Thalassemia in ChildrenNCT07003256
Observational Study: Romiplostim 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 explore the long-term effects of roprastine given to promote platelet implantation in hematopoietic stem cell hemicongruent transplantation in children with thalassemia. 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?
- Romiplostim
- Type of treatment
- Pill or oral medicine, Injection or infusion
- How many people
- 16 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2024to 30 Apr 2026
- How long will I take part?
- 28 Days
- 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: Children aged 2 - 17 years old who were diagnosed with severe thalassemia at Haikou People's Hospital and received haploidentical hematopoietic stem cell transplantation.
Who may be able to join
- After undergoing Mediterranean gene testing, reviewing the history of blood transfusions, and conducting a blood routine examination, the patient was diagnosed with severe thalassemia.
- Children aged 2 - 17 years old.
- Agree to the haploidentical transplantation, and the team has evaluated that there are no transplantation contraindications.
Who cannot join
- There is a fully matched donor, and transplantation with a half - matched donor is not agreed.
- For donors and recipients, the transaminase is more than twice the normal value.
- Donor specific antibody Greater than 5000, and after antibody treatment, it should not be lower than 3000.
- Positive for hepatitis B DNA.
- There is an active infection.
- After evaluation by the transplantation team, there are contraindications for transplantation.
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 Romiplostim During Haploidentical Hematopoietic Stem Cell Transplantation
The objective of this observational study is to explore the long-term effects of roprastine given to promote platelet implantation in hematopoietic stem cell hemicongruent transplantation in children with thalassemia. The main questions it aimed to answer is:
Is roprastine safe and effective for platelet implantation in children with thalassemia hemicongruent transplantation? Participants who have already received roprastine as part of routine medical care for hematopoietic stem cell hemicongruent transplantation in children with thalassemia will answer online survey questions about the effects of their platelet implantation within 8 weeks.
Read the detailed description
Allogeneic hematopoietic stem cell transplantation is an important, if not the only, means of curing many diseases of the blood system. Thrombocytopenia after transplantation seriously affects the long-term survival rate of patients. allo - The incidence of thrombocytopenia in HSCT patients is 5-20% (< 20 x 10 ^ 9/L), increasing the risk and cost of treatment. There are currently few studies on the promotion of platelet growth in children with thalassemia. Repeated infusion of platelet suspension can lead to many adverse consequences, including blood transfusion reactions, platelet homeoimmune responses, and transfusion-associated virus infections. There is a black box warning of liver toxicity in eltropopa, and the incidence of real-world liver toxicity is 11.8%. Transplant patients are prone to diarrhea, which affects the absorption of oral platelet-raising drugs. Daily subcutaneous injection increases children's pain and poor tolerance. However, the long-acting platelet-raising drugs once after transplantation are well tolerated in children with thalassemia transplantation, and the efficacy is worth looking forward to. Up to now, there is a lack of relevant clinical data on the application of roprastine to promote platelet recovery in children with thalassemia hemiphase transplantation. Therefore, this study aimed to explore the observational study of roprastine to promote platelet implantation in children with thalassemia hemiphase transplantation, and to explore the efficacy and safety of the drug.
- Conditions
- Thalassemia in Children
- Being tested
- Romiplostim
- Keywords
- Thalassemia, Romiplostim, HSCT
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Also called Nplate
After transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine is considered ineffective.
| Study group | Role | What they receive |
|---|---|---|
| experimental group | After transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine is considered ineffective. Receives: Drug: Romiplostim |
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
The required dosage of platelet suspension
Measured: From enrollment to 28 days after transplantation
Secondary outcomes (8)
Adverse drug reaction rate
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 that occurred during the follow-up period
Measured: From enrollment to 28 days after transplantation
Thrombosis incidence rate
The incidence rate of thrombosis occurring during the follow-up period
Measured: From enrollment to 28 days after transplantation
Survival rate
The survival rate of patients after transplantation medication
Measured: From enrollment to 28 days after transplantation
Recurrence - free survival rate
The survival rate of patients without recurrence of the disease after using the drug
Measured: From enrollment to 28 days after transplantation
Transplant - related mortality
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
- 16 participants (actual)
- Target duration
- 28 Days
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
- NCT07003256
- Sponsor's protocol ID
- JVKY2024-0101015002
- 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 Romiplostim During Haploidentical Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07003256
- Acronym / sponsor ID
- JVKY2024-0101015002
- Conditions
- Thalassemia in Children
- Keywords
- Thalassemia, Romiplostim, HSCT
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 16 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 explore the long-term effects of roprastine given to promote platelet implantation in hematopoietic stem cell hemicongruent transplantation in children with thalassemia. The main questions it aimed to answer is:
Is roprastine safe and effective for platelet implantation in children with thalassemia hemicongruent transplantation? Participants who have already received roprastine as part of routine medical care for hematopoietic stem cell hemicongruent transplantation in children with thalassemia will answer online survey questions about the effects of their platelet implantation within 8 weeks.
Detailed description
Allogeneic hematopoietic stem cell transplantation is an important, if not the only, means of curing many diseases of the blood system. Thrombocytopenia after transplantation seriously affects the long-term survival rate of patients. allo - The incidence of thrombocytopenia in HSCT patients is 5-20% (< 20 x 10 ^ 9/L), increasing the risk and cost of treatment. There are currently few studies on the promotion of platelet growth in children with thalassemia. Repeated infusion of platelet suspension can lead to many adverse consequences, including blood transfusion reactions, platelet homeoimmune responses, and transfusion-associated virus infections. There is a black box warning of liver toxicity in eltropopa, and the incidence of real-world liver toxicity is 11.8%. Transplant patients are prone to diarrhea, which affects the absorption of oral platelet-raising drugs. Daily subcutaneous injection increases children's pain and poor tolerance. However, the long-acting platelet-raising drugs once after transplantation are well tolerated in children with thalassemia transplantation, and the efficacy is worth looking forward to. Up to now, there is a lack of relevant clinical data on the application of roprastine to promote platelet recovery in children with thalassemia hemiphase transplantation. Therefore, this study aimed to explore the observational study of roprastine to promote platelet implantation in children with thalassemia hemiphase transplantation, and to explore the efficacy and safety of the drug.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Target follow-up
- 28 Days
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| experimental group | – | Drug: RomiplostimAfter transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine is considered ineffective. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children aged 2 - 17 years old who were diagnosed with severe thalassemia at Haikou People's Hospital and received haploidentical hematopoietic stem cell transplantation.
Key inclusion criteria (3 listed)
- After undergoing Mediterranean gene testing, reviewing the history of blood transfusions, and conducting a blood routine examination, the patient was diagnosed with severe thalassemia.
- Children aged 2 - 17 years old.
- Agree to the haploidentical transplantation, and the team has evaluated that there are no transplantation contraindications.
Key exclusion criteria (6 listed)
- There is a fully matched donor, and transplantation with a half - matched donor is not agreed.
- For donors and recipients, the transaminase is more than twice the normal value.
- Donor specific antibody Greater than 5000, and after antibody treatment, it should not be lower than 3000.
- Positive for hepatitis B DNA.
- There is an active infection.
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 |
|---|---|---|
| RomiplostimAlso: NplateAfter transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine 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
The required dosage of platelet suspension
8 secondary outcomes
Adverse drug reaction rate
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
Recurrence - free survival rate
Time frame: From enrollment to 28 days after transplantation
Transplant - related mortality
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.