Ultra-transplantation (hypertransplantation) for major thalassemia

Clinical Study Protocol for Ultra-transplantation for the Treatment of Eastern Mediterranean Major

This project will try ultra-transplantation (hypertransplantation), a new stem-cell approach without pretreatment, in children aged 7–12 with severe thalassemia who cannot or decline standard stem cell or gene therapies.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 5 (estimated)
• Ages: 7 Years to 12 Years
• Sex: All
• Sponsor: The First People's Hospital of Yunnan (Academic / other)
• Drugs / interventions: chemotherapy, radiation
• Locations: 1 site (Kunming, Yunnan)
• Trial ID: NCT06743477 on ClinicalTrials.gov

What this trial studies

Researchers at Yunnan First People's Hospital will perform the 'Supergraft' ultra-transplantation, a hematopoietic stem-cell procedure designed to require no conditioning and to avoid graft‑versus‑host disease, building on promising animal data. Eligible children aged 7–12 with severe thalassemia and available HLA-matched or mismatched donors will be enrolled after informed consent and weight verification. The single-center intervention in Kunming will include donor screening, the Supergraft procedure, and follow-up to monitor safety, complications, and clinical outcomes such as transfusion independence. The trial seeks to determine whether this method is a safe and effective alternative to standard HSCT or lifelong transfusions.

Who should consider this trial

Good fit: Children aged 7–12 with severe thalassemia weighing under 40 kg who have available HLA-matched or mismatched donors and who cannot or refuse standard HSCT or gene therapy, and who provide informed consent, are the intended candidates.

Not a fit: Patients outside the 7–12 age range, those without any suitable donor, psychiatric patients, or those recently enrolled in other drug trials are unlikely to benefit or are excluded.

Why it matters

Potential benefit: If successful, this approach could offer a simpler, lower‑risk curative transplant that reduces or ends lifelong blood transfusions for children with severe thalassemia.

How similar studies have performed: The hypertransplantation concept is novel in humans: animal experiments reported good efficacy, but clinical data in people are currently limited or lacking.

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.

Where this trial is running

Kunming, Yunnan

• Yunnan First People's Hospital — Kunming, Yunnan, China (Recruiting)

Study contacts

• Principal investigator: Yang T Yang, master — The First People's Hospital of Yunnan
• Study coordinator: Yang tonghua T Yang, master

Conditions Stem Cell Transplantation