Von Hippel-Lindau DiseaseMyVHLNCT03749980
MyVHL: Patient Natural History Study
An observational study of Von Hippel-Lindau Disease and Hereditary Leiomyomatosis and Renal Cell Cancer, run by Joshua Mann, MPH at 1 site in United States.
Trial summary
MyVHL is a multi-patient database which helps researchers identify patterns across VHL patients. MyVHL provides you -and researchers -with more complete information about VHL, like how your lifestyle, medications, and other factors impact the disease and quality of life. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Von Hippel-Lindau Disease, Hereditary Leiomyomatosis and Renal Cell Cancer, Birt-Hogg-Dube Syndrome
- How many people
- 10,000 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2012to Dec 2028
- How long will I take part?
- 1 Year
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Patients with von Hippel-Lindau Disease (VHL)
Who may be able to join
- All patients with von Hippel-Lindau Disease (VHL)
Who cannot join
- -
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
VHL Alliance
Boston, Massachusetts, 02132
Joshua Mann, MPH, 617-277-5667 ext. 4, josh.mann@vhl.org
About this study
MyVHL is a multi-patient database which helps researchers identify patterns across VHL patients. MyVHL provides you -and researchers -with more complete information about VHL, like how your lifestyle, medications, and other factors impact the disease and quality of life. These insights help you better understand the condition and help researchers know where to focus their efforts.
Due to its rarity, there is less understanding of VHL and the factors that may have an impact. The data individuals provide in MyVHL helps researchers identify and uncover factors that may increase risk, inhibit or slow tumor growth, or lead to an effective cure.
- Conditions
- Von Hippel-Lindau Disease, Hereditary Leiomyomatosis and Renal Cell Cancer, Birt-Hogg-Dube Syndrome, SDHB Gene Mutation
- Keywords
- VHL, BHD, HLRCC, SDHB, MyVHL
- Medical subject headings
- von Hippel-Lindau Disease, Hereditary leiomyomatosis and renal cell cancer, Birt-Hogg-Dube Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with CNS, kidney, adrenal, retinal, thyroid, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, as they relate to VHL, BHD, HLRCC, and SDHB and specific genetic mutation.
Data regarding changes in number of CNS, kidney, adrenal, retinal, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, over a lifetime.
Measured: Through study completion, an average of 1 year.
Size of tumors in patients with CNS, kidney, adrenal, retinal, thyroid, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, as they relate to VHL, BHD, HLRCC, and SDHB and specific genetic mutation.
Data regarding changes in size of CNS, kidney, adrenal, retinal, ear, and pancreatic tumors, along with cystadenomas and lesions in the lungs, liver and skin, over a lifetime.
Measured: Through study completion, an average of 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2018
- Study startJan 2012
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Von Hippel-Lindau DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 10,000 participants (estimated)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- Joshua Mann, MPH
- Sponsor type
- University, hospital or other
- Collaborators
- National Organization for Rare Disorders
- Responsible party
- Sponsor-investigator, Joshua Mann, MPH, VHL Alliance
- Investigators
- Joshua Mann, MPH, Principal investigator, VHL Alliance
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03749980
- Sponsor's protocol ID
- 0001
- Acronym
- MyVHL
- Status last verified
- Apr 2024
- First submitted
- 30 May 2018
- Last updated
- 26 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: When participant information is stored, the investigators are careful to protect any patient identifying information from discovery by others. Strict security safeguards are in place to reduce the chance of misuse or unplanned release of information. Researchers will only use participant information in a de-identified manner. De-identified means that the researchers will use participant information without knowing their identity. In some cases, they may use some identifying information about the participant for research purposes, subject to an approval process through the VHL Alliance MyVHL Research Committee. At times, the researchers will use participant information with a code, instead of their name; the code would allow results of the research to be linked back to the participant.
Publications
Larcher A, Rowe I, Belladelli F, Fallara G, Raggi D, Necchi A, Montorsi F, Capitanio U, Salonia A; OSR VHL Program. Von Hippel-Lindau disease-associated renal cell carcinoma: a call to action. Curr Opin Urol. 2022 Jan 1;32(1):31-39. doi: 10.1097/MOU.0000000000000950.
PubMed 34783716