30 closest matches · ranked by relevance
PIK3CA-Related Overgrowth Spectrum (PROS) · Growth Disorder · Genetics
Primary: Percent Change in Unaffected and Affected Fibrofatty Tissue Measured by DXA — 7.9; 4.8; 0.71; 6.5 mean percentage change in tissue volume
PIK3CA-Related Overgrowth Spectrum (PROS)
Primary: Percentage of Patients Responders and Non-responders at Week 24 — 30.4; 55.6; 69.6; 44.4 Percentage of participants
PTEN Gene Mutation · PTEN Hamartoma Tumor Syndrome
Primary: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability], Dropout — 3; 2; 5; 2 Participants
Cowden's Disease · Hamartoma Syndrome, Multiple
Primary: Biochemical Changes in Benign and Malignant Tumor Tissues as Assessed by Immunohistochemistry.
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
Kaposiform Hemangioendotheliomas · Tufted Angioma · Capillary Venous Lymphatic Malformation
Primary: Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score — 47; 7; 3 Participants
Hemangioma
Primary: MRI/MRA of Head/Neck/Chest. — 357 participants
Breast Cancer
Primary: Part II: Objective Response Rate of HER2-negative Metastatic Breast Cancer (by FISH Testing) — 7 percentage of participants
Fibrodysplasia Ossificans Progressiva
Primary: Annualized New Heterotopic Ossification (HO) — 9427.1; 23720.2 cubic millimeters (mm^3)
Fibrodysplasia Ossificans Progressiva
Primary: Parts A and B: Percentage of Flare-ups With No New Heterotopic Ossification (HO) at Week 12 — 64.3; 72.5 percentage of flare-ups
Adenomatous Polyposis Coli
Primary: Change in Marked Polyp Size — 16; 4; 0 Size of marked polyps
Phelan-McDermid Syndrome · Epilepsy
Primary: Number of Adverse Events — 19 events
Acute Coronary Syndrome (ACS)
Primary: Prospect II: Patient Level Non-culprit Lesion Related Non-Culprit Major Adverse Cardiac Event (NC-MACE) Adjudicated to an Originally Untreated Non-culprit Lesion During…
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Fibrodysplasia Ossificans Progressiva
Primary: Percentage of Responders at Week 6 — 100; 88.9; 88.9 percentage of subjects — p=0.1664
Bardet-Biedl Syndrome · POMC Deficiency Obesity · PCSK1 Deficiency Obesity
Primary: Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52 — 85.7; 80.0 percentage of participants
Desmoid Tumor
Primary: Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens — 5.22; 7.44; 4.47; 9.39 score on a scale — p=0.63
Short Stature
Primary: Height Velocity — 3.0; 4.3; 6.2; 4.8 cm/y
Neurofibromatosis 1 · Neurofibroma, Plexiform
Primary: Median Time to Disease Progression — 13.2 Months
Metastatic Breast Cancer
Primary: Number of Participants With and Without Progression Free Survival (PFS) Events — 92; 30 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
Advanced Malignant Solid Neoplasm · Ann Arbor Stage III Non-Hodgkin Lymphoma · Ann Arbor Stage IV Non-Hodgkin Lymphoma
Primary: Objective Response Rate — 0 percentage of patients
Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN
Primary: Percent of Participants With a 20% or More Change in Target Tumor Volume — 0; 8; 11; 0 Participants
TSC1 · TSC2 · Tuberous Sclerosis Complex
Primary: Objective Response Rate — 2 Participants
Stroke · Ischemic Stroke
Primary: Depression — .7; .79 units on a scale
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
Pachyonychia Congenita
Primary: Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels — 15; 15; 15; 14 participants
Acromegaly
Primary: Percentage of Participants With Growth Hormone (GH) and Insulin-like Growth Factor 1 (IGF-1) Observed Response by Dose Class — 20; 28.6; 14.3; 23.1 percentage of…
Tuberous Sclerosis Complex · Self-injury · Autism
Primary: Number of Participants With Compliance to the Treatment Protocol. — 100; 58; 58 percentage of completed visits/measures
Breast Cancer
Primary: Core Phase: Percentage of Participants Who Were Alive Without Disease Progression at 6 Months — 53.8; 46.5; 53.0 Percentage of participants