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

Guangzhou Women and Children's Medical Center

Status: Enrolling

Conditions: Thalassemia Majors (Beta-Thalassemia Major); Haplo-identical Donors

Treatments: Drug: super-transplantation in 3 severe β -thalassemia pediatric patients.

Study type: Interventional

Funder types: Other

Identifiers

NCT06734520

2024217A01

Details and patient eligibility

About

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.

Full description

Patient screening:

1. Two months before transplantation, pediatric patients are evaluated by our center, and the patients meeting the inclusion criteria will be screened into the group.
2. Investigators and doctors will give detailed information about the benefits and risks of participating in the study, and the informed consents are further signed.

Transplant conduction:

1. Donor mobilization: HLA-fully matched or haplo-identical donors are mobilized subcutaneously for five days with human granulocyte colony-stimulating factor (G-CSF) and the peripheral blood mononuclear cells (PBMCs) that contain a certain numbers of CD34+ stem cells and CD3+ lymphocytes will be extracted by blood cell separators.
2. Transplant regimen: Hydroxycarbamide will be used to reduce the total white blood cells before transplant. However, no traditional transplantation conditioning will be performed, including but not limited to busulfan, fludarabine, Cyclophosphamide, ATG, and irradiation. Splenectomy should be performed if the longitudinal diameter of the spleen exceeds the normal value by 4 cm.
3. Cell infusion: Pediatric patients will be infused with the PBMCs intravenously at day 0.
4. GVHD prophylaxis: The dosage of immunosuppressants is half of the conventional post-transplantation level, but adjustments to the dosage may be necessary based on specific circumstances.
5. Infection management: Antibacterial, antifungal medications, and anti-Pneumocystis medications need to be administered during the period of granulocytopenia. The preemptive treatment for cytomegalovirus will be performed when the virus serological test is positive.

Enrollment: 3 estimated patients

Sex: All

Ages: 7 to 10 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

Severe β -thalassemia

Experimental group

Description:

Children with β-thalassemia major received super transplantation, and chimerism tests were performed at 3 months, 6 months, 9 months, and 1 year after transplantation to evaluate the safety and effectiveness.

Treatment:

Drug: super-transplantation in 3 severe β -thalassemia pediatric patients.