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

Novartis

Status: Enrolling

Conditions: Sickle Cell Disease

Treatments: Other: OTQ923

Study type: Observational

Funder types: Industry

Identifiers

NCT06155500

CADPT03A12001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 5 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

5 participants in 1 patient group

OTQ923

Description:

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

Treatment:

Other: OTQ923