NCT04208529

Brief 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).

Trial Health

82
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
160

participants targeted

Target at P25-P50 for phase_3

Timeline
164mo left

Started Jan 2021

Longer than P75 for phase_3

Geographic Reach
6 countries

20 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress28%
Jan 2021Sep 2039

First Submitted

Initial submission to the registry

December 20, 2019

Completed
3 days until next milestone

First Posted

Study publicly available on registry

December 23, 2019

Completed
1.1 years until next milestone

Study Start

First participant enrolled

January 20, 2021

Completed
18.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2039

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2039

Last Updated

March 25, 2026

Status Verified

August 1, 2025

Enrollment Period

18.7 years

First QC Date

December 20, 2019

Last Update Submit

March 20, 2026

Conditions

Beta-ThalassemiaThalassemiaSickle Cell DiseaseHematologic DiseasesHemoglobinopathiesGenetic Diseases, InbornSickle Cell Anemia

Outcome Measures

Primary Outcomes (5)

  • 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

Secondary Outcomes (36)

  • TDT and SCD: Total Hemoglobin (Hb) concentration over time

    Up to 15 years post CTX001 infusion

  • TDT and SCD: Fetal Hemoglobin (HbF) concentration over time

    Up to 15 years post CTX001 infusion

  • TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time

    Up to 15 years post CTX001 infusion

  • TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time

    Up to 15 years post CTX001 infusion

  • TDT and SCD: Change in patient-reported outcome (PRO) over time in participants ≥18 years of age assessed using EuroQol quality of life scale (EQ-5D-5L) for participants from study 111 and 121 only

    Up to 5 years post CTX001 infusion

  • +31 more secondary outcomes

Study Arms (1)

CTX001

EXPERIMENTAL

All participants who complete or discontinue one of the multiple parent studies (CTX001-111, CTX001-121, CTX001-141, CTX001-151 and CTX001-161) after CTX001 infusion will be asked to participate in this long-term follow-up study.

Biological: CTX001

Interventions

CTX001BIOLOGICAL

CTX001 infusion.

Also known as: Exagamglogene autotemcel, Exa-cel
CTX001

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology

Chicago, Illinois, 60611, United States

Location

Herbert Irving Pavilion - Hematology

New York, New York, 10032, United States

Location

New York Presbyterian Hospital - Morgan Stanley Children's Hospital

New York, New York, 10032, United States

Location

Levine Children's Hospital - Hematology

Charlotte, North Carolina, 28203, United States

Location

The Children's Hospital of Philadelphia - Hematology

Philadelphia, Pennsylvania, 19104, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

TriStar Medical Group Children's Specialists - Pediatric Oncology

Nashville, Tennessee, 37203, United States

Location

Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital

San Antonio, Texas, 78229, United States

Location

Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology

Brussels, Belgium

Location

Hospital for Sick Children - Hematology

Toronto, Canada

Location

Toronto General Hospital - Hematology

Toronto, Canada

Location

St. Paul's Hospital - Hematology

Vancouver, Canada

Location

University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology

Düsseldorf, Germany

Location

Center for Pediatric Clinical Studies (CPCS)

Klinik Für Kinder- Und Jugendmedizin, Germany

Location

Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine

Regensburg, Germany

Location

IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica

Rome, Italy

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

Hammersmith Hospital - Haematology Dept

London, United Kingdom

Location

University College London Hospital NHS Foundation - Main

London, United Kingdom

Location

Related Publications (2)

  • Fuente J, Frangoul H, Lang P, Wall D, Meisel R, Corbacioglu S, Li AM, Shah AJ, Carpenter B, Kwiatkowski JL, Mapara MY, Liem RI, Rupprecht J, Kuo KHM, Merkeley H, Algeri M, Smith W, Kohli P, Li N, Rubin J, Zhang S, Hobbs W, Locatelli F. Improvements in health-related quality of life in patients with transfusion-dependent beta-thalassemia after exagamglogene autotemcel. Blood Adv. 2025 Dec 23;9(24):6502-6510. doi: 10.1182/bloodadvances.2025016702.

    PMID: 40862696DERIVED

  • Sharma A, Locatelli F, Bhatia M, Molinari L, Mapara MY, Liem RI, Dedeken L, Wall D, Eckrich MJ, Kuo KHM, Smith W, Imren S, Kohli P, Li N, Liu T, Rubin J, Hobbs W, Grupp SA, Frangoul H. Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel. Blood Adv. 2025 Dec 23;9(24):6481-6490. doi: 10.1182/bloodadvances.2025016701.

    PMID: 40857358DERIVED

MeSH Terms

Conditions

beta-ThalassemiaThalassemiaAnemia, Sickle CellHematologic DiseasesHemoglobinopathiesGenetic Diseases, Inborn

Interventions

exagamglogene autotemcel

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHemic and Lymphatic DiseasesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Model Details: Long Term Safety Follow-up
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 20, 2019

First Posted

December 23, 2019

Study Start

January 20, 2021

Primary Completion (Estimated)

September 30, 2039

Study Completion (Estimated)

September 30, 2039

Last Updated

March 25, 2026

Record last verified: 2025-08

Data Sharing

IPD Sharing
Will not share

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/

Locations

BE(1)DE(3)CA(3)IT(1)US(9)GB(3)