A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Transfusion-Dependent ß-Thalassemia or Severe Sickle Cell Disease

Phase 1

Recruiting

• Conditions: Transfusion-dependent ß-thalassemia (TDT), Severe sickle cell disease (SCD); MedDRA version: 21.0Level: PTClassification code: 10040641Term: Sickle cell anaemia Class: 100000004850; MedDRA version: 20.1Level: LLTClassification code: 10054660Term: Thalassemia beta Class: 10010331; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2024-514641-12-00
• Lead Sponsor: Vertex Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-06-27
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 22

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 ß-thalassemia or compound heterozygous ß-thalassemia including ß-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

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 a-thalassemia and >1 alpha deletion, or alpha multiplications Participants with sickle cell ß-thalassemia variant, Participants with SCD: History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified