vhl alliance

Clinical Trials Currently in Progress

The best method for learning about clinical trials currently in progress for von Hippel-Lindau is to visit clinicaltrials.gov.


Kidney

Pazopanib in Von Hippel-Lindau (VHL) Syndrome
Sponsor:  MD Anderson Cancer Center
Collaborator:  Novartis Oncology
Information provided by (Responsible Party):  M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:  NCT01436227
Contact:  Eric Jonasch, MD; Tel: 713.792.2830; Email: ejonasch@mdanderson.org

Summary: The overall response was 42% overall response rate (50% for kidney as defined by at least 30% shrinkage) in 32 VHL patients. Similar results were seen in the pancreas. A small response was seen in hemangioblastomas including retinal lesions. Toxicity was seen in 7 patients including hemorrhaging. See:
–  http://www.ascopost.com/videos/2017-asco-annual-meeting/eric-jonasch-md-on-von-hippel-lindau-disease-pazopanib-trial/
–  http://abstracts.asco.org/199/AbstView_199_191318.html
–  http://www.practiceupdate.com/content/asco-gu-2017-dr-eric-jonaschs-abstract-recommendations/49645

 


Retina

A Phase I/II Trial for Intravitreous Treatment of Severe Ocular Von Hippel-Lindau Disease Using a Combination of the PDGF Antagonist E10030 and the VEGF Antagonist Ranibizumab (New Study)
Sponsor:  National Eye Institute (NEI)
Information provided by (Responsible Party):  National Institutes of Health Clinical Center (CC) (National Eye Institute (NEI) )
ClinicalTrials.gov Identifier:  NCT0285944 
Contact:  Katherine Hall Shimel, RN; Tel: 301.4402.2863 (office), 240.383.7047(cell); Email: katherine.shimel@nih.gov

National Eye Institute Biorepository for Retinal Diseases
Sponsor:  National Eye Institute (NEI)
Information provided by (Responsible Party):  National Institutes of Health Clinical Center (CC) (National Eye Institute (NEI) )
ClinicalTrials.gov Identifier:  NCT01496625
Contact:  Katherine Hall Shimel, RN; Tel: 301.4402.2863; Email: katherine.shimel@nih.gov

A Phase I/II Trial for Intravitreous Treatment of Severe Ocular Von Hippel-Lindau Disease Using a Combination of the PDGF Antagonist E10030 and the VEGF Antagonist Ranibizumab
Sponsor:  National Eye Institute (NEI)
Information provided by (Responsible Party):  National Institutes of Health Clinical Center (CC) (National Eye Institute (NEI)
ClinicalTrials.gov Identifier:  NCT02859441 
Contact:  Katherine Hall Shimel, RN; Tel: 301.4402.2863; Email: katherine.shimel@nih.gov

 


CNS

Effect of Vorinostat on Nervous System Hemangioblastomas in Von Hippel-Lindau Disease (Missense Mutation Only) (By invitation only)
Sponsor:  National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):  National Institute of Neurological Disorders and Stroke (NINDS)
ClinicalTrials.gov Identifier:  NCT02108002 
Contact:  Prashant Cittiboina, MD; Tel: 301.496.5728; Email: prashant.chittiboina@nih.hhs.gov

 


Pheochromocytoma Diagnosis

Diagnosis of Pheochromocytoma
Sponsor:  Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Information provided by (Responsible Party):  National Institutes of Health Clinical Center (CC) (Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )
ClinicalTrials.gov Identifier:  NCT00004847 
Contact:  Karen Adams, CRNP; Tel: 301.402.7785; Email: adamskt@mail.nih.gov

 


Neuroendocrine Tumors Screening

Evaluation of 68Gallium-DOTATATE PET/CT for Detecting Neuroendocrine Tumors
Sponsor:  National Cancer Institute (NCI)
Information provided by (Responsible Party): National Institutes of Health Clinical Center (CC) (National Cancer Institute (NCI) )
ClinicalTrials.gov Identifier: NCT01967537
Contacts:  Kaitlyn Chambers; Tel: 301.402.4395; Email: katilyn.chambers@nih.gov; Roxanne E Merkel; Tel: 301.402.4395; Email: merkelre@mail.nih.gov

 


Endolympatic Sac Tumors (ELST) Screening

Screening for Endolymphatic Sac Tumours (ELSTs) in von Hippel-Lindau Patients
Sponsor:  Marie Luise Bisgaard, MD
Collaborators:  Rigshospitalet, Denmark, St Thomas’ Hospital, London, Fundación Hospital de Madrid, National Cancer Centre, Singapore
Yokosuka City Shimin Hospitalm, Amrita Institute of Medical Sciences & Research Center
Information provided by (Responsible Party): Marie Luise Bisgaard, MD, University of Copenhagen
ClinicalTrials.gov Identifier: NCT02420067 
Contact:  Marie Luise Bisgaard, MD; Tel: 004531333582; Email: mlbi@sund.ku.dk

 


Genetic Testing

Familial Investigations of Childhood Cancer Predisposition (JSFAMILY) (New Study)
Sponsor:  St. Jude Children’s Research Hospital
Information provided by (Responsible Party):  St. Jude Children’s Research Hospital
ClinicalTrials.gov Identifier:  NCT03050268
Contacts:  Chimene Kesserwan, MD; Tel: 866-278-5833; Email: referralinfo@stjude.org; Lynn Harrison, MPA; Tel: 866-278-5833; Email: referralinfo@stjude.org


Psychosocial Consequences

Psychosocial Consequences of the Screening of Von Hippel Lindau Diseases for Patients Operated for a hémangioblastoma of Nervous Central System (PsychoVHL)
Sponsor:  Assistance Publique Hopitaux De Marseille
Information provided by (Responsible Party):  Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier:  NCT02120040 
Contact:  Philippe Metellus, MD; Tel: 33191385541; Email: pmetellus@ap-hm.fr

 


General

Assessment of Residual VHL Function in Tumors – Can it Predict the Patients’ Individual Course of Disease?
By invitation only
Sponsor:  Marie Luise Bisgaard, MD
ClinicalTrials.gov Identifier:  NCT02207686
Information provided by (Responsible Party):  Marie Luise Bisgaard, MD, University of Copenhagen
Contact:  Marie Luise Bisgaard, MD; Tel: +453.133.3582 713.792.2830; Email: mlbi@sund.ku.dk