NCT05745532RecruitingEarly phase 1
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
Source: ClinicalTrials.gov · Last updated 29 Nov 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
- Status (registry)
- Recruiting
- Phase (registry)
- Early phase 1
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 16 Feb 2023
- Start date
- 1 Dec 2020
- Primary completion date
- 30 May 2025
- Lead sponsor
- Shenzhen Hemogen
- Diseases
- Thalassaemia
- NCT number
- NCT05745532
What you commit to
- Study type: Interventional · Early phase 1
- Planned participants: 10
- From 1 Dec 2020 to the primary completion date, 30 May 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Received gene therapy and allogeneic HSCT in the past.
Other illness
- The physical condition does not meet the requirements for hematopoietic stem cell mobilization and transplantation myeloablation;
- Other unsuitable conditions identified by doctors.
Other
- Have an available HLA matched donor.
- Enrolling in another clinical trial.
Who the study is looking for
- 8-16 years old. Subject and/or subject's legal guardian fully understand and voluntarily sign informed consent;
- Clinically diagnosed as transfusion-dependent β-thalassemia major;
- With sufficient RBC infusion, subjects must maintain hemoglobin ≥9g/dL, serum ferritin threshold ≤ 3000 ng/mL and the liver iron overload mild or absent for at least 3 months before mobilization of hematopoietic stem cell;
- Follow the arrangements for treatment and regular medical checks within two years post-transplantation
Age in the registry: 8 years to 16 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Shenzhen Children's Hospital Coordinator of this site? Confirm its status | Shenzhen | China | Recruiting |
Related publications
This page is information, not medical advice. Discuss any trial with your doctor.