Super transplantation for treating severe beta-thalassemia in children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical study evaluates the safety and efficacy of a novel preconditioning-free super-transplantation approach for pediatric patients with severe beta-thalassemia. The study involves screening patients for eligibility, mobilizing haplo-identical or fully matched donors, and infusing peripheral blood mononuclear cells without traditional conditioning. Participants will receive reduced dosages of immunosuppressants and will be monitored for infection management. The goal is to assess the effectiveness of this innovative transplantation method in improving patient outcomes.

Why it matters

Who can join: the full rules

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.

Where it's running

• Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University
  Guangzhou, Guangdong, China Recruiting
• Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University
  Guangzhou, Guangdong, China Active not recruiting

Study contacts

• Study coordinator: Hua Jiang, Doctor
• Email: jiang_hua18@sina.cn
• Phone: 86-020-38076501

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Prophylactic treatment using donor lymphocytes after haploidentical stem cell transplantation

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• Impact of haploidentical stem cell donation on donor psychology

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• Transplanting stem cells from mismatched donors for sickle cell disease and other anemias

  This study is testing if using stem cells from mismatched donors can help people with severe sickle cell disease and other anemias who don't have matching donors.

  Recruiting Phase 1/2 University of Pittsburgh · Pittsburgh, Pennsylvania · NCT03653338

• SP-420 to remove excess iron in adults with transfusion-dependent beta thalassemia or low‑risk MDS

  This will test whether SP-420 can clear excess liver iron in adults who receive regular transfusions for beta thalassemia or transfusion‑dependent low‑risk myelodysplastic syndrome.

  Recruiting Phase 2 Pharmacosmos A/S · Copenhagen · NCT05693909

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.