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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT03351829
Other study ID # GIMI-IRB-17008
Secondary ID
Status Not yet recruiting
Phase N/A
First received November 20, 2017
Last updated November 28, 2017
Start date December 1, 2017
Est. completion date December 31, 2020

Study information

Verified date November 2017
Source Shenzhen Geno-Immune Medical Institute
Contact Lung-Ji Chang, PhD
Phone 86-075586725195
Email c@szgimi.org
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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 erythropoiesis, hemolysis and anemia. Currently, the only cure for thalassemia is bone marrow transplantation from a related, compatible donor, which has, however, the significant risk of transplant related mortality, graft versus host disease and limited source. Therefore, gene transfer, achieved by transplantation of the patient's own stem cells that have been genetically-modified with the corrected gene, could potentially cure thalassemia.

This study will use an experimental gene transfer procedure performed in a laboratory to insert the related gene into the participant's autologous stem cells using a self-inactivating lentiviral vector. The purpose of this study is to evaluate the safety and effectiveness of the gene transfer procedure and to determine the ability of the gene-corrected cells at generating new, healthy blood cells in individuals.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 20
Est. completion date December 31, 2020
Est. primary completion date January 1, 2019
Accepts healthy volunteers No
Gender All
Age group 4 Years to 70 Years
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 HCV (HCV antibody positive), HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV 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.

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
Gene-modified autologous stem cells
1 infusion for 5x10^6~1x10^7 gene-modified cells; or more infusions depending on the circumstances

Locations

Country Name City State
China Shenzhen Geno-immune Medical Institute Shenzhen Guangdong

Sponsors (1)

Lead Sponsor Collaborator
Shenzhen Geno-Immune Medical Institute

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events Physiological parameter (measuring cytokine response, fever, symptoms) 6 months
Secondary Treatment responses Blood routine indexes will be got before and after treatment. Objective response, such as complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) will be assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria. 1 year
Secondary Quality of life Quality of life will be measured using the Functional Assessment of Cancer Therapy-General (FACT-G) before and after treatment. 1 year
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