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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03351829
Recruitment Status : Unknown
Verified November 2017 by Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute.
Recruitment status was:  Not yet recruiting
First Posted : November 24, 2017
Last Update Posted : November 30, 2017
Information provided by (Responsible Party):
Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute

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 evaluate the safety and efficacy of the gene transfer clinical protocol.

Condition or disease Intervention/treatment Phase
Beta-Thalassemia Genetic: Gene-modified autologous stem cells Not Applicable

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 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Estimated Study Start Date : December 1, 2017
Estimated Primary Completion Date : January 1, 2019
Estimated Study Completion Date : December 31, 2020

Arm Intervention/treatment
Experimental: Gene-modified autologous stem cells
Autologous stem cells transduced with lentiviral vector carrying the related gene ex vivo
Genetic: Gene-modified autologous stem cells
1 infusion for 5x10^6~1x10^7 gene-modified cells; or more infusions depending on the circumstances

Primary Outcome Measures :
  1. Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events [ Time Frame: 6 months ]
    Physiological parameter (measuring cytokine response, fever, symptoms)

Secondary Outcome Measures :
  1. Treatment responses [ Time Frame: 1 year ]
    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.

  2. Quality of life [ Time Frame: 1 year ]
    Quality of life will be measured using the Functional Assessment of Cancer Therapy-General (FACT-G) before and after treatment.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   4 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03351829

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Contact: Lung-Ji Chang, PhD 86-075586725195

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China, Guangdong
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China, 518000
Sponsors and Collaborators
Shenzhen Geno-Immune Medical Institute
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Principal Investigator: Lung-Ji Chang, PhD Shenzhen Geno-Immune Medical Institute
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Responsible Party: Lung-Ji Chang, President, Shenzhen Geno-Immune Medical Institute Identifier: NCT03351829    
Other Study ID Numbers: GIMI-IRB-17008
First Posted: November 24, 2017    Key Record Dates
Last Update Posted: November 30, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute:
Beta Thalassemia
Lentiviral vector
Additional relevant MeSH terms:
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Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn