A Phase 2 interventional study of 68Ga-NY104 PET/CT Contrast-enhanced MRI of the brain and contrast-enhanced CT of abdomen and pelvis) 68Ga-NODAGA-LM3 PET/CT (exploratory) in Von Hippel-Lindau Disease, sponsored by Peking Union Medical College Hospital. Recruiting at 1 site in China. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2023-05-11.
Sponsored by Peking Union Medical College Hospital · Phase 2, Interventional, and Diagnostic
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
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.
Hypothesis
The hypotheses of this study are that
Objectives
Primary objective 1. To determine the sensitivity of 68Ga-NY104 PET/CT using conventional imaging as reference.
Secondary objectives
Exploratory objective
Endpoints
Primary endpoint 1. Per-patient, per-region, and per-lesion positive rate of 68Ga-NY104 using conventional imaging as ground truth.
Secondary endpoints
Exploratory endpoints
35 studies on the registry are indexed under Von Hippel-Lindau Disease; 10 are open to participants now.
This study's planned enrollment of 19 is above the median of 10 across 22 interventional studies indexed under Von Hippel-Lindau Disease.
Browse Von Hippel-Lindau Disease studies →Peking Union Medical College Hospital is the lead sponsor of 1,115 studies on the registry; 463 are open to participants now.
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Exclusion Criteria:
68Ga-NY104 PET/CT Contrast-enhanced MRI of the brain and contrast-enhanced CT of abdomen and pelvis 68Ga-NODAGA-LM3 PET/CT (exploratory)
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.
Binary reading of lesions identified on 68Ga-NY104 PET/CT
Define lesion as PET positive or PET negative lsion
Time frame: From study completion to 1 month after completion
The number of (68Ga-NY104) PET positive lesions
Count the number of lesions (if more than 10, record it as \>10)
Time frame: From study completion to 1 month after completion
The number of (68Ga-NY104) PET positive regions
Any region with at least one (68Ga-NY104) PET positive lesion is considered (68Ga-NY104) PET positive VHL-related region
Time frame: From study completion to 1 month after completion
The number of (68Ga-NY104) PET positive patients
Any patient with at least one (68Ga-NY104) PET positive lesion is considered (68Ga-NY104) PET positive VHL-related patient
Time frame: From study completion to 1 month after completion
Scoring of lesions identified on conventional imaging as very unlikely / unlikely / indeterminate / likely / very likely (score 1-5)
The likelihood of VHL-related neoplasm will be rated combining all imaging features available, including enhancement pattern, typical location, and solitary or multicentric or bilateral distribution.
Time frame: From study completion to 1 month after completion
The number of conventional imaging positive lesions
Count the number of lesions (if more than 10, record it as \>10)
Time frame: From study completion to 1 month after completion
The number of conventional imaging positive regions
Any region with at least one conventional-imaging positive VHL-related lesion is considered conventional-imaging positive VHL-related region
Time frame: From study completion to 1 month after completion
The number of conventional imaging positive patients
Any patient with at least one conventional-imaging positive VHL-related lesion is considered conventional-imaging positive VHL-related patient
Time frame: From study completion to 1 month after completion
The intent of initial management plan (Plan 1) based on conventional imaging
Management plan intent (curative, palliative or surveillance): it should be noted that the curative intent in the present study could be organ specific. For example, if the patient has non-symptomatic CNS hemangioblastomas and a solitary renal cell carcinoma, the intent to resect the renal cell carcinoma should be defined as "curative" even if the CNS hemangioblastomas are left undealt with.
Time frame: From study completion to 1 month after completion
The intent of post-PET management plan (Plan 2) based on all information available, including the results of conventional imaging, 68Ga-NY104 PET/CT, and additional confirmatory studies.
Management plan intent (curative, palliative or surveillance): it should be noted that the curative intent in the present study could be organ specific. For example, if the patient has non-symptomatic CNS hemangioblastomas and a solitary renal cell carcinoma, the intent to resect the renal cell carcinoma should be defined as "curative" even if the CNS hemangioblastomas are left undealt with.
Time frame: From study completion to 1 month after completion
The specified management plan of initial management plan (Plan 1) based on conventional imaging
Specified management plan for each patient including details of surgery or medical treatment or active surveillance. For surgery, this will include type of surgery (e.g., partial nephrectomy or radical nephrectomy, with / without lymph node dissection). For medical treatment, this will include the regimen, dose, and frequency of the treatment plan. For active surveillance, this will include the type and frequency of modality.
Time frame: From study completion to 1 month after completion
The specified management plan of post-PET management plan (Plan 2) based on all information available, including the results of conventional imaging, 68Ga-NY104 PET/CT, and additional confirmatory studies.
Specified management plan for each patient including details of surgery or medical treatment or active surveillance. For surgery, this will include type of surgery (e.g., partial nephrectomy or radical nephrectomy, with / without lymph node dissection). For medical treatment, this will include the regimen, dose, and frequency of the treatment plan. For active surveillance, this will include the type and frequency of modality.
Time frame: From study completion to 1 month after completion
Binary reading of lesions identified on 68Ga-NODAGA-LM3 PET/CT as PET positive lesions or PET negative lesions
Define lesion as PET positive or PET negative lsion
Time frame: From study completion to 1 month after completion
The number of (68Ga-NODAGA-LM3) PET positive lesions
Count the number of lesions (if more than 10, record it as \>10)
Time frame: From study completion to 1 month after completion
The number of (68Ga-NODAGA-LM3) PET positive regions
Any region with at least one (68Ga-NODAGA-LM3) PET positive lesion is considered (68Ga-NODAGA-LM3) PET positive VHL-related region
Time frame: From study completion to 1 month after completion
The number of (68Ga-NODAGA-LM3) PET positive patients
Any patient with at least one (68Ga-NODAGA-LM3) PET positive lesion is considered (68Ga-NODAGA-LM3) PET positive VHL-related patient
Time frame: From study completion to 1 month after completion
Plan to share: Yes — Available upon request
Supporting information: Study protocol, Sap, Icf, Csr, Analytic code
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Peking Union Medical College Hospital