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

Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age7-10
SponsorGuangzhou Women and Children's Medical Center

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