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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
EU CTIS ID: 2023-503247-34-00
What this study is testing
Evaluate efficacy of a single dose of exa-cel in adolescent and adult subjects with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
You likely can't join if
- 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.
See the full eligibility criteria
Who can join
- 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.
What rules you out
- 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)
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.