Long-Term Follow-Up of Subjects Treated With Seattle Children's Therapeutics Gene Therapy Products
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Long term follow up in all patients who received an SCTx gene therapy product.
- Who it may be relevant to
- Registry conditions: CAR T Cell, CAR T Cell Therapy. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
Subjects exposed to gene therapy products (GTPs) may be at risk for delayed or long-term adverse events. This is a long-term follow-up (LTFU) protocol designed to evaluate the long-term safety of Seattle Children's Therapeutics (SCTx) GTPs and incorporates in monitoring guidance from the U.S. Food and Drug Administration (FDA). Subjects will be followed for up to 15 years starting from the date of the most recent infusion of an SCTx GTP. Subjects planning to receive, or who have received, at least one infusion of an SCTx GTP will be offered participation in this LTFU study. Subjects enrolled in this study will have safety assessments and laboratory evaluations performed at scheduled intervals for each unique SCTx GTP received. No treatment is administered in this LTFU study.
Interventions
- Other Long term follow up in all patients who received an SCTx gene therapy product
Participants will be followed for 15 years following the receipt of their final infusion on a separate SCTx gene therapy protocol to monitor for the long term effects of CAR T cell therapy
Primary outcome measures
- Incidence of delayed adverse events [Time frame: 15 years post-final infusion of SCTx GTP]
- Long-term persistence of SCTx Gene Therapy Products [Time frame: 15 years post-final infusion]
- Long-term clinical outcomes following treatment with SCTx GTP [Time frame: 15 years post-final infusion]
Eligibility criteria
Inclusion criteria
- Enrolled in an SCTx GTP clinical trial;
- Received or planned to receive an SCTx GTP;
- Subject and/or legally authorized representative have provided consent/assent for study participation.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- Seattle Children's Hospital — Seattle
Identifiers
NCT: NCT07424157 · LTFU-01