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A drug that boosts platelets may speed recovery after stem cell transplants in kids with thalassemia

NCT ID NCT07003269

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 28, 2026 · Last updated Jul 29, 2026 · Updated 1 time

Summary

This observational study tests whether hetrombopag, a platelet-boosting drug, can safely speed platelet recovery in children with thalassemia who receive a half-matched (haploidentical) stem cell transplant. Thirty children aged 2 to 17 will be followed for 28 days after transplant to track how quickly their platelet counts rise and how many transfusions they need. The study also monitors for side effects like bleeding or blood clots.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
hetrombopag, a drug that stimulates platelet production, given as a shot after stem cell transplant
What this could lead to
If hetrombopag proves safe and effective, it could speed platelet recovery and reduce the need for platelet transfusions in children with thalassemia after a stem cell transplant.
What could go wrong
This is a small observational study with only 30 children, so results may not apply to everyone. The drug may cause side effects like bleeding or blood clots.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

30 people

The number who actually took part.

Started

Sep 2024

Finished

Apr 2026

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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.

Ages

2 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Haikou Affiliated Hospital of Central South University Xiangya School of Medicine

    Haikou, Hainan, 570208, China

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