Status:
UNKNOWN
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Lead Sponsor:
Shenzhen Geno-Immune Medical Institute
Conditions:
Beta-Thalassemia
Eligibility:
All Genders
4-70 years
Phase:
NA
Brief Summary
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to eval...
Detailed Description
Thalassemia is considered the most common genetic disorder worldwide. Beta-thalassemia is caused by mutations in the beta-globin gene which encodes the beta-globin protein, leading to the ineffective ...
Eligibility Criteria
Inclusion
- Diagnosis of Beta Thalathemia.
- Age: ≥ 4 years.
- Karnofsky: ≥ 80%.
- Left ventricular ejection fraction (LVEF): \> 50%; no obvious heart disease and pulmonary hypertension.
- Pulmonary function is normal; forced expiratory volumein one second (FEV1) and vital capacity greater than 60% and DLCO \> 50%.
- Serum creatinine ≤ 2 × upper limit of normal range.
- MRI showed no super-iron load in the heart and liver, and no severe cirrhosis.
- Normal Coagulation.
- Written, informed consent obtained prior to any study-specific procedures.
Exclusion
- Diagnosis of active malignant disease (other than Bowen disease or cervical cancer); or has family history of cancer.
- Myelopathy, tumor-related cytogenetic changes or other more severe blood diseases.
- Has alcoholism experience within 6 months prior to enrollment.
- History of epilepsy.
- History of bone marrow transplantation.
- Existence of an available HLA-identical related donor.
- Pregnant or lactating females.
- Subject infected with HCV (HCV antibody positive), HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive.
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
Key Trial Info
Start Date :
December 1 2017
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2020
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT03351829
Start Date
December 1 2017
End Date
December 31 2020
Last Update
November 30 2017
Active Locations (1)
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1
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China, 518000