A PHASE 3 STUDY TO EVALUATE THE SAFETY AND EFFICACY OF A SINGLE DOSE OF CTX001 IN PEDIATRIC SUBJECTS WITH TRANSFUSION-DEPENDENT ¿-THALASSEMIA

Registry ID

CTX001-141

Source registry

HEALTH_CANADA

Status

Ongoing

Phase

PHASE3

Study type

INTERVENTIONAL

Sponsor

VERTEX PHARMACEUTICALS (CANADA) INCORPORATED

Start date

2024-06-18

Last update

2021-10-28

Conditions

• TRANSFUSION DEPENDENT THALASSEMIA

Related clinical trials

• A MULTICENTER STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF A SINGLE DOSE OF AUTOLOGOUS CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS GENE-EDITED CD34+ HUMAN HEMATOPOIETIC STEM AND PROGENITOR CELLS (EDIT 301) IN TRANSFUSION DEPENDENT BETA THALASSEMIA (TDT)
• A PHASE 1/2 STUDY OF THE SAFETY AND EFFICACY OF A SINGLE DOSE OF AUTOLOGOUS CRISPR-CAS9 MODIFIED CD34+ HUMAN HEMATOPOIETIC STEM AND PROGENITOR CELLS (HHSPCS) IN SUBJECTS WITH TRANSFUSION-DEPENDENT B THALASSEMIA
• MULTICENTER, OPEN-LABEL, DOSE-ESCALATION STUDY OF SP 420 IN SUBJECTS WITH TRANSFUSION-DEPENDENT THALASSEMIA
• THE USE OF THE CALCIUM CHANNEL BLOCKER AMLODIPINE AS AN ADJUVANT TREATMENT TO IRON CHELATION FOR THE PREVENTION OF IRON OVERLOAD CARDIOMYOPATHY IN PATIENTS WITH THALASSEMIA

View on source registry