Super transplantation for treating severe beta-thalassemia in children
Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
This study is testing a new way to do a stem cell transplant for children with severe beta-thalassemia to see if it can help them feel better without the usual heavy treatments.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 3 (estimated) |
| Ages | 7 Years to 10 Years |
| Sex | All |
| Sponsor | Guangzhou Women and Children's Medical Center Academic / other |
| Drugs / interventions | Cyclophosphamide, fludarabine |
| Locations | 2 sites (Guangzhou, Guangdong and 1 other locations) |
| Trial ID | NCT06734520 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients aged 7-10 years with a diagnosis of severe beta-thalassemia who weigh less than 40 kg.
Not a fit: Patients with no suitable HLA-incompatible donors or those with mental health issues may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a safer and more effective treatment option for children suffering from severe beta-thalassemia.
How similar studies have performed: While this approach is innovative, similar studies have shown promise in the field of transplantation, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Guangzhou, Guangdong and 1 other locations
- Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University — Guangzhou, Guangdong, China (Active_not_recruiting)
- Guangzhou Women and Children's Medical Center Affiliated to Guangzhou Medical University — Guangzhou, Guangdong, China (Recruiting)
Study contacts
- Study coordinator: Hua Jiang, Doctor
- Email: jiang_hua18@sina.cn
- Phone: 86-020-38076501
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.