CAT-VHL Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-Hippel Lindau Disease

IRCCS Ospedale San Raffaele

Status and phase

Enrolling

Phase 2

Conditions

VHL - Von Hippel-Lindau Syndrome

Treatments

Drug: investigating the role of CAIX as target in VHL disease and in VHL-/- tumors.

Study type

Interventional

Funder types

Other

Identifiers

NCT07171905

PNRR-POC-2023-12377493

Details and patient eligibility

About

The study is a phase 2, non-comparative and non-randomized, single arm, national clinical trial testing the hypothesis that CAIX-PET has diagnostic and theranostic potential in VHL disease and in VHL-/- tumors. Participants will receive a single dose of the diagnostic radiopharmaceutical [89Zr]Zr-DFO-Girentuximab and subsequently will be subjected to imaging with an hybrid PET/CT scanner. Sensitivity and diagnostic accuracy of experimental imaging will be assessed against standard of truth derived from standard of care procedures such as MRI or pathology following surgery. A theranostic approach will be simulated by replacing the physical decay of the diagnostic isotope [89Zr]Zr with the physical decay of therapeutic isotopes and evaluating tissue dosimetry.

Enrollment

38 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Voluntarily given informed consent
Age ≥18 years old
Performance Status ECOG/WHO score 0-2
For females of reproductive potential, negative pregnancy test and use of highly effective contraception for 30 days following IMP administration
For males of reproductive potential, use of highly effective contraception for 30 days following IMP administration.

And, for the primary cohort:

Diagnosis of VHL disease requiring surveillance following confirmation of pathogenic variant at genetic test

Alternatively, for the secondary cohort:

- Clinical and/or pathological diagnosis of hemangioblastoma, pheochromocytoma, pancreatic neuroendocrine tumor or clear cell renal cell carcinoma requiring surgery.

Exclusion criteria

Performance Status ECOG/WHO score >2
Women who are pregnant or breastfeeding or are planning pregnancy during the study
Men who are planning fatherhood during the study
Exposure to any murine or chimeric antibodies within 5 years prior to the planned IMP administration
Exposure to any experimental diagnostic or therapeutic drug within 30 days from the planned IMP administration
Surgery, biopsy, ablative procedure, radiotherapy or any other local treatment for any primary tumor within 4 weeks prior to the planned IMP administration
Exposure to any systemic agent within 4 weeks prior to the planned IMP administration or in case of continuing adverse effects with grade >1 from such therapy
Current exposure to systemic agents or scheduled therapy in the next 6 months following the planned IMP administration
Serious non-malignant disease (e.g. psychiatric, infectious, autoimmune or metabolic) that may interfere with the objectives of the study or within the safety of compliance of the subjects as judged by the Investigator
Known hypersensitivity to [89Zr]Zr-DFO-Girentuximab or DFO (Desferrioxamine)
Severe chronic kidney disease with glomerular filtration rate ≤ 30 mL/min/1.73m2
Other vulnerable categories than rare disease (e.g, being in detention)