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

Launched by HU PENG · Dec 17, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new method called hypertransplantation to treat major thalassemia, a serious blood disorder that affects how the body makes hemoglobin, the protein that carries oxygen in the blood. Traditional treatment methods, like stem cell transplantation, can be complicated and have risks, but hypertransplantation does not require pre-treatment and aims to reduce complications. This study will explore whether this innovative approach can provide a safe and effective treatment for children with thalassemia.

To participate in the trial, children aged 7 to 12 years old who have been diagnosed with severe Mediterranean anemia may be eligible. They can weigh up to 40 kg and may or may not have matched donors for transplantation. Families must agree to the treatment and sign a consent form. Participants will receive hypertransplantation treatment under careful supervision, and the goal is to determine how well this method works and how safe it is for young patients. It's important to note that certain individuals, such as those with psychiatric conditions or who are currently involved in other clinical trials, will not be able to join this study.

Gender

ALL

Eligibility criteria

• Patient inclusion criteria :
• • 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.
• （2） Exclusion criteria for patients:
• • 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.