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Gene Therapy Trial Report

Summary

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia


NCTID NCT03207009 (View at clinicaltrials.gov)
Description
Development Status Approved
Indication Beta-Thalassemia Major
Disease Ontology Term DOID:0080771
Compound Name ZYNTEGLO
Compound Alias betibeglogene autotemcel, LentiGlobin BB305
Compound Description autologous CD34+ cells encoding βA-T87Q-globin gene
Sponsor Genetix Biotherapeutics Inc.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 19 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant HBB
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type BB305 LV
Editor Type none
Dose 1 Minimum dose: 5.0E6 CD34+ cells/kg

Study Record Dates


Current Stage Phase3
Submit Date 2017-06-29
Completion Date 2022-11-15
Last Update 2024-03-07

Participation Criteria


Eligible Age 0 Years - 50 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: • Participants less than or equal to (\<=) 50 years of age at the time of consent or assent (as applicable), and able to provide written consent (adults, or legal guardians, as applicable) or assent (adolescents or children). Provided that the data monitoring committee (DMC) has approved enrolling participants younger than 5 years of age, participants younger than 5 years of age may be enrolled if they weigh a minimum of 6 kilograms (kg) and are reasonably anticipated to be able to provide at least the minimum number of cells required to initiate the manufacturing process. * Diagnosis of TDT with a history of at least 100 milliliter per kilogram per year (mL/kg/year) of pRBCs in the 2 years preceding enrollment (all participants), or be managed under standard thalassemia guidelines with \>= 8 transfusions of pRBCs per year in the 2 years preceding enrollment (participants \>=12 years). * Clinically stable and eligible to undergo HSCT. * Treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history. Exclusion Criteria: * Presence of a mutation characterized as other then β0 (e.g., β+, βE, βC) on at least one β-globin gene (HBB) allele. * Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 and HIV-2), hepatitis B virus (HBV), or hepatitis C (HCV). * A white blood cell (WBC) count less than (\<) 3×10\^9/liter (L), and/or platelet count \<100×10\^9/L not related to hypersplenism. * Uncorrected bleeding disorder. * Any prior or current malignancy. * Prior HSCT. * Advanced liver disease. * A cardiac T2\* \<10 ms by MRI. * Any other evidence of severe iron overload that, in the investigator's opinion, warrants exclusion. * Participation in another clinical study with an investigational drug within 30 days of Screening. * Any other condition that would render the participant ineligible for HSCT, as determined by the attending transplant physician or investigator. * Prior receipt of gene therapy. * Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile participant. * A known and available human leukocyte antigen (HLA) matched family donor. * Any contraindications to the use of granulocyte colony stimulating factor (G-CSF) and plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 9
Locations Greece,United States,Italy,United Kingdom,France,Germany

Regulatory Information


Has US IND True
FDA Designations Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates FDA approval granted 8/17/22, $2.8M estimated cost/treatment

Resources/Links