CAIX-targeted PET imaging to find tumors in people with von Hippel‑Lindau (VHL) disease
Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-hippel Lindau Disease
This trial gives a single dose of a CAIX-targeting radiolabeled antibody and does a PET/CT to see if the scan can find VHL‑related tumors in adults with VHL or in people with VHL-associated tumors needing surgery.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 38 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | IRCCS Ospedale San Raffaele Academic / other |
| Drugs / interventions | Girentuximab |
| Locations | 1 site (Milan, Italia) |
| Trial ID | NCT07171905 on ClinicalTrials.gov |
What this trial studies
This is a phase 2, single‑arm, non‑randomized study in which participants receive one injection of [89Zr]Zr‑DFO‑girentuximab followed by hybrid PET/CT imaging to visualize carbonic anhydrase IX (CAIX) expression. The imaging results will be compared to standard-of-care references such as MRI and pathology from surgery to measure sensitivity and diagnostic accuracy. The protocol includes a primary cohort of adults with genetically confirmed VHL under surveillance and a secondary cohort of patients with hemangioblastoma, pheochromocytoma, pancreatic neuroendocrine tumor, or clear‑cell renal cell carcinoma scheduled for surgery. The study also models a theranostic approach by substituting the diagnostic isotope’s decay with therapeutic isotopes in dosimetry simulations to estimate potential therapeutic targeting.
Who should consider this trial
Good fit: Adults (≥18 years) with genetically confirmed VHL needing surveillance or adults with hemangioblastoma, pheochromocytoma, pancreatic neuroendocrine tumor, or clear‑cell renal cell carcinoma planned for surgery who have ECOG performance status 0–2 and agree to contraception and study procedures are ideal candidates.
Not a fit: People who are pregnant or breastfeeding, have ECOG >2, have recent exposure to murine/chimeric antibodies, cannot receive the antibody tracer, or whose tumors do not express CAIX are unlikely to gain benefit.
Why it matters
Potential benefit: If successful, this approach could improve detection of VHL‑associated tumors and help guide targeted therapy decisions by revealing CAIX expression in lesions.
How similar studies have performed: Prior work with 89Zr‑girentuximab has shown good detection of CAIX‑expressing clear‑cell renal tumors, but applying CAIX PET as a diagnostic plus simulated theranostic approach in VHL is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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)
Where this trial is running
Milan, Italia
- IRCCS Ospedale San Raffaele — Milan, Italia, Italy (Recruiting)
Study contacts
- Study coordinator: Alessandro Larcher
- Email: larcher.alessandro@hsr.it
- Phone: +39 02.2643.4121
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.