Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Apr 7, 2022

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment called CTX001 for children with severe sickle cell disease (SCD), especially for those who have not responded well to or cannot tolerate the standard treatment, hydroxyurea. Sickle cell disease is a genetic condition that affects the shape and function of red blood cells, leading to painful episodes and other serious health issues. The trial will involve giving participants a one-time treatment using their own modified stem cells, which are the cells in the body that can develop into different types of blood cells. The main goals of the study are to see if this new treatment is safe and effective in reducing the symptoms of sickle cell disease.

To be eligible for the trial, children must be diagnosed with severe sickle cell disease and have experienced at least two painful crises each year for the past two years. They also need to have not benefited from hydroxyurea treatment or cannot take it due to side effects. It's important to note that children will not be able to participate if they have an available healthy sibling who is a perfect match for a stem cell transplant or if they have had a stem cell transplant before. During the trial, participants will be closely monitored to ensure their safety and to evaluate the effects of the treatment. Overall, this study aims to offer hope for improved health and quality of life for children suffering from this challenging condition.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• * 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
• • Hydroxyurea (HU) failure unless HU intolerant
• • Eligible for autologous stem cell transplant as per investigators judgment
• Key Exclusion Criteria:
• • A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor
• • Prior hematopoietic stem cell transplant (HSCT).
• • Clinically significant and active bacterial, viral, fungal, or parasitic infection
• • Other protocol defined Inclusion/Exclusion criteria may apply.