Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
NCT ID: NCT05477563
Last Updated: 2025-11-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
26 participants
INTERVENTIONAL
2022-08-02
2027-06-09
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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CTX001
CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive a single infusion of CTX001 through a central venous catheter.
CTX001
Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan
Interventions
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CTX001
Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
* 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
12 Years
35 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, United States
Levine Children's Hospital - Hematology
Charlotte, North Carolina, United States
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, United States
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
Countries
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Central Contacts
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Other Identifiers
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2024-514641-12-00
Identifier Type: OTHER
Identifier Source: secondary_id
VX21-CTX001-161
Identifier Type: -
Identifier Source: org_study_id