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

Running, not enrolling · Phase 1

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT06155500

Run by

Novartis Pharmaceuticals

People needed

4

Starts

2024-04-16

Expected to finish

2039-01-11

Last updated by the study team

2026-07-28

Who can join

Age: 18 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

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).

Where it is running

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

Full record on ClinicalTrials.gov

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