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Gene Therapy Trial Report

Summary

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease


NCTID NCT05477563 (View at clinicaltrials.gov)
Description
Development Status Approved
Indication Beta-Thalassemia, Sickle Cell Disease
Disease Ontology Term DOID:0081445; DOID:0080771
Compound Name CASGEVY
Compound Alias exagamglogene autotemcel, CTX001
Sponsor Vertex Pharmaceuticals Incorporated
Funder Type Industry
Recruitment Status
Enrollment Count 26 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant BCL11A
Therapy Type Gene editing
Therapy Route Ex-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Electroporation
Vector Type RNP
Editor Type Cas9 mRNA
Dose 1 Minimum dose: 3E6 CD34+ cells/kg
Dose 2 Maximum dose: 20E6 CD34+ cells/kg

Study Record Dates


Current Stage Phase3
Submit Date 2022-07-26
Completion Date 2027-06-09
Last Update 🔄 2026-03-23

Participation Criteria


Eligible Age 12 Years - 35 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key Inclusion Criteria: * Participants with TDT and SCD: * Eligible for autologous stem cell transplant as per investigator's judgment. * Participants with TDT: * Diagnosis of TDT as defined by: * Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning * History of at least 100 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening * Participants with SCD: * Diagnosis of severe SCD as defined by: * Documented SCD genotypes * History of at least two severe VOCs events per year for the previous two years prior to enrollment Key Exclusion Criteria: * Participants with TDT and SCD: * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement * Prior hematopoietic stem cell transplant (HSCT) * Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator * Participants with TDT: * Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications * Participants with sickle cell β-thalassemia variant * Participants with SCD: * History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening Other protocol defined Inclusion/Exclusion criteria may apply.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 6
Locations Saudi Arabia,United States,Italy,Germany

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates FDA approved 12/8/23, price/treatment $2.2M, expanded indication to beta thalassemia 1/16/24

Resources/Links