Long-Term Follow-Up of Subjects Treated With Seattle Children's Therapeutics Gene Therapy Products
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Seattle Children's Hospital
- Enrollment
- 500
- Locations
- 1
- Primary Endpoint
- Incidence of delayed adverse events
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Incidence of delayed adverse events
Time Frame: 15 years post-final infusion of SCTx GTP
The type, frequency, severity, and duration of adverse events considered related to the SCTx gene therapy product (GTP) the participant received.
Long-term persistence of SCTx Gene Therapy Products
Time Frame: 15 years post-final infusion
Monitor for long-term persistence of SCTx GTPs.
Long-term clinical outcomes following treatment with SCTx GTP
Time Frame: 15 years post-final infusion
Death from any cause and first relapse following an initial remission
Secondary Outcomes
No secondary outcomes reported
Investigators
Colleen Annesley
SCTx Medical Director
Seattle Children's Hospital