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

NCT06155500 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: CADPT03A12001
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Sickle Cell Disease

Keywords

Gene therapy; genome-edited hematopoietic stem and progenitor cellular therapy; sickle cell; autologous transplant; BCL11A

Outcome Measures

Primary Outcomes (1)

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

  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 Outcomes (2)

• Persistence of fetal hemoglobin expression

  Up to 15 years

• WBC chimerism in peripheral blood

  5 years

Study Arms (1)

OTQ923

EXPERIMENTAL

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

Interventions

OTQ923 BIOLOGICAL

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

OTQ923

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (3)

University of Chicago

Chicago, Illinois, 60637, United States

Location

Memorial Sloan Kettering Cancer Ctr

New York, New York, 10065, United States

Location

St Jude Childrens Research Hospital

Memphis, Tennessee, 38105, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2023
• First Posted: December 4, 2023
• Study Start: April 16, 2024
• Primary Completion (Estimated): January 11, 2039
• Study Completion (Estimated): January 11, 2039
• Last Updated: October 3, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)