A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects with Severe Sickle Cell Disease, ßS/ßC Genotype

Phase 1

Active, not recruiting

• Conditions: MedDRA version: 21.0Level: PTClassification code: 10040641Term: Sickle cell anaemia Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; Sickle cell anaemia

• Registration Number: CTIS2023-503247-34-00
• Lead Sponsor: Vertex Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-05-17
• Last Update Posted: 2024-07-22

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: All
• Target Recruitment: 14

Inclusion Criteria

Documented ßS/ßC (HbSC) genotype. Subject can be enrolled based on historical genotype results, but confirmation of genotype is required before start of busulfan conditioning., Subjects with severe SCD. Severe SCD is defined by the occurrence of at least 2 of the following events per year during the 2-year period before screening, while receiving appropriate supportive care: • Acute pain event that requires a visit to a medical facility and administration of pain medications or RBC transfusions • Acute chest syndrome, as indicated by the presence of a new pulmonary infiltrate associated with pneumonia-like symptoms, pain, or fever • Priapism lasting >2 hours and requiring a visit to a medical facility • Splenic sequestration, as defined by an enlarged spleen, left upper quadrant pain, and an acute decrease in Hb concentration of =2 g/dL., Karnofsky performance status of =80% for subjects =16 years of age or Lansky performance status of =80% for subjects <16 years of age., Eligible for autologous stem cell transplant as per investigator’s judgment., Willing to participate in either a long-term follow-up study (VX18-CTX001- 131) or registry study (if available) for a total of 15 years follow-up post-exa-cel infusion.

Exclusion Criteria

A willing and healthy 10/10 Human Leukocyte Antigen (HLA)-matched related donor is available per investigator’s judgement., Intolerance, contraindication, or known sensitivity to plerixafor or busulfan. Subject must not have any risk factors in the opinion of the investigator that would increase the likelihood of busulfan-related toxicities., Pregnancy or breastfeeding, Prior hematopoietic stem cell transplant (HSCT)., Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator., White blood cell (WBC) count <3 × 109 /L or platelet count <50 × 109 /L, not related to hypersplenism per investigator judgment., Subjects with history of alloimmunization to RBC antigens and for whom the investigator anticipates that there will be insufficient RBC units available for the duration of the study., HbF level >15.0%, irrespective of concomitant treatment with HbF-inducing treatments such as HU., History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk to the subject., Any prior or current malignancy or myeloproliferative disorder or a significant immunodeficiency disorder., Advanced liver disease (as defined in the protocol)

Study & Design

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