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

NCT06743477 | Recruiting DMC

• 2 other identifiers: KHLL2024-KY216Hu Peng
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• 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.

  3 months after transplantation

Secondary Outcomes (1)

• carriers of the Mediterranean anemia gene

  one year

Other Outcomes (1)

• evaluation of growth and development

  one year

Study Arms (1)

Supergraft

EXPERIMENTAL

Hypertransplantation is an innovative treatment plan of Professor Ai Huisheng's team, which uses hematopoietic stem cell transplantation from healthy donors who are haplotype compatible with the patient, and observes the patient's cell reinfusion response, hematopoietic reconstitution, hemoglobin level and blood transfusion, thalassemia clonal clearance, immune reconstitution, endocrine function recovery, gastrointestinal function recovery, as well as the incidence of graft-versus-host disease, infection and other complications after transplantation.

Other: Supergraft

Interventions

Supergraft OTHER

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.

Supergraft

Eligibility Criteria

• Age: 7 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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;
• The patient and their family agree to receive super transplantation treatment and sign a written informed consent form before the transplantation trial.

You may not qualify if:

• 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.
• （3） Supplier screening criteria:
• HLA typing matches the patient's haplotype
• KIR configuration
• NIMA
• DSA negative
• Routine physical examination
• Genetic screening for carriers of thalassemia, mild or no carriers of thalassemia
• Sign the informed consent form.

Sponsors & Collaborators

• Hu Peng: lead

Study Sites (1)

Yunnan First People's Hospital

Kunming, Yunnan, 650000, China

RECRUITING

Study Officials

• Yang T Yang, master

  The First People's Hospital of Yunnan

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yang tonghua T Yang, master

CONTACT

86-13888555608; ynanblood@aliyun.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Deputy Chief Physician

Study Record Dates

• First Submitted: November 24, 2024
• First Posted: December 20, 2024
• Study Start: August 29, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: September 2, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF
• Time Frame: within 6 months after the end of the trial
• Access Criteria: The IPD and supporting information will be accessible to researchers, academics, and the public with a legitimate interest in the study. They will be able to access the raw data, including metadata and case record form data, which are essential components of the study. Access to this data will be facilitated through a clinical trial public management platform, where the IPD will be openly available for query. Additionally, interested parties can directly contact the researchers to request access to the data. This approach ensures that the data is not only available to those within the scientific community but also to the general public, promoting transparency and broadening the impact of the research findings.

Locations

CN (1)