Actively Recruiting
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease
Led by Vertex Pharmaceuticals Incorporated · Updated on 2026-03-23
26
Participants Needed
6
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of a single dose of CTX001, an autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cell therapy, in participants aged 12 to 35 years with transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD). This Phase 3b open-label study aims to understand how this gene-edited therapy may impact these serious blood disorders that require frequent transfusions or cause severe complications. Participants will receive one infusion of CTX001 through a central venous catheter after undergoing myeloablative conditioning with busulfan. The study focuses on a single-dose administration of these genetically modified stem cells targeting the BCL11A gene enhancer to potentially improve hemoglobin production. No placebo or comparison group is involved, and the intervention is delivered intravenously. During the study, participants will be closely monitored for up to 12 months after infusion, including assessments of fetal and total hemoglobin levels over time. Safety evaluations will track adverse events, serious adverse events, and transplant-related mortality. Researchers will also measure engraftment success, genetic modification presence in blood and bone marrow, transfusion needs, and rates of vaso-occlusive crises and hospitalizations for SCD. Participants will undergo laboratory tests and clinical evaluations to gather comprehensive data on treatment impact and safety throughout the study period.
CONDITIONS
Brief 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 aged between 12 and 35 years
- Eligible for autologous stem cell transplant as determined by the investigator
- Diagnosed with transfusion-dependent beta-thalassemia (including documented homozygous or compound heterozygous beta-thalassemia) or severe sickle cell disease
- For beta-thalassemia participants: history of at least 100 mL/kg/year or 10 units/year of packed red blood cell transfusions in the prior 2 years
- For sickle cell disease participants: history of at least two severe vaso-occlusive crisis events per year for the previous two years prior to enrollment
You will not qualify if you...
- A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available
- Prior hematopoietic stem cell transplant
- Clinically significant and active bacterial, viral, fungal, or parasitic infection
- For beta-thalassemia participants: associated alpha-thalassemia with more than 1 alpha deletion or alpha multiplications
- For sickle cell disease participants: history of untreated moyamoya syndrome or presence of moyamoya syndrome at screening
- Other protocol-defined exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 12 months
Participants receive a single intravenous infusion of CTX001 following myeloablative conditioning with busulfan.
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
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here