external researchers. Without proper protocols in place, there is a risk of misuse or misinterpretation of the data, which could lead to inaccurate research outcomes and potential reputational damage to our institution. For these reasons, we have decided not to share the IPD at this time.
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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.
Xiaoyang Yang, MD principal investigator
Department of Hematology, Haikou People's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thalassemia in Children, run by Haikou Affiliated Hospital of Central South University Xiangya School of Medicine 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 3 inclusion and 6 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 28 Jul 2026. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
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?
Platelet recovery time (time frame: From enrollment to 28 days after transplantation). 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?
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 28 Jul 2026.