super-transplantation in 3 severe β -thalassemia pediatric patients.
super-transplantation in 3 severe β -thalassemia pediatric patients.: Haplo-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
Study 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.
Eligibility
Sex
ALL
Min age
7 Years
Max age
10 Years
Healthy volunteers
No
Inclusion Criteria:
1. Diagnosis of severe β -thalassemia
2. Age between 7-10 years old, male and female; Weight \< 40kg
3. 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;
4. There are fully compatible or incompatible HLA donors, and the physical examination meets the donor conditions;
5. The patient and family members agree to receive hypertransplant therapy and sign a written informed consent prior to the transplant trial.
Exclusion Criteria:
1. Mental patients;
2. Participants in other drug clinical trials within the past 1 month;
3. There are no suitable HLA-incompatible donors.
4. Other researchers decide that it is not suitable to participate in this researcher.
Primary outcome measure(s)
chimeric status — 1 year after transplantation. Detecting the chimerism in recipients' peripheral blood and bone marrow.Chimerism is generally measured in percentages(%)
Trial sites (2)
Facility
City
Region
Status
Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University
Guangzhou
Guangdong
Active Not Recruiting
Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University
Guangzhou
Guangdong
Recruiting
More Guangzhou Women and Children's Medical Center trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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