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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
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
Exploratory endpoints
1. Per-patient, per-region, and per-lesion positive rate of 68Ga-NODAGA-LM3 using conventional imaging as ground truth.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Diagnostic imaging arm | Experimental | 68Ga-NY104 PET/CT Contrast-enhanced MRI of the brain and contrast-enhanced CT of abdomen and pelvis 68Ga-NODAGA-LM3 PET/CT (exploratory) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Binary reading of lesions identified on 68Ga-NY104 PET/CT | Define lesion as PET positive or PET negative lsion | 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) | 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 | 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 | 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. | 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) | From study completion to 1 month after completion |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | From study completion to 1 month after completion |
| The number of (68Ga-NODAGA-LM3) PET positive lesions |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Li Huo, MD | Contact | 18612672038 | huoli@pumch.cn | |
| Wenjia Zhu, MD | Contact | 18614080164 | zhuwenjia_pumc@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Li Huo, MD | Peking Uion Medical College Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Recruiting | Beijing | Beijing Municipality | 100730 | China |
Available upon request
Within 2 years after the publication of the main results
No limit.
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| ID | Term |
|---|---|
| D006623 | von Hippel-Lindau Disease |
| ID | Term |
|---|---|
| D020752 | Neurocutaneous Syndromes |
| D009422 | Nervous System Diseases |
| D000798 | Angiomatosis |
| D014652 | Vascular Diseases |
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|
| 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 |
| 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 | From study completion to 1 month after completion |
| 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. | 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. | 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. | From study completion to 1 month after completion |
Count the number of lesions (if more than 10, record it as >10) |
| 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 | 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 | From study completion to 1 month after completion |
| D002318 |
| Cardiovascular Diseases |
| D000072661 | Ciliopathies |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |