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

Last updated: September 29, 2025
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Not Recruiting

Phase

1

Condition

Red Blood Cell Disorders

Sickle Cell Disease

Treatment

OTQ923

OTQ923

Clinical Study ID

NCT06155500
CADPT03A12001
  • Ages 18-100
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Patients must have received gene therapy treatment from the parent treatmentprotocol (CADPT03A12101).

  2. Patients must provide informed consent prior to their entry into this study.

Exclusion

Exclusion Criteria:

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

Study Design

Total Participants: 4
Treatment Group(s): 2
Primary Treatment: OTQ923
Phase: 1
Study Start date:
April 16, 2024
Estimated Completion Date:
January 11, 2039

Study 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.

Connect with a study center

  • University of Chicago

    Chicago 4887398, Illinois 4896861 60637
    United States

    Site Not Available

  • Memorial Sloan Kettering Cancer Ctr

    New York 5128581, New York 5128638 10065
    United States

    Site Not Available

  • St Jude Childrens Rsrch Hospital

    Memphis, Tennessee 38105-3678
    United States

    Site Not Available

  • St Jude Childrens Research Hospital

    Memphis 4641239, Tennessee 4662168 38105
    United States

    Site Not Available

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