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Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

Non Recruiting
18 years and older
All
Phase N/A
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Overview

CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.

Description

This study is monitoring patients treated with OTQ923, an investigational drug product of ex vivo genome-edited autologous hematopoietic stem and progenitor cells (HSPCs) that induces fetal hemoglobin (HbF) production, for a total of 15 years following infusion to monitor long-term safety and efficacy.

Eligibility

Inclusion Criteria:

  1. Accepted invitation to join based on prior treatment with gene therapy.
  2. Patients must provide informed consent prior to their entry into this study.

Exclusion Criteria:

  1. Completion of less than 1 year of safety follow-up in the treatment protocol (CADPT03A12101)

Study details
    Sickle Cell Disease

NCT06155500

Novartis Pharmaceuticals

21 October 2025

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FAQs

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

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

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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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