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

September 29, 2025 updated by: Novartis Pharmaceuticals
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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Illinois
      • Chicago, Illinois, United States, 60637
        • University of Chicago
    • New York
      • New York, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Ctr
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • St Jude Childrens Research Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Study Population

Target population includes all patients who have received OTQ923 within the treatment protocol (CADPT03A12101) and have been monitored for at least 1 year post-infusion.

Description

Inclusion Criteria:

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: OTQ923
Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Patients enrolled on this LTFU study will not be administered any study treatment.
Biological: OTQ923
There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Up to 15 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Up to 15 years
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
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 16, 2024

Primary Completion (Estimated)

January 11, 2039

Study Completion (Estimated)

January 11, 2039

Study Registration Dates

First Submitted

November 24, 2023

First Submitted That Met QC Criteria

November 24, 2023

First Posted (Actual)

December 4, 2023

Study Record Updates

Last Update Posted (Estimated)

October 3, 2025

Last Update Submitted That Met QC Criteria

September 29, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CADPT03A12001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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