Actively Recruiting
A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
Led by First Affiliated Hospital of Guangxi Medical University · Updated on 2023-05-09
5
Participants Needed
1
Research Sites
401 weeks
Total Duration
On this page
Sponsors
F
First Affiliated Hospital of Guangxi Medical University
Lead Sponsor
G
Genmedicn Biopharma Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced with LentiRed Lentivrial vector.
CONDITIONS
Official Title
A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subject or legal guardian must understand the study and provide written consent
- Age between 5 and 35 years, any gender
- Clinical diagnosis of transfusion-dependent beta-thalassemia with specific genotypes and severe anemia requiring regular transfusions
- Karnofsky or Lansky Performance Score of 70 or higher
- Determined suitable for autologous hematopoietic stem cell transplantation by investigator
- Treated and followed for at least 2 years at a specialized center with detailed transfusion records
You will not qualify if you...
- Positive tests for hepatitis B, hepatitis C, HIV, syphilis, or tuberculosis
- White blood cell count below 3 x 10^9/L or platelet count below 100 x 10^9/L; previous splenectomy
- Unresolved bleeding disorders
- History or current diagnosis of cancer, myeloproliferative, or immune deficiency diseases
- Immediate family history of known familial cancer syndromes
- Previous hematopoietic stem cell transplant
- Advanced liver disease or significant liver abnormalities
- Estimated glomerular filtration rate below 70 mL/min/1.73 m2
- Uncontrolled seizure disorder
- Lung diffusion capacity below 50% predicted
- Cardiac MRI T2* less than 20 ms
- Severe iron overload deemed exclusionary by physician
- Clinically significant pulmonary hypertension
- Participation in another investigational drug study within 30 days
- Failure to obtain informed consent
- Other conditions making subject ineligible for transplant
- Contraindications to conditioning regimen
- Prior genetic stem cell therapy
- Significant psychiatric disorder affecting participation
- Pregnancy, breastfeeding, or inadequate contraception
- Live vaccines within 6 weeks before screening
- Known allergy to trial ingredients
- Investigator's assessment of noncompliance with study procedures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
The affiliated hospital of guangxi medical university
Nanning, Guangxi, China, 530021
Actively Recruiting
Research Team
Y
Yongrong Lai, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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