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

Novartis

Status and phase: Active, not recruiting; Phase 1

Conditions: Sickle Cell Disease

Treatments: Biological: OTQ923

Study type: Interventional

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: 4 patients

Sex: All

Ages: 18 to 100 years old

Volunteers: No Healthy Volunteers

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).

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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4 participants in 1 patient group

OTQ923

Experimental group

Description:

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

Treatment:

Biological: OTQ923