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New treatment option for Thalassemia Majors (Beta-Thalassemia Major)
Official title Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
ClinicalTrials.gov ID: NCT06734520
What this study is testing
What is super-transplantation in 3 severe β -thalassemia pediatric patients.?
super-transplantation in 3 severe β -thalassemia pediatric patients. is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for thalassemia majors (beta-thalassemia major).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 7 to 10
You may be able to join if
- Diagnosis of severe β -thalassemia
- Age between 7-10 years old, male and female; Weight \< 40kg
- The patient has or does not have an HLA-compatible or semi-compatible donor, but unconditional transplantation or refusal of blood stem cell...
- There are fully compatible or incompatible HLA donors, and the physical examination meets the donor conditions;
- The patient and family members agree to receive hypertransplant therapy and sign a written informed consent prior to the transplant trial.
You likely can't join if
- Mental patients;
- Participants in other drug clinical trials within the past 1 month;
- There are no suitable HLA-incompatible donors.
- Other researchers decide that it is not suitable to participate in this researcher.
The study team makes the final eligibility decision.
Where it's taking place
- Guangzhou, Guangdong, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 7 years to 10 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Guangzhou, Guangdong, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.