Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Authorised, recruitment pending
Participants planned
38
Countries
1
Sites
2
Von-Hippel Lindau (VHL) Disease
To explore the role of Carbonic Anhydrase IX as diagnostic and theranostic target in Von-Hippel Lindau disease.
Key facts
- Sponsor
- Ospedale San Raffaele S.r.l.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutics [E02], Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01]
- Decision date (initial)
- 2025-10-24
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Diagnosis, Therapy, Safety
To explore the role of Carbonic Anhydrase IX as diagnostic and theranostic target in Von-Hippel Lindau disease.
Conditions and MedDRA coding
Von-Hippel Lindau (VHL) Disease
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-515679-36-00 | CAT-VHL - Exploring the role of Carbonic Anhydrase IX as diagnostic and Theranostic target in Von-Hippel Lindau disease | Ospedale San Raffaele S.r.l. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 7
- 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
- For the Primary Cohort: diagnosis of VHL disease requiring surveillance following confirmation of pathogenic variant at genetic test
- 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 12
- 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)
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- To evaluate the efficacy of CAIX-PET for the detection of tumors in patients with VHL disease.
Secondary endpoints 3
- To evaluate the diagnostic accuracy of CAIX-PET in VHL -/- tumors diagnosed in the sporadic setting.
- To evaluate the diagnostic accuracy of CAIX-PET in VHL disease.
- To evaluate the safety of CAIX-PET.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD9614939 · Product
- Active substance
- Zirconium (89ZR) Girentuximab
- Substance synonyms
- 89Zr-TLX250, 89Zr-DFO-girentuximab
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS INJECTION
- Max daily dose
- 37 MBq megabecquerel(s)
- Max total dose
- 37 MBq megabecquerel(s)
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- TELIX INTERNATIONAL PTY LTD
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Ospedale San Raffaele S.r.l.
- Sponsor organisation
- Ospedale San Raffaele S.r.l.
- Address
- Via Olgettina 60
- City
- Milan
- Postcode
- 20132
- Country
- Italy
Scientific contact point
- Organisation
- Ospedale San Raffaele S.r.l.
- Contact name
- Alessandro Larcher
Public contact point
- Organisation
- Ospedale San Raffaele S.r.l.
- Contact name
- Alessandro Larcher
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Authorised, recruitment pending | 38 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 13 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol Signature Page_2024-515679-36-01 | 3.0 |
| Protocol (for publication) | D1_Protocol_2024-515679-36-01_for pubblication | 3.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS adults_privacy_for pubblication | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF adults_for pubblication | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_family doctor letter | 1 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_patient card | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_URBBAN consent intro_for publication | 2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_URBBAN consent_for pubblication | 2 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_URBBAN privacy_for publication | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | E1_IB 89Zr-TLX250_V12-1_20-OCT-25_redacted | 12.1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_ENG_2024-515679-36-01_for pubblication | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis_ITA_2024-515679-36-01_for pubblication | 3.0 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-05-30 | Italy | Acceptable 2025-10-24
|
2025-10-24 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-11-10 | Italy | Acceptable | 2026-01-08 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2026-01-24 | Italy | Acceptable with conditions 2026-04-13
|
2026-04-15 |