VHL - Von Hippel-Lindau SyndromePhase 2CAT-VHLNCT07171905
CAT-VHL Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-Hippel Lindau Disease
A Phase 2 study testing investigating the role of CAIX as target in VHL disease and in VHL-/- tumors. in people with VHL - Von Hippel-Lindau Syndrome, run by IRCCS Ospedale San Raffaele at 1 site in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- VHL - Von Hippel-Lindau Syndrome
- What is being tested?
- investigating the role of CAIX as target in VHL disease and in VHL-/- tumors.
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteItaly
- Study dates
- 25 Feb 2026to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
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- 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.
Who cannot join
- 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
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- 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)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
IRCCS Ospedale San Raffaele
Milan, Italia, 20132
Alessandro Larcher, +39 02.2643.4121, larcher.alessandro@hsr.it
Sabrina Bettini, +39 0226436268, bettini.sabrina@hsr.it
About this study
Official title: Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-hippel Lindau Disease
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
- Being tested
- investigating the role of CAIX as target in VHL disease and in VHL-/- tumors.
- Medical subject headings
- von Hippel-Lindau Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| investigating the role of CAIX as target in VHL disease and in VHL-/- tumors | Experimental | Not described Receives: Drug: investigating the role of CAIX as target in VHL disease and in VHL-/- tumors. |
What is being measured
The primary outcome is the main question the study is designed to answer.
to assess the efficacy of CAIX-PET
to assess the Sensitivity of CAIX-PET for the diagnosis of tumors in VHL disease
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2025
- Study start25 Feb 2026
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for VHL - Von Hippel-Lindau SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- phase 2, non-comparative and non-randomized, single arm, open-label, multicenter, national, no-profit clinical
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- IRCCS Ospedale San Raffaele
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Larcher Alessandro, IRCCS Ospedale San Raffaele
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07171905
- Sponsor's protocol ID
- PNRR-POC-2023-12377493
- Acronym
- CAT-VHL
- Status last verified
- Nov 2025
- First submitted
- 25 Mar 2025
- Last updated
- 9 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-hippel Lindau Disease
- ClinicalTrials.gov ID
- NCT07171905
- Acronym / sponsor ID
- CAT-VHL / PNRR-POC-2023-12377493
- Conditions
- VHL - Von Hippel-Lindau Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- IRCCS Ospedale San RaffaeleUniversity, hospital or other
- Study start
- 25 Feb 2026 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 15 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- phase 2, non-comparative and non-randomized, single arm, open-label, multicenter, national, no-profit clinical
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| investigating the role of CAIX as target in VHL disease and in VHL-/- tumors.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. | Drug | investigating the role of CAIX as target in VHL disease and in VHL-/- tumors |
Primary outcome
to assess the efficacy of CAIX-PET
Time frame: 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.
to assess the Sensitivity of CAIX-PET for the diagnosis of tumors in VHL disease
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing investigating the role of CAIX as target in VHL disease and in VHL-/- tumors. in people with VHL - Von Hippel-Lindau Syndrome, run by IRCCS Ospedale San Raffaele at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alessandro Larcher (+39 02.2643.4121, larcher.alessandro@hsr.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
to assess the efficacy of CAIX-PET (time frame: 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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2026.