Clinical Trials in China / NCT03351829
Starting soon
Not applicable
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
NCT03351829 · tracked via the Priya Life Science China tracker
Condition(s) studied
Beta-Thalassemia
Investigational drug(s) / intervention(s)
Gene-modified autologous hematopoeitic stem cells
Gene-modified autologous hematopoeitic stem cells: 1 infusion of 5x10\^6\~1x10\^7 per kilogram body weight gene-modified cells; or more infusions depending on the circumstances
Study 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 evaluate the safety and efficacy of the gene transfer clinical protocol.
Eligibility
Inclusion Criteria:
1. Diagnosis of Beta Thalathemia.
2. Age: ≥ 4 years.
3. Karnofsky: ≥ 80%.
4. Left ventricular ejection fraction (LVEF): \> 50%; no obvious heart disease and pulmonary hypertension.
5. Pulmonary function is normal; forced expiratory volumein one second (FEV1) and vital capacity greater than 60% and DLCO \> 50%.
6. Serum creatinine ≤ 2 × upper limit of normal range.
7. MRI showed no super-iron load in the heart and liver, and no severe cirrhosis.
8. Normal Coagulation.
9. Written, informed consent obtained prior to any study-specific procedures.
Exclusion Criteria:
1. Diagnosis of active malignant disease (other than Bowen disease or cervical cancer); or has family history of cancer.
2. Myelopathy, tumor-related cytogenetic changes or other more severe blood diseases.
3. Has alcoholism experience within 6 months prior to enrollment.
4. History of epilepsy.
5. History of bone marrow transplantation.
6. Existence of an available HLA-identical related donor.
7. Pregnant or lactating females.
8. Subject infected with HIV (HIV antibody positive), Treponema pallidum antibody positive or TB culture positive.
9. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
Primary outcome measure(s)
- Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events — 6 months
Physiological parameter (measuring cytokine response, fever, symptoms) - Tolerability of transplanted cells that are transduced ex vivo & transplanted in subjects with ß-thalassemia major conditioned with a reduced-intensity non-myeloablative preparative regimen. — 1 year
Monitoring the following: The occurrence of insertional oncogenesis, which will be investigated by monitoring peripheral blood cell counts \& leukocyte clonality using PCR and sequencing analysis, and qPCR for vector copy number.
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| Shenzhen Geno-immune Medical Institute | Shenzhen | Guangdong |
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Other trials for the same condition
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
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A Long-term Follow-up Study in Participants Who Received CS-101
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT03351829 on ClinicalTrials.gov ↗ ← All trials in China