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

NCT07424157 | Not Yet Recruiting

• 1 other identifier: LTFU-01
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 1

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.

Conditions

CAR T Cell; CAR T Cell Therapy

Outcome Measures

Primary Outcomes (3)

• 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

Interventions

Long term follow up in all patients who received an SCTx gene therapy product OTHER

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

Sponsors & Collaborators

• Seattle Children's Hospital: lead

Study Sites (1)

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Leftover samples of your blood, bone marrow, spinal fluid or other tissue obtained as part of the LTFU study will be retained as part of the SCTx Biobank for use in future research.

Central Study Contacts

Adam Lamble

CONTACT

206-987-2106; CBDCIntake@seattlechildrens.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: SCTx Medical Director

Study Record Dates

• First Submitted: February 13, 2026
• First Posted: February 20, 2026
• Study Start: March 31, 2026
• Primary Completion (Estimated): March 31, 2041
• Study Completion (Estimated): March 31, 2041
• Last Updated: February 20, 2026

Record last verified: 2026-02

Locations

US (1)