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
This trial does not accept healthy volunteersQualifiers
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
Design model
Single group
Treatments tested in this trial
super-transplantation in 3 severe β -thalassemia pediatric patients.
DrugHaplo-identical donors are mobilized with G-CSF and PBMCs that contain CD34+ stem cells and CD3+ lymphocytes will be extracted by blood cell separators. Then the patients will be infused with the PBMC
Treatment groups
3 Participants
are divided into 1 treatment groupGroup A: Severe β -thalassemiaExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
chimeric status
Detecting the chimerism in recipients' peripheral blood and bone marrow.Chimerism is generally measured in percentages(%)
Time frame
1 year after transplantation.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov