Long-term follow-up of patients treated with gene-edited stem cells for sickle cell disease
Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
This study is tracking patients with sickle cell disease who received a gene therapy to see how well it works and if it’s safe over 15 years.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 5 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Novartis Industry-sponsored |
| Locations | 1 site (Memphis, Tennessee) |
| Trial ID | NCT06155500 on ClinicalTrials.gov |
What this trial studies
This observational study aims to monitor patients who have received OTQ923, a gene therapy product involving genome-edited autologous hematopoietic stem and progenitor cells, for a duration of 15 years. The focus is on assessing the long-term safety and efficacy of the treatment, particularly its ability to induce fetal hemoglobin production in patients with sickle cell disease. The study involves multiple centers and aims to gather comprehensive data on patient outcomes over an extended period.
Who should consider this trial
Good fit: Ideal candidates for this study are patients who have previously undergone gene therapy and are willing to provide informed consent.
Not a fit: Patients who have not completed at least one year of safety follow-up in the prior treatment protocol will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term effects and benefits of gene therapy for patients with sickle cell disease.
How similar studies have performed: Other studies involving gene therapy for sickle cell disease have shown promising results, indicating that this approach is gaining traction in the field.
Eligibility criteria
Show full inclusion / exclusion 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)
Where this trial is running
Memphis, Tennessee
- St Jude Childrens Rsrch Hospital — Memphis, Tennessee, United States (Recruiting)
Study contacts
- Study coordinator: Novartis Pharmaceuticals
- Email: novartis.email@novartis.com
- Phone: 1-888-669-6682
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.