Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
About this study
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
- Condition
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
- Tested
- CTX001
- Sponsor
- Vertex Pharmaceuticals Incorporated
Who can join
- Age
- 12 to 35 years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
Inclusion 13
- Minimum age: 12 years
- Maximum age: 35 years
- Study condition: Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
- Participants with TDT and SCD:
- Eligible for autologous stem cell transplant as per investigator's judgment.
- Participants with TDT:
- Diagnosis of TDT as defined by:
- Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning
- History of at least 100 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
- Participants with SCD:
- 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
Exclusion 10
- Participants with TDT and SCD:
- A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement
- Prior hematopoietic stem cell transplant (HSCT)
- Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
- Participants with TDT:
- Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications
- Participants with sickle cell β-thalassemia variant
- Participants with SCD:
- History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening
- Other protocol defined Inclusion/Exclusion criteria may apply.
Where
6 sites, 6 recruiting
University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
Al Mathar Ash Shamali, Saudi Arabia
Levine Children's Hospital - Hematology
Charlotte, North Carolina, United States
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, United States
New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, United States
Contact
-
Medical Information
6173416777 medicalinfo@vrtx.com
Potential match only. Final eligibility is determined by the study team.