30 closest matches · ranked by relevance
Adrenal Insufficiency · Excess Androgen · Congenital Adrenal Hyperplasia (CAH)
Primary: Number of Patients With 17-OHP Levels Equal or Below 1,200 ng/dL at 0700 — 0 Participants
CAH - Congenital Adrenal Hyperplasia
Primary: Percent Change From Baseline to Day 14 in 17-hydroxyprogesterone (17-OHP) Morning Window Averages — -60; -58; -53; -64 Percent (%)
Congenital Adrenal Hyperplasia
Primary: Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101 — 2…
Congenital Adrenal Hyperplasia · 21-Hydroxylase Deficiency · Adrenogenital Syndrome
Primary: Chronocort vs. Cortef Cortisol Concentrations (AUC Over 24 Hours - Time Points 0,.5,1,1.5,2,3,4,5,6,7,8,10,10.5,11, 11.5,12,13,15,17,17.5,18,18.5,19,20,22,24 Post Dose)…
Congenital Adrenal Hyperplasia (CAH)
Primary: Adult Height Relative to General Population — -0.34; -0.6 Standard Deviation Score (SDS) units
Congenital Adrenal Hyperplasia · Hyperplasia · Adrenal Hyperplasia
Primary: Cortisol Exposure — 110 hr*mcg/dL
Congenital Adrenal Hyperplasia
Primary: Change From Baseline to 24 Weeks of the Mean of the 24-hour Standard Deviation Score (SDS) Profile for 17-OHP — -0.403; -0.172 Z-score — p==0.5521
Congenital Adrenal Hyperplasia · CAH - Congenital Adrenal Hyperplasia
Primary: Safety of SPR001 in Patients With CAH — 6; 5; 4 Participants
CAH - Congenital Adrenal Hyperplasia
Primary: Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average) — -69.48 percent change
Congenital Adrenal Hyperplasia · CAH - Congenital Adrenal Hyperplasia · CAH - 21-Hydroxylase Deficiency
Primary: The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH — 9; 2 Participants
Congenital Adrenal Hyperplasia
Primary: Percentage of Participants Who Were Biochemical Responders at Week 28 — 40.0; 14.3 percentage of participants — p=0.0003
Endocrine Disease · Adrenal Insufficiency · Congenital Adrenal Hyperplasia
Primary: Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia — 601.213 nmol/L
Adrenal Insufficiency
Primary: Serum Cortisol Concentration up to 240 Minutes — 575.8 nmol/L
Adrenal Hyperplasia, Congenital
Primary: Percent Difference in the Mean Log Transformed Area Under the Curve of 17-hydroxyprogesterone Between Regimens — 3.16; 3.91 Log Mean Area Under the Curve — p=0.09
Development
Primary: 17-hydroxyprogesterone Response to ACTH — 2.4; 3.7; 3.1; 2.9 ng/mL
PCOS
Primary: 17OHP Levels After hCG — 3.2; 1.3 ng/ml
Polycystic Ovary Syndrome
Primary: Estradiol Level in Female Offspring — 18606; 6467 pg/ml — p=0.049
Polycystic Ovary Syndrome
Primary: 17-hydroxyprogesterone Responses to hCG in PCOS Women and Normal Controls — 1.3; 3; 1.1 ng/ml
Congenital Adrenal Hyperplasia
Primary: Safety and Tolerability - Number of Participants With Adrenal Insufficiency — 25; 41 Participants
Congenital Hyperinsulinism
Primary: Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind) — 2; 0 Participants
Congenital Hyperinsulinism
Primary: Median of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline (BL) and End of Treatment (EOT) — 81.7; 72.9; 83.4…
Infantile Refsum's Disease · Zellweger Syndrome · Adrenoleukodystrophy
Primary: Number of Participants With Excretion of Atypical Bile Acids in Urine by Category — 10; 51; 11; 9 Participants
Congenital Hyperinsulinism · Hyperinsulinism · Persistent Hyperinsulinemic Hypoglycemia of Infancy
Primary: The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism — 40; 6; 3; 46 Participants
Acne Vulgaris
Primary: Change in HPA Axis Response as Measured by CST — 12.50; 22.95 mcg/dL
Congenital Hyperinsulinism (CHI) · Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI)
Primary: Accuracy of 18F-DOPA PET/CT Scans to Detect Focal Lesions in Children With Congenital Hyperinsulinism — 34; 10; 4; 52 cases
Smith-Lemli-Opitz Syndrome
Primary: Change in Plasma Cholesterol — 37.8 change (%) compared to baseline — p=0.011
Adrenal Insufficiency · Cirrhosis · Spur Cell Anemia
Primary: Number of Participants With Cholesterol Esterification Deficiency — 10; 11 Participants
Congenital Sucrase-isomaltase Deficiency (CSID)
Primary: Prevalence of CSID Genetic Variants — 27; 0 Participants
Polycystic Ovary Syndrome
Primary: Serum 17OHP Responses to hCG — 2.0; 1.2; 1.1; 1.0 ng/ml — p=<0.05
Congenital Heart Defects
Primary: Transition Readiness Assessment Questionnaire (TRAQ) Score — 2.77; 2.95; 3.80; 3.92 units on a scale