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

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)

Facility	City	Region
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

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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