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

Von Hippel-Lindau Disease Clinical Trial

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

Status Recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Von Hippel-Lindau Disease

• NCT number: NCT05810246
• Study type: Interventional
• Source: Peking Union Medical College Hospital
• Contact: Li Huo, MD
• Phone: 18612672038
• Email: huoli@pumch.cn
• Status: Recruiting
• Phase: Phase 2
• Start date: May 1, 2023
• Completion date: December 31, 2025