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Enrolling by invitation NCT04208529

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

Phase III Interventional Beta-Thalassemia Thalassemia Sickle Cell Disease Hematologic Diseases

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: CTX001.
Who it may be relevant to
Registry conditions: Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases. Basic parameters: from 2 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Belgium, Canada, Germany, Italy +1
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

Overview

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).

Interventions

  • Biological CTX001
    CTX001 infusion.

Primary outcome measures

  • New malignancies [Time frame: Signing of informed consent up to 15 years post CTX001 infusion]
  • New or worsening hematologic disorders [Time frame: Signing of informed consent up to 15 years post CTX001 infusion]
  • All-cause mortality [Time frame: Signing of informed consent up to 15 years post CTX001 infusion]
  • Serious adverse events (SAEs) [Time frame: Signing of informed consent up to 15 years post CTX001 infusion]
  • CTX001-related adverse events (AEs) [Time frame: Signing of informed consent up to 15 years post CTX001 infusion]
Secondary outcome measures (12)
  • TDT and SCD: Total Hemoglobin (Hb) concentration over time [Time frame: Up to 15 years post CTX001 infusion]
  • TDT and SCD: Fetal Hemoglobin (HbF) concentration over time [Time frame: Up to 15 years post CTX001 infusion]
  • TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time [Time frame: 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 [Time frame: 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 [Time frame: Up to 5 years post CTX001 infusion]
  • TDT and SCD: Change in PROs over time in participants ≥18 years of age assessed using functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) questionnaire for participants from study 111, 121 and 161 only [Time frame: Up to 5 years post CTX001 infusion]
  • TDT and SCD: Change in PROs over time in participants <18 years assessed using EQ-5D-Youth (EQ-5D-Y) from study 111,121,141 and 151 only [Time frame: Up to 5 years post CTX001 infusion]
  • TDT and SCD: Change in PROs over time in participants <18 years assessed using pediatric quality of life inventory (PedsQL) Core [Time frame: Up to 5 years post CTX001 infusion]
  • TDT: Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12) [Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion]
  • TDT: Proportion of participants achieving transfusion independence for at least 6 consecutive months (TI6) [Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion]
  • TDT: Proportion of participants achieving at least 95%, 90%, 85%, 75%, 50% reduction from baseline in annualized volume of RBC transfusions starting after month 10 after CTX001 infusion for participants who have not achieved TI12 [Time frame: From Month 10 up to 15 years post-CTX001 infusion]
  • TDT: Duration of transfusion free in participants who have achieved TI12 [Time frame: From 60 days after last RBC transfusion up to 15 years post CTX001 infusion]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Other

Study locations

United States · 9 centers
  • Lucile Packard Children's Hospital — Palo Alto
  • Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology — Chicago
  • Herbert Irving Pavilion - Hematology — New York
  • New York Presbyterian Hospital - Morgan Stanley Children's Hospital — New York
  • Levine Children's Hospital - Hematology — Charlotte
  • The Children's Hospital of Philadelphia - Hematology — Philadelphia
  • St. Jude Children's Research Hospital — Memphis
  • TriStar Medical Group Children's Specialists - Pediatric Oncology — Nashville
  • … and 1 more center
Canada · 3 centers
  • Hospital for Sick Children - Hematology — Toronto
  • Toronto General Hospital - Hematology — Toronto
  • St. Paul's Hospital - Hematology — Vancouver
Germany · 3 centers
  • University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinica — Düsseldorf
  • Center for Pediatric Clinical Studies (CPCS) — Klinik Für Kinder- Und Jugendmedizin
  • Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medici — Regensburg
United Kingdom · 3 centers
  • Great Ormond Street Hospital for Children — London
  • Hammersmith Hospital - Haematology Dept — London
  • University College London Hospital NHS Foundation - Main — London
Belgium · 1 center
  • Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology — Brussels
Italy · 1 center
  • IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellula — Rome

Publications

  • Sheth S, Corbacioglu S, de la Fuente J, Algeri M, Rupprecht J, Kuo KHM, Shah AJ, Lang P, Merkeley H, Carpenter B, Mapara MY, Liem RI, Grupp S, Chopra Y, Li AM, Kwiatkowski JL, Kirby-Allen M, Cappellini MD, Kattamis A, Zairis S, Liu T, Hobbs W, Frangoul H, Locatelli F, Meisel R; CLIMB THAL-111 and CLIMB-131 Study Groups. Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis PMID 42252696
  • 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): PMID 40862696
  • 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 40857358

Identifiers

NCT: NCT04208529 · VX18-CTX001-131 · 2024-512654-19-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