A Long-term Follow-up Study in Participants Who Received CTX001

NCT04208529 · tracked via the Priya Life Science Germany tracker

Sponsor

Vertex Pharmaceuticals Incorporated

Phase

Phase 3

Started

2021-01-20

Last updated

2026-08-11

Condition(s) studied

Beta-ThalassemiaThalassemiaSickle Cell DiseaseHematologic DiseasesHemoglobinopathiesGenetic Diseases, InbornSickle Cell Anemia

Investigational drug(s) / intervention(s)

CTX001 →

CTX001: CTX001 infusion.

Study summary

This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \[SCD\] studies).

Eligibility

Sex

ALL

Min age

2 Years

Max age

—

Healthy volunteers

Inclusion Criteria: * Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form * Participants must have received CTX001 infusion in a parent study Exclusion Criteria: * There are no exclusion criteria

Primary outcome measure(s)

New malignancies — Signing of informed consent up to 15 years post CTX001 infusion
New or worsening hematologic disorders — Signing of informed consent up to 15 years post CTX001 infusion
All-cause mortality — Signing of informed consent up to 15 years post CTX001 infusion
Serious adverse events (SAEs) — Signing of informed consent up to 15 years post CTX001 infusion
CTX001-related adverse events (AEs) — Signing of informed consent up to 15 years post CTX001 infusion

Trial sites (20)

Facility	City	Region
Lucile Packard Children's Hospital	Palo Alto	California
Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology	Chicago	Illinois
Herbert Irving Pavilion - Hematology	New York	New York
New York Presbyterian Hospital - Morgan Stanley Children's Hospital	New York	New York
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
Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital	San Antonio	Texas
Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology	Brussels	Belgium
Hospital for Sick Children - Hematology	Toronto	Canada
Toronto General Hospital - Hematology	Toronto	Canada
St. Paul's Hospital - Hematology	Vancouver	Canada
University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology	Düsseldorf	Germany
Center for Pediatric Clinical Studies (CPCS)	Klinik Für Kinder- Und Jugendmedizin	Germany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine	Regensburg	Germany
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica	Rome	Italy
Great Ormond Street Hospital for Children	London	United Kingdom
Hammersmith Hospital - Haematology Dept	London	United Kingdom
University College London Hospital NHS Foundation - Main	London	United Kingdom

More Vertex Pharmaceuticals Incorporated trials in Germany

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A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes Recruiting

A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes Active, not 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

Other trials for the same condition

The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease Recruiting

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia Recruiting

PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS] Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) Active, not recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials Recruiting

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 NCT04208529 on ClinicalTrials.gov ↗ ← All trials in Germany