Trial Outcomes & Findings for Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease (NCT NCT00062166)

NCT ID: NCT00062166

Last Updated: 2018-11-14

Results Overview

Pancreatic lesions defined by simple cysts, microcystic adenomas, neuroendocrine tumors and other solid lesions of the pancreas were evaluated by 18F Fludeoxyglucose (18F-FDG-PET) imaging to determine if the participant developed metastatic disease (e.g. tumor spreads to different organs).

• Recruitment status: COMPLETED
• Target enrollment: 340 participants
• Primary outcome timeframe: 8 years
• Results posted on: 2018-11-14

Participant Flow

Participant milestones

Measure	Von Hippel Lindau Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.
Overall Study STARTED	340
Overall Study COMPLETED	334
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Measure	Von Hippel Lindau Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.
Overall Study Withdrawal by Subject	6

Baseline Characteristics

Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease

Baseline characteristics by cohort

Measure	Von Hippel Lindau n=340 Participants Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	327 Participants n=5 Participants
Age, Categorical >=65 years	13 Participants n=5 Participants
Age, Continuous	43 years STANDARD_DEVIATION 13 • n=5 Participants
Sex: Female, Male Female	168 Participants n=5 Participants
Sex: Female, Male Male	172 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	29 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	310 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	10 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	2 Participants n=5 Participants
Race (NIH/OMB) Black or African American	15 Participants n=5 Participants
Race (NIH/OMB) White	287 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	25 Participants n=5 Participants
Region of Enrollment United States	340 Participants n=5 Participants

PRIMARY outcome

Timeframe: 8 years

Population: Only 319/340 records are available in the database program for this outcome measure. No records are available for the remaining 21 participants.

Pancreatic lesions defined by simple cysts, microcystic adenomas, neuroendocrine tumors and other solid lesions of the pancreas were evaluated by 18F Fludeoxyglucose (18F-FDG-PET) imaging to determine if the participant developed metastatic disease (e.g. tumor spreads to different organs).

Outcome measures

Measure	Von Hippel Lindau n=319 Participants Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.
Number of Patients With Pancreatic Lesions Defined by Simple Cysts, Microcystic Adenomas, Neuroendocrine Tumors & Other Solid Lesions of the Pancreas Who Had Significant Growth in Lesions or Symptoms Related to the Lesions Requiring Surgical Intervention	47 Participants

SECONDARY outcome

Timeframe: 8 years

Population: The exon mutation information only is available in 63 patients. This protocol did not repeat the gene testing in any of the patients.

Percentage of patients by the location of germline VHL mutations.

Outcome measures

Measure	Von Hippel Lindau n=63 Participants Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.
Percentage of Participants With Exon 3 Mutation Compared to Participants With Exon 1 or 2 Von Hippel Lindau (VHL) Mutations Who Required an Intervention exon 1 and 2 mutation	53.97 percentage of participants
Percentage of Participants With Exon 3 Mutation Compared to Participants With Exon 1 or 2 Von Hippel Lindau (VHL) Mutations Who Required an Intervention exon 3 mutation	46.03 percentage of participants

SECONDARY outcome

Timeframe: initiation of study therapy until 2009, approximately 6 years

Population: Only 319/340 records are available in the database program for this outcome measure. No records are available for the remaining 21 participants.

Count of participants from which we obtained tissue from pancreatic tumor and normal tissue (when applicable) for Deoxyribonucleic acid (DNA), ribonucleic acid (RNA), and proteins extraction.

Outcome measures

Measure	Von Hippel Lindau n=319 Participants Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.
Number of Participants From Which We Obtained Tissue From Pancreatic Lesions and Normal Tissue for Genetic Analysis	46 Participants

SECONDARY outcome

Timeframe: 8 years

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Measure	Von Hippel Lindau n=340 Participants Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.
Count of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)	22 Participants

SECONDARY outcome

Timeframe: 8 years

Count of participants by the type of Von Hippel-Lindau (VHL) gene mutations.

Outcome measures

Measure	Von Hippel Lindau n=156 Participants Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.
Number of Participants With Missense or Non-missense Mutations missense mutations	76 Participants
Number of Participants With Missense or Non-missense Mutations non-missense mutations	80 Participants

Adverse Events

Von Hippel Lindau

Serious events: 22 serious events

Other events: 0 other events

Deaths: 22 deaths

Serious adverse events

Measure

Von Hippel Lindau n=340 participants at risk Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. Other tumors include kidney cancers, pheochromocytoma, eye and central nervous system tumors. These tumors occur at a higher frequency rate than normal population.

General disorders Death

6.5% 22/340 • Number of events 22 • 8 years Because this is natural history protocol, deaths or complications from treatments were not considered as AE or SAE. However, because 22 deaths are noted in the mortality module, 22 deaths are also noted in the SAEs per the Results Data Elements Definitions which state that "SAEs include AEs that result in death. Typically, the event(s) leading to death is listed as an AE Term in the SAE table and the number of participants who died due to any cause are reported in the All-Cause Mortality table."

Other adverse events

Adverse event data not reported

Additional Information

Dr. Naris Nilubol

National Cancer Institute

Phone: 301-451-2355

Email: niluboln@nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place