Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Jul 14, 2023

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment called exa-cel for people with severe sickle cell disease, specifically those with a certain genetic type known as βS/βC genotype (HbSC). The main goal is to see how well this treatment works and how safe it is for adolescents and adults who may need a stem cell transplant. The trial is not yet recruiting participants, but when it does, it will include individuals aged between 18 and 65 who have been diagnosed with this specific type of sickle cell disease.

To be eligible for the trial, participants must have a documented βS/βC genotype and be judged by the study doctors as suitable for a stem cell transplant. However, if a healthy family member is available to be a donor or if the participant has already had a stem cell transplant, they won't be able to join the study. Those who do participate can expect to receive a single dose of the treatment and will be closely monitored for its effects. This research could potentially lead to new options for managing sickle cell disease, so it's an important opportunity for those affected.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • Participants with documented βS/βC (HbSC) genotype
• • Participants must be eligible for autologous stem cell transplant as per investigator's judgment
• Key Exclusion Criteria:
• • A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement
• • Participants with prior hematopoietic stem cell transplant (HSCT)
• • Treatment with regular RBC transfusions that, in the opinion of the investigator, cannot be interrupted after engraftment.
• • Other protocol defined Inclusion/Exclusion criteria may apply.