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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 26 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MyVHL: Patient Natural History Study
- ClinicalTrials.gov ID
- NCT03749980
- Acronym / sponsor ID
- MyVHL / 0001
- Conditions
- Von Hippel-Lindau Disease, Hereditary Leiomyomatosis and Renal Cell Cancer, Birt-Hogg-Dube Syndrome, SDHB Gene Mutation
- Keywords
- VHL, BHD, HLRCC, SDHB, MyVHL
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10,000 participants (estimated)
- Lead sponsor
- Joshua Mann, MPHUniversity, hospital or other
- Collaborators
- National Organization for Rare Disorders
- Study start
- Jan 2012 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 21 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 1 Year
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with von Hippel-Lindau Disease (VHL)
Key inclusion criteria (1 listed)
- All patients with von Hippel-Lindau Disease (VHL)
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Time frame: Through study completion, an average of 1 year.
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.
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.
Time frame: Through study completion, an average of 1 year.
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Apr 2024.
- Derived from this study: PubMed 34783716Larcher 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.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Joshua Mann, MPH principal investigator
VHL Alliance
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Joshua Mann, MPH (161727756674 ext. 4, josh.mann@vhl.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: Through study completion, an average of 1 year.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03749980, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Apr 2024.