A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]

An Open Label Study Evaluating the Safety and Efficacy of Gene Therapy for Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a LentiRed Lentiviral Vector (GMCN-508B Drug Product, Also Called LentiRed)

NCT05762510•First Affiliated Hospital of Guangxi Medical University

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Summary

This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced with LentiRed Lentivrial vector.

Detailed Description

Subject participation for this study will be 5 years.

Eligibility

Age

5 - 35 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. The subject himself/herself or one legal guardian/agent of the subject is required to fully understand the study and voluntarily sign a written informed consent.
•2. Ages 5 to 35, no gender limitation.
•3. The clinical diagnosis of TDT includes β0/β0, β+/β0, βE/β0 and β+/β+ genotypes. TDT was defined as severe anemia in patients with thalassemia (Hb persistent \<70 g/L), regular RBC transfusion and standard iron removal therapy to survive for life.
•4. Karnofsky Level of Performance (KPS) score ≥70 in adult subjects and Lansky Level of Performance (LPS) score ≥70 in children subjects.
•5. Subjects were determined to undergo autologous hematopoietic stem cell transplantation by the principle investigator.
•6. Subjects must have been treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.

Exclusion Criteria

•1. Hepatitis B virus (HBV) : HbsAg or HbcAb positive, nucleic acid test positive; Hepatitis C virus (HCV) : HCAb positive, nucleic acid test positive; Positive for Human immunodeficiency virus (HIV) antibody or Treponema pallidum (TP) specific antibody; Tuberculosis: positive interferon gamma release test.
•2. A white blood cell (WBC) count \<3×10\^9/L and/or platelet count \<100×10\^9/L, splenectomy was performed before.
•3. Uncured bleeding abnormalities.
•4. Any previous or current malignancy, myeloproliferative disease, or immune deficiency disease.
•5. Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndromes, hereditary non-polyposis colorectal cancer syndromes and familial adenomatous polyposis).
•6. Previous hematopoietic stem cell transplantation (HSCT).
•7. Advanced liver disease, defined as: 1) Baseline alanine aminotransferase (ALT) or direct bilirubin ≥3 normal upper limit (ULN), or 2) Liver biopsy demonstrating cirrhosis, any evidence of bridging fibrosis, or acute hepatitis.
•8. Baseline estimated glomerular filtration rate (eGFR) \< 70 mL/min /1.73 m2, as determined using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation for ≥18 years of age, and Besides Schwartz Equation calculator \< 18 years of age.
•9. Uncontrolled seizure disorder.
•10. Diffusion capacity of Carbon monoxide dispersion (DLco) \<50% of predicted (corrected for hemoglobin and or alveolar ventilation, as clinically indicated ).
+13 more criteria

Locations (1)

The affiliated hospital of guangxi medical university

Nanning, Guangxi, China

Key Dates

Start Date

February 22, 2023

Primary Completion Date

April 1, 2028

Completion Date

October 31, 2030

Last Updated

May 9, 2023

Enrollment

ESTIMATED participants

Conditions

Transfusion Dependent Beta-Thalassemia

Interventions

GMCN-508B (LentiRed)

GENETIC

Contacts

Yongrong Lai, PhD

CONTACT

86-771-5356746 laiyongrong@hotmail.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: May 9, 2023Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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