A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects with Severe Sickle Cell Disease, βS/βC Genotype
Key facts
- Where
- Creteil, FranciaországSee the site
- Phase
- 3. fázis
Where in Franciaország
- Assistance Publique Hopitaux De ParisCreteil, FranciaországToborzás még nem indult
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Vertex Pharmaceuticals Inc.
- Trial code
- EU 2023-503247-34-00
- Record updated
- 2025. augusztus 19.
- Original description
Evaluate efficacy of a single dose of exa-cel in adolescent and adult subjects with severe sickle cell disease (SCD), βS/βC genotype (HbSC).Read full original descriptionShow less
Evaluate efficacy of a single dose of exa-cel in adolescent and adult subjects with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
Inclusion: Documented βS/βC (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: Prior hematopoietic stem cell transplant (HSCT); HbF level >15.0%, irrespective of concomitant treatment with HbF-inducing treatments such as HU; Any prior or current malignancy or myeloproliferative disorder or a significant immunodeficiency disorder; Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator; 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; 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; Advanced liver disease (as defined in the protocol); A willing and healthy 10/10 Human Leukocyte Antigen (HLA)-matched related donor is available per investigator’s judgement; White blood cell (WBC) count <3 × 109 /L or platelet count <50 × 109 /L, not related to hypersplenism per investigator judgment; 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.