NCT03351829Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Source: ClinicalTrials.gov · Last updated 2 Nov 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Study type
- Interventional
- Enrollment
- 20 (estimated)
- First submitted
- 20 Nov 2017
- Start date
- 31 Dec 2026
- Primary completion date
- 31 Dec 2029
- Lead sponsor
- Shenzhen Geno-Immune Medical Institute
- Diseases
- Thalassaemia
- NCT number
- NCT03351829
What you commit to
- Study type: Interventional
- Planned participants: 20
- From 31 Dec 2026 to the primary completion date, 31 Dec 2029
- 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
- Has alcoholism experience within 6 months prior to enrollment.
Other illness
- 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.
- History of epilepsy.
- History of bone marrow transplantation.
- Subject infected with HIV (HIV antibody positive), Treponema pallidum antibody positive or TB culture positive.
Other
- Existence of an available HLA-identical related donor.
- Pregnant or lactating females.
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
Who the study is looking for
- 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.
Age in the registry: 4 years to 70 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 Geno-immune Medical Institute Coordinator of this site? Confirm its status | Shenzhen | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.