NCT05810246

68Ga-NY104 PET/CT in Von Hippel-Lindau Disease

68Ga-NY104 PET/CT and Conventional Imaging in Patients With Von Hippel-Lindau Disease: a Prospective, Single-center, Single-arm, Comparative Imaging Study

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 19 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, single-center, single-arm, diagnostic phase 2 study in patients with von Hippel-Lindau disease. VHL disease is a rare syndrome characterized by VHL gene mutation and HIF activation. Although genetic testing is available, the manifestations of the syndrome are protean; therefore, imaging plays a crucial role in the identification of abnormalities and subsequent follow-up of lesions. For now, conventional imaging serves as the main radiologic modality in the characterization of VHL disease. In this study, we aim to evaluate the sensitivity of 68Ga-NY104 PET/CT in patients with VHL disease. 68Ga-NY104 is a novel small molecule PET tracer targeting carbonic anhydrase IX, which is a down-streaming target of HIF and overexpressed in HIF activation. 68Ga-NY104 PET/CT is likely to function as a sensitive imaging tool to identify VHL-related tumors and to impact patient management if additional lesions are identified. The hypotheses of this study are that * 68Ga-NY104 PET/CT can be used as an effective imaging modality in VHL syndrome with high sensitivity * 68Ga-NY104 PET/CT may detect lesions that are missed on conventional imaging and can result in management impact. A total of 19 patients will be recruited at Peking Union Medical College Hospital. As an exploratory end-point, a 68Ga-NODAGA-LM3 PET/CT sub-study will be performed in patients with evidence of neuroendocrine tumors.

Detailed description

Hypothesis The hypotheses of this study are that * 68Ga-NY104 PET/CT can be used as an effective imaging modality in VHL syndrome with high sensitivity * 68Ga-NY104 PET/CT may detect lesions that are missed on conventional imaging and can result in management impact. Objectives Primary objective 1. To determine the sensitivity of 68Ga-NY104 PET/CT using conventional imaging as reference. Secondary objectives 1. To determine the incremental management impact of 68Ga-NY104 PET/CT 2. To assess the interobserver agreement of 68Ga-NY104 PET/CT by comparing the two blinded independent readings Exploratory objective 1\. To compare the per-patient, per-region, and per-lesion sensitivity of 68Ga-NY104 PET/CT to 68Ga-NODAGA-LM3 PET/CT in an exploratory endpoint Endpoints Primary endpoint 1. Per-patient, per-region, and per-lesion positive rate of 68Ga-NY104 using conventional imaging as ground truth. Secondary endpoints 1. Incremental impact of 68Ga-NY104 PET/CT on choice of management, defined as a decision to alter the original plan of treatment (based on conventional imaging) after considering the result of 68Ga-NY104 PET/CT (Impact is categorized as high, medium, low or no incremental impact.) 2. Observer agreement in interpretation of 68Ga-NY104 PET/CT between the two independent nuclear medicine readers. Exploratory endpoints 1\. Per-patient, per-region, and per-lesion positive rate of 68Ga-NODAGA-LM3 using conventional imaging as ground truth.

Conditions

• Von Hippel-Lindau Disease

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	68Ga-NY104 PET/CT Contrast-enhanced MRI of the brain and contrast-enhanced CT of abdomen and pelvis) 68Ga-NODAGA-LM3 PET/CT (exploratory)	68Ga-NY104 PET/CT is expected to be completed within 21 days following screening. Participants will be administered a single, intravenous bolus of 68Ga-NY104 (1.8-2.2 MBq per kilogram bodyweight). PET/CT scanning will occur at 45 -75 minutes following injection of 68Ga-NY104. Conventional imaging should be performed within one month from 68Ga-NY104 PET/CT. It includes contrast-enhanced MRI of brain and contrast-enhanced CT of abdomen and pelvis. 68Ga-NODAGA-LM3 PET/CT is optional in patients with evidence of or in suspicion of pheochromocytoma, paraganglioma, or pancreatic neuroendocrine tumor. They are encouraged to undergo exploratory 68Ga-NODAGA-LM3 PET/CT for better evaluation of neuroendocrine tumors. The decision, however, is up to the participants. 68Ga-NODAGA-LM3 PET/CT should be performed within one month after 68Ga-NY104 PET/CT. The details of 68Ga-NODAGA-LM3 PET/CT are similar to 68Ga-NY104 PET/CT.

Timeline

• Start date: 2023-05-01
• Primary completion: 2025-05-01
• Completion: 2025-12-31
• First posted: 2023-04-12
• Last updated: 2023-05-11

Locations

1 site across 1 country: China