About this trial

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.

Eligibility criteria

Qualifiers

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;

Disqualifiers

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.

Trial design

Treatments tested in this trial

  • super-transplantation in 3 severe β -thalassemia pediatric patients.

Treatment groups

3 Participants
are divided into 1 treatment group

Locations

2
Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University51000, GuangzhouGuangdong, China
Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University GuangzhouGuangdong, China

Sponsors and collaborators

Guangzhou Women and Children's Medical Center

Lead sponsor