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

Recruiting

• Conditions: Sickle Cell Disease
• Interventions: Other: OTQ923

• Registration Number: NCT06155500
• Lead Sponsor: Novartis Pharmaceuticals

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.

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 Timeline

• Study First Submitted: 2023-11-24
• Study First Submitted QC: 2023-11-24
• Study First Posted: 2023-12-04
• Study Start: 2024-04-16
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-22
• Last Update Posted: 2024-05-23
• Primary Completion: 2039-01-11
• Study Completion: 2039-01-11

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
OTQ923	OTQ923	Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Enrolled patients on this study will not be administered any study treatment.

Primary Outcome Measures

Name	Time	Method
Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy	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 Outcome Measures

Name	Time	Method
Persistence of fetal hemoglobin expression	Up to 15 years	Assessment of fetal hemoglobin (HbF) expression persistence in peripheral blood will be done by assessing hemoglobin fractionation
WBC chimerism in peripheral blood	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

Trial Locations

Locations (1)

St Jude Childrens Rsrch Hospital; 🇺🇸 Memphis, Tennessee, United States