27 closest matches · ranked by relevance
Von Hippel-Lindau Disease
Primary: Number of Patients With Pancreatic Lesions Defined by Simple Cysts, Microcystic Adenomas, Neuroendocrine Tumors & Other Solid Lesions of the Pancreas Who Had Significant…
Von Hippel-Lindau Syndrome
Primary: Overall Response Rate (Complete Response + Partial Response) — 13 Participants
Hippel-Lindau Disease · Kidney Cancer
Primary: Number of Participants With a Renal Tumor Response — 0 participants
Von Hippel-Lindau Syndrome
Primary: Tabulation of Adverse Events — 13 adverse events
VHL Gene Mutation · VHL · VHL Syndrome
Primary: Overall Response Rate (ORR) in VHL Disease-Associated ccRCC Tumors — 0.0 Percentage of Participants
Von-Hippel Lindau · Kidney Disease, Chronic
Primary: Comparison in Radiologist's Lesion Evaluation: CEUS Versus B-mode Ultrasound — 0.13; 0.20 Dichotomized score
Renal Cancer · Von Hippel Lindau
Primary: Overall Response Rate. — 3; 0; 75; 0 percentage of participants
Hemangioma
Primary: MRI/MRA of Head/Neck/Chest. — 357 participants
HLRCC · Sporadic Papillary Renal Cell Cancer
Primary: Overall Response Rate — 72; 35 Percentage of participants
Hereditary Hemorrhagic Telangiectasia
Primary: Change in Lesion Area of Treated Telangiectasia. — -6; -8 mm^2
Peripheral Artery Disease
Primary: Six-minute Walk Distance — 17.01; 3.46 meters — p=0.7586
Tuberous Sclerosis Complex (TSC) · Lymphangioleiomyomatosis (LAM)
Primary: Angiomyolipoma Response Rate as Per Central Radiology Review — 41.8; 0; 58.0 Percentage of Participants — p=<0.0001
Hemangioma
Primary: Frequency of Hepatic Hemangiomas Identified on Abdominal Ultrasound — 24 participants
Nonmalignant Neoplasm · Tuberous Sclerosis · Lymphangioleimyomatosis
Primary: Objective Response Rate — 44.4 percentage of participants
Pheochromocytoma · Paraganglioma
Primary: Number of Participants With Clinical Response (Partial Response + Complete Response) — 5; 0; 5; 2 Participants
Cowden's Disease · Hamartoma Syndrome, Multiple
Primary: Biochemical Changes in Benign and Malignant Tumor Tissues as Assessed by Immunohistochemistry.
Hereditary Haemorrhagic Telangiectasia
Primary: HHT Patients Who by the Questionnaire Did or Did Not Self Report a Medical Complication During Flight — 77 percentage of patients
Telangiectasia, Hereditary Hemorrhagic
Primary: Change From Baseline Epistaxis Severity Score — -1; -1.1; -1.3; -1.2 units on a scale
Albinism · Inborn Errors of Metabolism · Oculocutaneous Albinism
Primary: Change in Forced Vital Capacity (36 Months) — -23.52; -20.93 % of predicted volume
Familial Hyperlipidemia
Primary: Familial Hypercholesterolemia (FH) Determination for Family Members of School Children With Elevated Cholesterol and Documented FH Variant — 3; 8 participants
Renal Cell Carcinoma
Primary: Progression-free Survival — 1.7 months
Secondary Hyperparathyroidism
Primary: The Level of Activated Vitamin D (1,25-dihydroxyvitamin D) After Parathyroid Hormone Infusion at Baseline, 4 and 8 Hours — 40.6; 46.2; 39.8; 58.8 pg/ml — p=0.1
Birt-Hogg-Dube Syndrome
Primary: Number of Patients With Birt-Hogg-Dube Syndrome Who Experienced Spontaneous Pneumothorax During or Within 24 Hours Following Air Travel — 8 Participants
Cancer
Primary: Maximum Observed Plasma Concentration and Concentration at Steady-state Following Multiple Doses of Vismodegib — 23.6; 30.7; 30.5; 20.5 micromolar
Paraganglioma · Pheochromocytoma
Primary: Clinical Benefit Rate (CBR) Which is Defined as Either a Partial Response (PR) Complete Response (CR) or Stable Disease (SD) for ≥ 12 Weeks Measured Using Response…
Hereditary Haemorrhagic Telangiectasia (HHT)
Primary: Number of Participants That Achieving Personal Recommended Intake of Iron — 43 Participants
Lymphangioleiomyomatosis
Primary: Changes in Forced Expiratory Volume in One-second (FEV1) Slope in Milliliters Per Month — 1; -12 milliliters per month — p=<0.001