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Clinical Trials in the UK / NCT05329649
Active, not recruiting Phase 3

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

NCT05329649 · tracked via the Priya Life Science UK tracker
Sponsor
Vertex Pharmaceuticals Incorporated
Phase
Phase 3
Started
2022-05-02
Last updated
2026-07-01

Condition(s) studied

Sickle Cell DiseaseHydroxyurea FailureHydroxyurea IntoleranceHemoglobinopathiesHematological Diseases

Investigational drug(s) / intervention(s)

CTX001 →

CTX001: Administered by intravenous infusion following myeloablative conditioning with busulfan.

Study summary

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Eligibility

Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
Key Inclusion Criteria: * 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 * Hydroxyurea (HU) failure unless HU intolerant * Eligible for autologous stem cell transplant as per investigators judgment Key Exclusion Criteria: * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor * Prior hematopoietic stem cell transplant (HSCT). * Clinically significant and active bacterial, viral, fungal, or parasitic infection Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcome measure(s)

  • Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12) — Up to 24 Months After CTX001 Infusion

Trial sites (7)

FacilityCityRegionStatus
Levine Children's Hospital - Hematology Charlotte North Carolina
The Children's Hospital of Philadelphia - Hematology Philadelphia Pennsylvania
St. Jude Children's Research Hospital Memphis Tennessee
TriStar Medical Group Children's Specialists - Pediatric Oncology Nashville Tennessee
University Hospital Duesseldorf - 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
St.Mary's Hospital - Haematology Dept London United Kingdom

More Vertex Pharmaceuticals Incorporated trials in the UK

Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropathy Recruiting
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) Active, not recruiting
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) Active, not recruiting
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting
A Long-term Follow-up Study in Participants Who Received CTX001 Enrolling by invitation

Other trials for the same condition

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS) Active, not recruiting
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) Active, not recruiting
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease Recruiting
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD) Recruiting
A Long-term Follow-up Study in Participants Who Received CTX001 Enrolling by invitation
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05329649 on ClinicalTrials.gov ↗ ← All trials in the UK