CompletedNot Applicable

Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT

China30 participantsStarted 2024-09-01

Plain-language 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. 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.

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: * 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. Exclusion Criteria: * 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.

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 IDNCT07003269

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