Sickle Cell Disease
Conditions
Keywords
Gene therapy, genome-edited hematopoietic stem and progenitor cellular therapy, sickle cell, autologous transplant, BCL11A
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.
Interventions
BIOLOGICALOTQ923
There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907)
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 100 Years
Healthy volunteers
No
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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
United States
Contacts
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed