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

NCT07171905 | Recruiting Phase 2 DMC

• 1 other identifier: PNRR-POC-2023-12377493
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

VHL - Von Hippel-Lindau Syndrome

Outcome Measures

Primary Outcomes (1)

• to assess the efficacy of CAIX-PET

  to assess the Sensitivity of CAIX-PET for the diagnosis of tumors in VHL disease

  IMP administration at day 0; CAIX-PET from day 3 to 7; MRI according to standard of care and imaging assessment from day 1 to 60.

Study Arms (1)

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

EXPERIMENTAL

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

Interventions

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

Participants VHL disease and in VHL-/- tumors, will receive a single dose of the diagnostic radiopharmaceutical \[89Zr\]Zr-DFO-Girentuximab and subsequently undergo imaging with a hybrid PET/CT scanner.

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• IRCCS Ospedale San Raffaele: lead

Study Sites (1)

IRCCS Ospedale San Raffaele

Milan, Italia, 20132, Italy

RECRUITING

MeSH Terms

Conditions

von Hippel-Lindau Disease

Condition Hierarchy (Ancestors)

Neurocutaneous Syndromes; Nervous System Diseases; Angiomatosis; Vascular Diseases; Cardiovascular Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Central Study Contacts

Alessandro Larcher

CONTACT

+39 02.2643.4121; larcher.alessandro@hsr.it

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Doctor

Model Details: phase 2, non-comparative and non-randomized, single arm, open-label, multicenter, national, no-profit clinical

Study Record Dates

• First Submitted: March 25, 2025
• First Posted: September 15, 2025
• Study Start: February 25, 2026
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: March 9, 2026

Record last verified: 2025-11

Locations

IT (1)