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.