Last Update: May 23, 2024
Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
ClinicalTrials.gov Identifier:
NCT06155500
Novartis Reference Number:CADPT03A12001
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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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. 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.

Sickle Cell Disease
Recruiting
5
Apr 16, 2024
All
18 Years - (Adult, Older Adult)

Interventions

Other

OTQ923

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

Eligibility Criteria

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)

Worldwide Contacts

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Novartis Pharmaceuticals

1-888-669-6682