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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 (RMAT)
Recent Updates FDA approved 12/8/23, price/treatment $2.2M, expanded indication to beta thalassemia 1/16/24

Resources/Links