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

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

Enrolling By Invitation
2 years and older
All
Phase 3

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).

Eligibility

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

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Study details
    Beta-Thalassemia
    Thalassemia
    Sickle Cell Disease
    Hematologic Diseases
    Hemoglobinopathies
    Genetic Diseases
    Inborn
    Sickle Cell Anemia

NCT04208529

Vertex Pharmaceuticals Incorporated

18 April 2026

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FAQs

Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

How long does a clinical trial take place?

The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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