Evaluation of Efficacy and Safety of a Single Dose of CTX... | NCT05477563

Studies
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563

Sponsor

Vertex Pharmaceuticals Incorporated

Status

Recruiting

Last Update Posted

Mar 23, 2026Actual

Enrollment

26Estimated

Phase

Phase 3

Conditions

Beta-Thalassemia
Thalassemia
Hematologic Diseases
Genetic Diseases, Inborn
Hemoglobinopathies
Sickle Cell Disease
Sickle Cell Anemia

Interventions

CTX001

Countries

United States
Germany
Italy
Saudi Arabia

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

ID	Type	Description	Link
2024-514641-12-00	Other Identifier	EU CT number

Status Module

Record Verification Date

Mar 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 2, 2022Actual

Primary Completion Date

Jun 9, 2027Estimated

Completion Date

Jun 9, 2027Estimated

First Submitted Date

Jul 26, 2022

First Submission Date that Met QC Criteria

Jul 26, 2022

First Posted Date

Jul 28, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 18, 2026

Last Update Posted Date

Mar 23, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

26Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
CTX001	Experimental	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.	Biological: CTX001

Interventions

Name	Type	Description	Arm Group Labels	Other Names
CTX001	Biological	Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan	CTX001

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Fetal Hemoglobin (HbF) Concentration Over Time		Up to 12 Months After CTX001 Infusion
Total Hemoglobin (Hb) Concentration Over Time		Up to 12 Months After CTX001 Infusion

Secondary Outcomes

Measure	Description	Time Frame
TDT and SCD: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		From Signing of Informed Consent up to 12 Months After CTX001 Infusion
TDT and SCD: Proportion of Participants With Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count (ANC) >=500 per Microliter [mcgL] on 3 Different Days)

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Key Inclusion Criteria:

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

Key Exclusion Criteria:

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

12 Years

Maximum Age

35 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Name	Role	Phone	Extension	Email
Medical Information	Contact	6173416777		medicalinfo@vrtx.com

Facility	Status	City	State	ZIP	Country	Contacts
New York Presbyterian Hospital - Morgan Stanley Children's Hospital	Recruiting	New York	New York	10032	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

ID	Term
D017086	beta-Thalassemia
D013789	Thalassemia
D006402	Hematologic Diseases
D030342	Genetic Diseases, Inborn
D006453	Hemoglobinopathies
D000755	Anemia, Sickle Cell

ID	Term
D000745	Anemia, Hemolytic, Congenital
D000743	Anemia, Hemolytic
D000740	Anemia
D006425	Hemic and Lymphatic Diseases
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities