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

Launched by GUANGZHOU WOMEN AND CHILDREN'S MEDICAL CENTER · Dec 12, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called "super-transplantation" for children with severe beta-thalassemia, which is a serious blood disorder. The trial aims to see how safe and effective this treatment is for young patients, ages 7 to 10, who weigh less than 40 kg. Children can participate whether they have a compatible donor or not, as long as they and their families agree to the treatment and sign a consent form.

If your child qualifies for this study, they will receive the super-transplantation without any preconditioning, which means they won’t need extra treatments to prepare their body for the transplant. The trial is currently recruiting participants, and it’s important to know that children with certain mental health conditions, those involved in other clinical trials recently, or those without suitable donors may not be eligible. Overall, this trial offers a potential new option for treating a challenging condition, and families will be closely involved in the decision-making process.

Gender

ALL

Eligibility criteria

• 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.