Phase 2 N=22 Hydroxyproline Influence on Oxalate Metabolism
Hyperoxaluria
Primary: Mean Percent Conversion of Hydroxyproline (Hyp) to Urinary Oxalate (UOx) — 12.8; 32.9; 14.8 percent converted
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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=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=20 An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
PH1 · Primary Hyperoxaluria · RNAi Therapeutic
Primary: Number of Participants With at Least One Adverse Event (AE) — 13; 7 Participants
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
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 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=22 A Pilot Study of Oxalate Absorption in Secondary Hyperoxaluria
Secondary Hyperoxaluria · Nephrolithiasis · Hyperoxaluria
Primary: Percent of Oxalate Absorption Normalized by Baseline Urinary Oxalate Excretion Over a 24 Hour Test Period — 2.84 percentage of oxalate absorption
Phase 3 N=25 A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
Primary Hyperoxaluria
Primary: Change From Baseline in Plasma Oxalate Concentration After 52 Weeks of Treatment — 14.8; 14.4; -0.71; 2.35 micromole/liter
Phase 2 N=10 Oxazyme in Patients With Hyperoxaluria
Hyperoxaluria
Primary: Urinary Oxalate Creatinine Ratio — 68.6; 38.7; 49.6; 34.7 mg/g — p=0.027
Phase 2 N=12 Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Primary Hyperoxaluria Patients Who Are on Dialysis
Primary Hyperoxaluria
Primary: Change in Pre Dialysis Plasma Oxalate (Total Plasma Oxalate) Level During Treatment With OC5 Compared With Baseline. — 147.57; 11.34; 7.46; -5.68 μmol/L
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=16 Evaluating ALLN-177 for Reducing Urinary Oxalate Excretion in Calcium Oxalate Kidney Stone Formers With Hyperoxaluria
Hyperoxaluria · Nephrolithiasis
Primary: Change From Baseline Period to Treatment Period 24-hour Urinary Oxalate Excretion — -13.92 mg/day
N/A N=58 Obesity and Endogenous Oxalate Synthesis
Obesity
Primary: Urinary Oxalate Excretion — 24 mg/day
Phase 2 N=2 Orlistat for the Treatment of Type I Hyperlipoproteinemia
Hyperlipoproteinemia Type I · Hypertriglyceridemia
Primary: Fasting Serum Triglycerides — 705; 1827; 1511; 1007 mg/dL
N/A N=23 Assessment of Endogenous Oxalate Synthesis
Healthy · Kidney Stone · Obesity
Primary: Endogenous Oxalate Synthesis Rate — 17 mg/day
Phase 2 N=27 Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function
Primary Hyperoxaluria · Primary Hyperoxaluria Type 1 · Primary Hyperoxaluria Type 2
Primary: Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups — -74.06; -68.34; -61.44; -17.98 Percent…
Phase 4 N=9 Effect of Allopurinol and Febuxostat on Urinary 2,8-Dihydroxyadenine Excretion
Adenine Phosphoribosyltransferase Deficiency
Primary: Urinary 2,8-dihydroxyadenine Excretion — 116; 45; 13 mg/24-h — p=<.05
Phase 2 N=40 Use of Oral Probiotics to Reduce Urinary Oxalate Excretion
Nephrolithiasis · Hyperoxaluria · Crohn's Disease
Primary: Change in 24-hour Urinary Supersaturation for Calcium Oxalate — 1.40; 1.36; 1.42 KJoules/mol — p=0.3
Phase 3 N=530 A Multicenter Clinical Trial of Allopurinol to Prevent Kidney Function Loss in Type 1 Diabetes
Diabetic Nephropathies · Coronary Artery Disease
Primary: iGFR at the End of the Wash-out Period — 61.2; 61.2 ml/min/1.73 m^2 — p=0.999
Phase 2 N=99 Febuxostat Versus Allopurinol or Placebo in Patients With Hyperuricosuria and Calcium Oxalate Stones
Hyperuricosuria · Kidney Stones
Primary: Percent Change From Baseline to Month 6 in 24-hour Urine Uric Acid (uUA) Excretion — -58.6; -36.4; -12.7 percent change — p=<0.001
Early Phase 1 N=16 Calcitriol Monotherapy for X-Linked Hypophosphatemia
X-linked Hypophosphatemia · Hypophosphatemic Rickets · Hypophosphatemic Rickets, X-Linked Dominant
Primary: Change in Serum Phosphate in Adults and Children With XLH Between Baseline and 12 Months — 2.6; 2.1 mg/dl — p=0.318
N/A N=26 Oxalobacter Formigenes Colonization and Urinary Oxalate Excretion
Calcium Oxalate Urolithiasis
Primary: Percent Decrease in Urinary Oxalate Excretion After Colonization With O.Formigenes — 14 Percentage decrease in urinary oxalate
Phase 3 N=19 Phentermine/Topiramate for Uric Acid Stones
Obesity · Uric Acid Stones · Type 2 Diabetes Mellitus in Obese
Primary: Kidney Stone Size (mm^3) Determined by Non-contrast CAT Scan — 6540; 4650; 3120; 5041 mm^3
Phase 4 N=12 Growth and Development Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm
Phase 1 N=14 Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
Glucose Transporter Type 1 Deficiency Syndrome · GLUT1 Deficiency Syndrome
Primary: Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time — 13 Participants
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 4 N=13 High Dose or High Dose Frequency Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis 2 Acid Maltase Deficiency
Primary: Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment — 0; 0; 0; 1…
Phase 3 N=12 Betaine and Peroxisome Biogenesis Disorders
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
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