N/A N=62 PHIL in the Treatment of Intracranial dAVF.
Arteriovenous Dural Fistula
Primary: Subjects With Neurological Death or Ipsilateral Stroke — 0 Participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
N/A N=129 Premenstrual Hormonal and Affective State Evaluation (PHASE) Project
Premenstrual Dysphoric Disorder
Primary: Hormone Analysis — 70.7; 57.8; 114; 97.3 ng/mL
Phase 2 N=12 Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
Tetrahydrobiopterin Deficiencies · Hyperphenylalaninemia, Non-Phenylketonuric
Primary: Blood Phenylalanine(Phe) Levels Measured at Specified Timepoints — 132.9; 72.2; 315.0; 104.1 μmol/L
Phase 3 N=21 A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
Primary Hyperoxaluria Type 1 · Primary Hyperoxaluria
Primary: Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6 — -33.33 percent change
Phase 3 N=18 A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Primary Hyperoxaluria · Primary Hyperoxaluria Type 1 (PH1)
Primary: Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 — -71.97 percent change
N/A N=39 Early-onset Obesity and Cognitive Impairment in Children With Pseudohypoparathyroidism
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Phase 2 N=35 A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
Primary Hyperoxaluria Type 1 (PH1) · Primary Hyperoxaluria Type 2 (PH2) · Kidney Diseases
Primary: AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox — 3507.4; -1664.4 Percent change in 24-hour Uox AUC — p=<0.0001
Phase 3 N=90 Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
Phenylketonurias
Primary: Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria — 20.9; 2.9 mg/kg/day — p=<0.001
Phase 2 N=8 A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
BH4 Deficiency · Hyperphenylalaninemia
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 2; 2; 4; 3 Participants
Phase 3 N=39 A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
Primary Hyperoxaluria Type 1 (PH1)
Primary: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 — -11.8; -65.4 percent change — p=<0.0001
Phase 2 N=6 Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy)
Pseudohypoparathyroidism Type 1a · Albright Hereditary Osteodystrophy
Primary: Change in Urine cAMP — 3048 fm/uL
Phase 2 N=29 Ph 1/2 Study Evaluating Safety and Tolerability of Inhaled AP-PA02 in Subjects With Chronic Pseudomonas Aeruginosa Lung Infections and Cystic Fibrosis
Cystic Fibrosis · Pseudomonas Aeruginosa · Pseudomonas
Primary: Incidence and Severity Treatment Emergent Adverse Events (TEAEs) — 1; 1; 1; 0 Participants
Phase 2 N=11 Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
Urea Cycle Disorders
Primary: Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing Adverse Events) — 4; 2 participants
Phase 3 N=23 Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
Urea Cycle Disorders
Primary: Adverse Events — 0; 6 participants
N/A N=799 Positive Health Check Evaluation Trial
Human Immunodeficiency Virus (HIV) Positive
Primary: Number of Participants With Viral Load Suppression — 210; 213 Participants — p=0.220
Phase 1 N=90 Safety and Tolerability of Preservative-free Polyhexamethylene Biguanide (PHMB) Ophthalmic Solution in Healthy Subjects
Acanthamoeba Keratitis
Primary: Number of Subjects With Dose-limiting Adverse Events — 0; 2; 3; 0 participants
Phase 2 N=32 Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
Primary Hyperoxaluria Type 1 (PH1)
Primary: Number of Participants With Adverse Events (AEs) — 5; 6; 2; 6 Participants
Phase 2 N=9 Hydroxychloroquine and Metabolic Outcomes in Patients Undergoing TPAIT
Chronic Pancreatitis · Insulin Dependent Diabetes
Primary: Quotient of Stimulated C-peptide/Glucose Level Normalized for IEQ/Kg Infused in Response to MMTT — .000000077; 0.000000079 ng/mg glucose/IEQ/kg
Phase 3 N=60 Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)
Urea Cycle Disorders
Primary: Rate of Adverse Events (Number of Participants Who Experienced Any AE Considered Related to Study Drug) — 33 participants
Phase 2 N=28 An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
Pitt Hopkins Syndrome
Primary: Safety and Tolerability — 15; 0; 4; 12 Participants
Phase 4 N=9 Vitamin D Repletion in Primary Hyperparathyroidism
Primary Hyperparathyroidism · Vitamin D Deficiency
Primary: Serum Parathyroid Hormone (PTH) Level
Phase 1 N=14 A Pharmacokinetic and Glucodynamic Study of Subcutaneously Administered Insulin Analogs With rHuPH20 Compared to Insulin Analogs Alone
Healthy
Primary: Area Under the Concentration-time Curve for Serum Insulin From Time 0 to 60 Minutes (AUC0-60) — 11667.14; 23807.14; 10687.14; 27850.00 minutes*nanomolars (min*nM)…
Phase 2 N=16 Investigation of H01 in Adults With Pulmonary Hypertension Including Interstitial Lung Disease (The SATURN Study).
Pulmonary Hypertension
Primary: Calculated Pulmonary Vascular Resistance (PVR) — 7.97; 8.23; 7.27; 7.11 Wood units (WU) — p=0.541
Phase 4 N=56 Canadian Hemophilia Prophylaxis Study
Severe Hemophilia A
Primary: Number of Participants Who Developed Target Joint Bleeding — 17 Participants
Phase 3 N=1,059 A Phase 3 Study to Investigate the Safety and Efficacy of PA21, a Phosphate Binder, in Dialysis Patients
Chronic Kidney Disease Requiring Chronic Dialysis
Primary: Change in Serum Phosphorus Levels From Week 24 to Week 27 — 0.25; 1.92 mg/dL
Phase 3 N=336 A Phase III, Multicentre, Double-Blind, Placebo-Controlled Withdrawal Study in Patients With Hyperphosphatemia
Chronic Kidney Disease · Hyperphosphatemia
Primary: Change in Serum Phosphorus Levels From Week 12 to Week 16 (LOCF) (ITT2) — -0.24; 1.27 mg / dL
Phase 2 N=22 Phase II Pharmacokinetics Study of Humalog and Humulin-R With and Without rHuPH20 in Type 1 Diabetes Mellitus
Type 1 Diabetes Mellitus
Primary: Area Under the Insulin Concentration-time Curve for the First Hour (AUC0-60) — 12346.67; 8297.62; 11607.65; 4331.76 nanomole*minute per liter (nmol*min/L) — p=0.0011
Phase 2 N=13 Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
Hyperoxaluria
Primary: Urinary Oxalate Excretion — 1.43; 1.04 umol/mg — p=0.007
N/A N=9 A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
Phenylketonuria
Primary: Change From Baseline in Rapid Visual Processing (RVP) Mean Response Latency (CANTAB Task) — -24.23; 18.03 Millisec
Phase 2 N=10 Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease
Huntington Disease
Primary: Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr — 0.44; 0.43; 0.44; 0.42 ratio