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MyVHL: Patient Natural History Study (MyVHL)

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ClinicalTrials.gov Identifier: NCT03749980
Recruitment Status : Recruiting
First Posted : November 21, 2018
Last Update Posted : November 21, 2018
Sponsor:
Collaborator:
National Organization for Rare Disorders
Information provided by (Responsible Party):
Ilene Sussman, PhD, VHL Alliance

Brief 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. 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.


Condition or disease
Von Hippel-Lindau Disease Hereditary Leiomyomatosis and Renal Cell Cancer Birt-Hogg-Dube Syndrome SDHB Gene Mutation

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 10000 participants
Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration: 1 Year
Official Title: MyVHL: Patient Natural History Study
Actual Study Start Date : January 2012
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024





Primary Outcome Measures :
  1. 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.

  2. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Patients with von Hippel-Lindau Disease (VHL)
Criteria

Inclusion Criteria:

  • All patients with von Hippel-Lindau Disease (VHL)

Exclusion Criteria:

-


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03749980


Contacts
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Contact: Joshua Mann, MPH 161727756674 ext 4 josh.mann@vhl.org
Contact: Ilene Sussman, PhD 16172775667 ext 4 director@vhl.org

Locations
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United States, Massachusetts
VHL Alliance Recruiting
Boston, Massachusetts, United States, 02132
Contact: Joshua Mann, MPH    617-277-5667 ext 4    josh.mann@vhl.org   
Sponsors and Collaborators
Ilene Sussman, PhD
National Organization for Rare Disorders

Additional Information:

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Responsible Party: Ilene Sussman, PhD, Executive Director, VHL Alliance
ClinicalTrials.gov Identifier: NCT03749980     History of Changes
Other Study ID Numbers: 0001
First Posted: November 21, 2018    Key Record Dates
Last Update Posted: November 21, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

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.

Supporting Materials: Study Protocol
Informed Consent Form (ICF)

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Ilene Sussman, PhD, VHL Alliance:
VHL
BHD
HLRCC
SDHB
MyVHL

Additional relevant MeSH terms:
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Carcinoma, Renal Cell
Von Hippel-Lindau Disease
Leiomyomatosis
Birt-Hogg-Dube Syndrome
Neoplastic Syndromes, Hereditary
Skin Neoplasms
Uterine Neoplasms
Adenocarcinoma
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Kidney Neoplasms
Urologic Neoplasms
Urogenital Neoplasms
Neoplasms by Site
Kidney Diseases
Urologic Diseases
Neurocutaneous Syndromes
Nervous System Diseases
Angiomatosis
Vascular Diseases
Cardiovascular Diseases
Ciliopathies
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Leiomyoma
Neoplasms, Muscle Tissue
Neoplasms, Connective and Soft Tissue