A Clinical Study of Ultra-transplantation for the Treatment of Major Thalassemia Scheme

ConditionStem Cell Transplantation

Trial statusRecruiting

Trial phaseNot applicable

Trial typeInterventional

Biological sexAll

Age7-12

SponsorHu Peng

About this trial

Yunnan is a high-incidence area of Eastern Mediterranean (thalassemia) in China, and the treatment cost of thalassemia patients is high, hematopoietic stem cell transplantation (HSCT) is the only means to cure thalassemia, but there are problems in donor screening and the risk of complications. Professor Ai Huisheng's team proposed a new concept of hypertransplantation, which does not require pretreatment and has no risk of GVHD, and animal experiments have shown good efficacy. Under the guidance of Professor Ai, the center plans to carry out clinical research on hypertransplantation and explore safe and effective new therapies for thalassemia.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosed with severe Mediterranean anemia, with no restrictions on alpha and beta types;

Age between 7-12 years old, male or female not limited; Weight<40kg

The patient has or does not have HLA matched or semi matched donors, but unconditionally transplants or refuses to undergo blood stem cell transplantation treatment; And patients who unconditionally or refuse to undergo gene therapy for thalassemia;

There are HLA matched or mismatched donors who meet the donor criteria through physical examination;

Disqualifiers

Psychiatric patients;

Participants in clinical trials of other drugs within the past month;

There are no suitable HLA mismatched donors available.

Other researchers have determined that they are not suitable to participate in this study.

Trial design

Design model

Sequential

Treatments tested in this trial

Supergraft
Other intervention
This study is a prospective, single-center, single-arm clinical study, and it is planned to include 3-5 patients with Eastern Mediterranean major who have no HSCT indication and cannot undergo Eastern Mediterranean gene therapy. The diagnosis of Eastern Mediterranean major was determined by the genotype of Eastern Mediterranean and the clinical manifestations of patients, and patients who met the inclusion criteria were screened and enrolled and received super transplant therapy.

Treatment groups

5 Participants

are divided into 1 treatment group

Group A: SupergraftExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

Donor cell implantation rate

Using polymerase chain reaction (PCR) to amplify short tandem repeat sequences Monitor the chimerism status of the donor and recipient to quantitatively evaluate the implantation rate of donor cells. Specifically, we will determine the percentage of donor cells and evaluate the implantation rate by comparing the DNA sequence differences between donor and recipient cells before and after transplantation.

Time frame

3 months after transplantation

Secondary outcomes

carriers of the Mediterranean anemia gene

Determine the status of carriers of Mediterranean anemia genes through gene sequencing technology. We will use specific molecular genetic methods, such as PCR and DNA sequencing, to identify and confirm specific mutations in the Mediterranean anemia gene.

Time frame

one year

Other outcomes

evaluation of growth and development

We will use standard assessment tools for children's growth and development, such as： Height Assessment: Outcome measure: Height in meters (m). Description: Monitor children's height growth and compare with standard data for children of the same age. weight assessment: Outcome measure: Body weight in kilograms. Description: Monitor children's weight gain and compare with standard data for children of the same age. Head circumference assessment: Outcome measure: Head circumference in centimeters (cm). Description: Monitor the growth of head circumference in children and compare it with standard data for children of the same age. Body mass index (BMI) assessment (if combined height and weight are required): Outcome measures: Body mass index (BMI) was measured in kg/m\^2. Description: BMI values are obtained by calculating the weight (kg) divided by the square of height (m) to assess the proportion of a child's weight relative to height.

Time frame

one year

Sponsors and contacts

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Hu Peng

Lead sponsor

The First People's Hospital of Yunnan

Sponsor institution

Source ClinicalTrials.gov

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