CompletedNot Applicable

Observational Study: Romiplostim for Platelet Recovery in Haploidentical HSCT

China16 participantsStarted 2024-09-01

Plain-language 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. 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.

Who can participate

Age range

2 Years – 17 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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. Exclusion Criteria: * 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Platelet recovery time

Timeframe: From enrollment to 28 days after transplantation

Platelet transfusion volume

Timeframe: From enrollment to 28 days after transplantation

Trial details

NCT IDNCT07003256

SponsorHaikou Affiliated Hospital of Central South University Xiangya School of Medicine

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2026-04-30

View on ClinicalTrials.gov More Thalassemia in Children trials