Actively Recruiting
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Led by Vertex Pharmaceuticals Incorporated · Updated on 2026-03-23
26
Participants Needed
6
Research Sites
253 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants with transfusion-dependent beta-thalassemia or severe sickle cell disease
- Eligible for autologous stem cell transplant as judged by investigator
- For beta-thalassemia: documented homozygous or compound heterozygous beta-thalassemia including beta-thalassemia/hemoglobin E
- History of at least 100 mL/kg/year or 10 units/year of packed red blood cell transfusions in the past 2 years
- For sickle cell disease: documented severe sickle cell disease genotypes
- History of at least two severe vaso-occlusive crisis events per year for the previous two years
You will not qualify if you...
- Availability of a healthy 10/10 HLA-matched related donor
- Prior hematopoietic stem cell transplant
- Clinically significant active infections (bacterial, viral, fungal, or parasitic)
- For beta-thalassemia: associated alpha-thalassemia with more than 1 alpha deletion or alpha multiplications
- For sickle cell disease: history or presence of untreated moyamoya syndrome
- Other protocol defined inclusion/exclusion criteria may apply
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 6 locations
1
New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, United States, 10032
Actively Recruiting
2
Levine Children's Hospital - Hematology
Charlotte, North Carolina, United States, 28203
Actively Recruiting
3
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, United States, 37203
Actively Recruiting
4
University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Actively Recruiting
5
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
Actively Recruiting
6
King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
Al Mathar Ash Shamali, Saudi Arabia
Actively Recruiting
Research Team
M
Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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