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NCT Number: NCT06155500

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Chicago, Chicago, Illinois, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101).
  • Patients must provide informed consent prior to their entry into this study.

Exclusion criteria

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

Treatment and study plan

OTQ923

Biological

There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907)

Primary outcomes

  1. Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy

    Time frame: Up to 15 years

    Number of participants with delayed adverse events including new secondary malignancies, new incidence or exacerbation of a prior autoimmune disorder, new incidence or exacerbation of a prior rheumatologic disorder, new hematologic disorder, and other adverse events considered to be related to OTQ923 therapy.

Secondary outcomes

  1. Persistence of fetal hemoglobin expression

    Time frame: Up to 15 years

    Assessment of fetal hemoglobin (HbF) expression persistence in peripheral blood will be done by assessing hemoglobin fractionation

  2. WBC chimerism in peripheral blood

    Time frame: 5 years

    Concern for loss of engraftment by monitoring the WBC chimerism annually. Modified white blood cells (WBCs) measured by droplet digital PCR (ddPCR) and NGS will be performed annually through year 5 with subsequent samples stored and run annually in the event of concern for loss of engraftment

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Important dates

Study start
2024
Primary completion
2039
Study completion
2039
First posted
Dec 4, 2023
Registry last updated
Jul 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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