Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Novartis Pharmaceuticals
- Enrollment
- 4
- Locations
- 6
- Primary Endpoint
- Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy
Study Overview
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.
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
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 100 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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.
Exclusion Criteria
- •1. Completion of less than 1 year of safety follow-up in the treatment protocol (CADPT03A12101).
Arms & Interventions
OTQ923
Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Patients enrolled on this LTFU study will not be administered any study treatment.
Intervention: OTQ923 (Biological)
Outcomes
Primary Outcomes
Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy
Time Frame: 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 Outcomes
- Persistence of fetal hemoglobin expression(Up to 15 years)
- WBC chimerism in peripheral blood(5 years)