Long-Term Follow-Up of Subjects Treated With Seattle Children's Therapeutics Gene Therapy Products (LTFU-01)

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 Overview

• Status: Not yet recruiting
• Conditions: CAR T Cell; CAR T Cell Therapy
• Intervention / Treatment: Other: Long term follow up in all patients who received an SCTx gene therapy product

• Study Type: Observational
• Enrollment (Estimated): 500

Contacts and Locations

• Study Contact: Name: Adam Lamble; Phone Number: 206-987-2106; Email: CBDCIntake@seattlechildrens.org

Study Locations

• United States
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital
      • Contact: Adam Lamble; Phone Number: 206-987-2106; Email: CBDCIntake@seattlechildrens.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients who have received treatment with a gene therapy product on an SCTx Sponsored clinical trial.

Description

Inclusion Criteria:

1. Enrolled in an SCTx GTP clinical trial;
2. Received or planned to receive an SCTx GTP;
3. Subject and/or legally authorized representative have provided consent/assent for study participation.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of delayed adverse events	The type, frequency, severity, and duration of adverse events considered related to the SCTx gene therapy product (GTP) the participant received.	15 years post-final infusion of SCTx GTP
Long-term persistence of SCTx Gene Therapy Products	Monitor for long-term persistence of SCTx GTPs.	15 years post-final infusion
Long-term clinical outcomes following treatment with SCTx GTP	Death from any cause and first relapse following an initial remission	15 years post-final infusion

Collaborators and Investigators

• Sponsor: Seattle Children's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): March 31, 2026
• Primary Completion (Estimated): March 31, 2041
• Study Completion (Estimated): March 31, 2041

Study Registration Dates

• First Submitted: February 13, 2026
• First Submitted That Met QC Criteria: February 13, 2026
• First Posted (Actual): February 20, 2026

Study Record Updates

• Last Update Posted (Actual): February 20, 2026
• Last Update Submitted That Met QC Criteria: February 13, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Other Study ID Numbers: LTFU-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No