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

NCT03351829 | Unknown

• 1 other identifier: GIMI-IRB-17008
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Beta-Thalassemia

Keywords

Beta Thalassemia; Lentiviral vector; Gene

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Treatment responses

  1 year

• Quality of life

  1 year

Study Arms (1)

Gene-modified autologous stem cells

EXPERIMENTAL

Autologous stem cells transduced with lentiviral vector carrying the related gene ex vivo

Genetic: Gene-modified autologous stem cells

Interventions

Gene-modified autologous stem cells GENETIC

1 infusion for 5x10\^6\~1x10\^7 gene-modified cells; or more infusions depending on the circumstances

Gene-modified autologous stem cells

Eligibility Criteria

• Age: 4 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shenzhen Geno-Immune Medical Institute: lead

Study Sites (1)

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, 518000, China

Location

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Lung-Ji Chang, PhD

  Shenzhen Geno-Immune Medical Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lung-Ji Chang, PhD

CONTACT

86-075586725195; c@szgimi.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: President

Study Record Dates

• First Submitted: November 20, 2017
• First Posted: November 24, 2017
• Study Start: December 1, 2017
• Primary Completion: January 1, 2019
• Study Completion: December 31, 2020
• Last Updated: November 30, 2017

Record last verified: 2017-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)