A Study to Evaluate the Safety and Dosimetry of 68Ga-OncoCAIX in Patients With CAIX-positive Cancer (OncoCAIX)

A Phase I Study to Evaluate the Safety and Dosimetry of 68Ga-OncoCAIX in Patients With CAIX-positive Cancer

Phase I and multicenter study to evaluate the safety and dosimetry of 68Ga-OncoCAIX in patients with clear cell renal cell carcinoma.

Study Overview

• Status: Completed
• Conditions: Clear Cell Renal Cell Carcinoma (ccRCC)
• Intervention / Treatment: Drug: [68Ga]Ga-OncoCAIX

Detailed Description

The Carbonic Anhydrase IX (CAIX) is a clinically validated marker of clear-cell renal cell carcinoma (ccRCC) and hypoxic tumors. OncoCAIX is a new ligand for CAIX.

This is a Phase I, multicenter study in patients with suspected ccRCC to evaluate the safety, dosimetry and biodistribution of 68Ga-OncoCAIX for imaging of ccRCC. In this trial, 68Ga-OncoCAIX is offered to ccRCC patients who already received standard of care imaging and might therefore complement available modalities.

All patients will undergo PET/CT imaging with [68Ga]Ga-OncoCAIX. The principal objective of the study is to evaluate safety and dosimetry of a single administration of [68Ga]Ga-OncoCAIX. In addition, data on the uptake, biodistribution, PK and excretion of [68Ga]Ga-OncoCAIX is collected and technical parameters are evaluated.

Patients are divided into two cohorts:

• Cohort A: 3 female + 3 male patients without visceral or bone metastases, which would interfere with dosimetry evaluation of healthy organs.
• Cohort B: all patients who meet the eligibility criteria (up to 14 patients)

Both cohorts are recruited in parallel, and patients are assigned to the respective cohort based on their disease extent. Whenever possible, patients are enrolled in cohort A, unless they are unsuitable for cohort A, or the required number of patients of a given sex has already been enrolled in cohort A.

All patients will receive a single intravenous bolus administration of [68Ga]Ga-OncoCAIX and biodistribution, PK, and dosimetry of [68Ga]Ga-OncoCAIX will be assessed based on a series of PET/CT scans, blood and urine sampling.

Full dosimetry evaluations will be performed for all patients in cohort A. Additional dosimetry in the other patients may be performed to the extent that it is possible based on their disease burden (i.e., only organs not affected by malignant lesions can be evaluated).

All patients will be assessed for safety. In addition, relative uptake to appropriate reference tissue of [68Ga]Ga-OncoCAIX as well as semiquantitative parameters such as SUVmax/SUVmean/SUVsd, are determined. The correlation of [68Ga]Ga-OncoCAIX uptake with immunopathology staining for CAIX will be evaluated in patients undergoing surgery or tumor biopsy collection. The lesion detection rate will be compared to 18F-FDG-PET/CT in patients who receive this as part of their standard diagnostic workup.

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 1

Contacts and Locations

Study Locations

• Italy
  • BG
    • Bergamo, BG, Italy, 24127
      • Asst Papa Giovanni XXIII
  • Milano
    • Milan, Milano, Italy, 20132
      • Irccs Ospedale San Raffaele
    • Milan, Milano, Italy, 20133
      • Fondazione IRCCS Istituto Nazionale dei Tumori

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Patients with suspected ccRCC (based on standard diagnostic workup).
2. Requirement for diagnostic imaging or diagnostic imaging recently performed.
3. For female patients: negative serum pregnancy test for women of childbearing potential* (WOCBP). WOCBP must agree to use, from the screening to six months following the study drug administration, highly effective contraception methods, as defined by the "Recommendations for contraception and pregnancy testing in clinical trials" issued by the Head of Medicine Agencies' Clinical Trial Facilitation Group (www.hma.eu/ctfg.html) and which include, for instance, progesterone only or combined hormonal contraception associated with inhibition of ovulation, intrauterine devices, intrauterine hormone-releasing systems, bilateral tubal occlusion or vasectomized partner.
4. For male patients: Subjects able to father children must agree to practice effective contraception for three months starting from the study drug administration.
5. Age ≥ 18
6. ECOG ≤ 1
7. Patient must not have any concomitant infections or active concomitant disease.
8. All acute toxic effects (excluding alopecia and fatigue) of any prior therapy (including surgery, radiation therapy, chemotherapy) must have resolved to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (v. 5.0) Grade ≤ 1.
9. Life expectancy of more than 12 weeks.
10. Ability to undergo imaging study procedures.
11. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
12. Willingness and ability to comply with the scheduled visits, plan, laboratory tests and other study procedures.

