TRX103-LTFU

Overview

About this study

The US Food and Drug Administration (FDA)/Center for Biologics Evaluation and Research (CBER) recommends an up to 15-year safety follow-up period for gene therapy medicinal products that utilize integrating vectors (e.g., lentiviral vectors). Because gene therapy products may be associated with delayed adverse events (AEs), CBER advises that subjects be monitored for an extended period of time. CBER also recognizes that assessment of risk is a continuous process, and the nature and duration of follow-up observations may be revised accordingly (FDA, CBER 2020).

This study will assess the long-term safety of TRX103 in subjects who received any amount of TRX103 in a clinical study sponsored by Tr1X, Inc. Subjects will be monitored for safety events, such as new incidence of malignancies, hematologic disorders, and infections, new incidence or exacerbation of pre-existing autoimmune diseases, related AEs and serious adverse events (SAEs), and if applicable, positivity for replication competent lentivirus (RCL) for up to 15 years after administration of TRX103.

Collection of such long-term effects will help to further define the risk-benefit profile of TRX103.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Subjects are eligible to be included in the study only if all of the following criteria apply:

1.

Subjects must have received TRX103 in a clinical study sponsored by Tr1X, Inc. and have either completed the study or have discontinued early from the study.

2.

Subjects must be able to understand, consent, and be willing and able to complete all specified follow-up activities for this LTFU study.

Subjects are excluded from the study if the following criterion applies:

1.

Withdrawal from a clinical study sponsored by Tr1X, Inc. prior to infusion of TRX103.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Manreet Kaur, M.B.B.S., M.D.

Closed-enrolling by invitation

What is this? (?)
"Close"
Not open to everyone who meets the eligibility criteria, but only those invited to participate by the study team.

Contact information:

David Amerson

4803019309

amerson.david@mayo.edu

More information

Publications

Publications are currently not available