Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT

Thomas Damgaard Sandahl

Status: Invitation-only

Conditions: Wilson Disease

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT07159581

1-10-72-146-24

Details and patient eligibility

About

The primary objective of this study is to investigate the effect of gene therapy (UX704) on copper distribution and excretion in Wilson disease patients. The effect is investigated using 64Cu positron emission tomography scans combined with a CT scan.

Full description

Patients entering this study are included from the ongoing clinical study "A Phase 1/ 2/ 3 Study of UX701 Gene Therapy in Adults With Wilson Disease" (NCT04884815). The patients will have received gene therapy as part of the clinical study. This study is an extension study that aims to specifically monitor the effect of gene therapy on copper distribution and excretion using 64Cu PET/CT.

Patients will receive 40-80 MBq 64CuCl2 intravenously and will undergo three PET/CT scans during the next 20 hours.

Enrollment: 5 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients with Wilson disease who have received gene therapy (UX701) as part of NCT04884815
• Age above 18 years
• Women of childbearing potential must accept to use safe contraception at the time of the PET scans: Spiral or hormonal contraception (oral pills, implants, transdermal patches, vaginal rings, or depot injections)
• At the day of the first PET scan, a negative urine pregnancy test is required for women of childbearing potential
• Before the first PET scan, a signed informed consent must be completed

Exclusion criteria

• Known hypersensitivity to Cu-64, copper in general, or other components of the radiotracer formulation
• Claustrophobia
• Pregnancy, breastfeeding, or plans to become pregnant

Trial design

5 participants in 1 patient group

Wilson disease patients

Description:

UX701