Exclusion Criteria:

1. Chronically impaired renal function as expressed by creatinine clearance < 60 mL/min or serum creatinine > 1.5 x ULN.
2. Presence of active hepatitis.
3. Presence of significant cardiac disorders (congestive heart failure, NYHA class III-IV, myocardial infarction within one year prior to study entry, uncontrolled hypertension, or arrhythmia).
4. Any concomitant condition which in the opinion of investigators makes it undesirable for the patient to participate in the study or which could jeopardize compliance with the protocol.
5. Major trauma including major surgery (such as abdominal/cardiac/thoracic surgery) within 4 weeks of administration of the study drug. Minimally invasive procedures such as biopsies are not considered as exclusion criteria.
6. Serious, non-healing wound, ulcer, or bone fracture.
7. Allergy to study medication or excipients in study medication.
8. Any anti-cancer therapy (e.g. cytotoxic chemotherapy, immunotherapy, radiation, surgery, etc.) within 3 weeks before [68Ga]Ga-OncoCAIX-PET/CT scan.
9. Subject has received, or is scheduled to receive, another investigational medicinal product (IMP) from one month before [68Ga]Ga-OncoCAIX injection to end of study participation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Coort A; 3 female + 3 male patients without visceral or bone metastases, which would interfere with dosimetry evaluation of healthy organs.	Drug: [68Ga]Ga-OncoCAIX; Single intravenous bolus administration.
Experimental: Cohort B; all patients who meet the eligibility criteria (up to 14 patients)	Drug: [68Ga]Ga-OncoCAIX; Single intravenous bolus administration.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cohort A: Dosimetry - Effective dose equivalent (mSv)	Whole-body effective dose (mSv) following administration of a single dose of [68Ga]Ga-OncoCAIX.	Assessed on day 1
Cohort A: Dosimetry - Effective dose absorbed doses (mGy)	Absorbed doses (mGy) of normal organs following administration of a single dose of [68Ga]Ga-OncoCAIX.	Assessed on day 1
All patients: Safety (AEs and SAEs)	Safety of administration of [68Ga]Ga-OncoCAIX, according to Common Terminology Criteria for Adverse Events (CTCAE v.5.0).	Throughout the study, until a maximum of 7 days after the administration of the study drug.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Biodistribution profile: SUVmax	Uptake of [ 68Ga]Ga-OncoCAIX in tumor lesions and healthy organs in terms of SUVmax	Assessed on day 1
Biodistribution profile: SUVmean	Uptake of [68Ga]Ga-OncoCAIX in tumor lesions and healthy organs in terms of SUVmean	Assessed on day 1
Biodistribution profile: SUVsd	Uptake of [68Ga]Ga-OncoCAIX in tumor lesions and healthy organs in terms of SUVsd	Assessed on day 1
Pharmacokinetics (PK) - Blood	Pharmacokinetics (PK) of [68Ga]Ga-OncoCAIX based on measurement on blood of residual radioactivity over time [kBq/mL].	Assessed on day 1
Excretion - Urine	Excretion of [68Ga]Ga-OncoCAIX based on measurement of residual radioactivity in the urine over time [kBq]	Assessed on day 1

Collaborators and Investigators

• Sponsor: Philogen S.p.A.

Study record dates

Study Major Dates

• Study Start (Actual): April 16, 2025
• Primary Completion (Actual): November 28, 2025
• Study Completion (Actual): November 28, 2025

Study Registration Dates

• First Submitted: February 17, 2025
• First Submitted That Met QC Criteria: February 17, 2025
• First Posted (Actual): February 21, 2025

Study Record Updates

• Last Update Posted (Actual): January 13, 2026
• Last Update Submitted That Met QC Criteria: January 9, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: clear cell renal cell carcinoma
• Additional Relevant MeSH Terms: Urogenital Diseases; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Adenocarcinoma; Urologic Neoplasms; Carcinoma; Kidney Neoplasms; Carcinoma, Renal Cell
• Other Study ID Numbers: PH-CAIXGA-02/24; 2024-515607-19-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No