Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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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 Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| OTQ923 Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Enrolled patients on this study will not be administered any study treatment. |
Other: OTQ923
There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907)
|
Outcome Measures
Primary Outcome Measures
- 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
- 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
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Accepted invitation to join based on prior treatment with gene therapy.
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Patients must provide informed consent prior to their entry into this study.
Exclusion Criteria:
- Completion of less than 1 year of safety follow-up in the treatment protocol (CADPT03A12101)
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Novartis Pharmaceuticals
Investigators
- Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CADPT03A12001