RecruitingNot Applicable

Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia

China3 participantsStarted 2024-10-01

Plain-language summary

This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.

Who can participate

Age range

7 Years – 10 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

. Diagnosis of severe β -thalassemia
. Age between 7-10 years old, male and female; Weight \< 40kg
. The patient has or does not have an HLA-compatible or semi-compatible donor, but unconditional transplantation or refusal of blood stem cell transplantation; Patients with thalassemia gene therapy without conditions or refusal;
. There are fully compatible or incompatible HLA donors, and the physical examination meets the donor conditions;
. The patient and family members agree to receive hypertransplant therapy and sign a written informed consent prior to the transplant trial.

Exclusion criteria

. Mental patients;
. Participants in other drug clinical trials within the past 1 month;
. There are no suitable HLA-incompatible donors.
. Other researchers decide that it is not suitable to participate in this researcher.

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

chimeric status

Timeframe: 1 year after transplantation.

Trial details

NCT IDNCT06734520

SponsorGuangzhou Women and Children's Medical Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2029-07-01

Contact for this trial

Hua Jiang, Doctor

86-020-38076501 jiang_hua18@sina.cn

View on ClinicalTrials.gov More Thalassemia Majors (Beta-Thalassemia Major) trials