Phase 2 N=328 Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
Cystinosis
Primary: Number of Participants With Serious and Non-Serious Adverse Events — 257 participants
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Phase 3 N=43 Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
Cystinosis
Primary: The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon® — 0.5152; 0.4367 nmol ½ Cystine / mg protein — p=0.0001
Phase 2 N=9 Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
Cystinosis
Primary: Plasma Pharmacokinetic Parameter: Cmax of Cysteamine — 42.02; 33.06 umol/L
Phase 2 N=10 The Effect of Sodium-glucose Cotransporter (SGLT) 2 Inhibitors on Cystine Stone Formation: A Preliminary Study
Cystinuria
Primary: 24hr Urine Measurements of the Effect of SGLT-2 Inhibitor Therapy on Cystine Production in Urine in Patients With Cystinuria — 999.3 24hr urine cystine concentration…
Phase 2 N=10 Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria
Cystinuria
Primary: Change in Cystine Capacity From Baseline — -63.6; 76; 53.4; 103.1 mg/L
Phase 3 N=60 Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
Cystinosis
Primary: Number of Participants With Treatment-emergent Adverse Events — 58; 37; 24; 32 Participants
Phase 3 N=41 Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis
Cystinosis
Primary: Average Difference Between Morning and Non-Morning Log White Blood Cell (WBC) Cystine Values — -0.229; 0.080 log [nmol ½ cystine/mg protein] — p=0.0048
Phase 4 N=10 Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria
Cystinuria
Primary: Cystine Capacity — 43.1 mg/L
Phase 3 N=17 Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
Cystinosis
Primary: Mean White Blood Cell (WBC) Cystine Concentration at Each Visit — 3.1709; 2.2899; 1.8474; 2.3403 nmol 1/2 Cystine/mg protein
Phase 4 N=10 Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
Infantile Neronal Ceroid Lipofuscinosis
Primary: Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells. — -0.3317 Average number of GRODs
Phase 3 N=169 Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children
Nonalcoholic Fatty Liver Disease (NAFLD)
Primary: Improvement in Nonalcoholic Fatty Liver Disease (NAFLD) — 25; 18 participants — p=0.34
Phase 2 N=50 Lipoic Acid Supplement for Cystine Stone
Cystinuria
Primary: Cystine Stone Recurrence — 16; 23 Participants
N/A N=4 TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study
Cystinuria
Primary: Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8) — 70 mg/L
N/A N=8 Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™
Cystinosis · Nephropathic Cystinosis
Primary: Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC) — 3618; 2799; 2457 min*uM — p=.04
Phase 2 N=14 Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine
Homocystinuria
Primary: Difference in Thiobarbituric Acid Reactive Substances (TBARS) in Individuals With Cystathionine Beta Synthase Deficient Homocystinuria (CBSDH) Pre and Post Taurine…
N/A N=442 Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Phase 3 N=238 Study of Ataluren (PTC124™) in Cystic Fibrosis
Cystic Fibrosis
Primary: Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at Baseline — 62.092; 60.232 percentage of predicted FEV1 — p=0.3264
Phase 3 N=191 Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Cystic Fibrosis
Primary: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) — 100; 97.9; 14.7; 18.8 percent of participants
Phase 2 N=20 Use of an Herbal Preparation to Prevent and Dissolve Kidney Stones
Cystinuria · Nephrolithiasis, Calcium Oxalate
Primary: 24 Hour Urine Supersaturation of Calcium Oxalate (CaOx) — 1.83; 1.83; 1.87; 1.78 KJoules/mol — p=0.32
Phase 2 N=35 Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Niemann-Pick Disease, Type C
Primary: Oxysterol Levels — 28.09; 27.64 ng/mL
Phase 2 N=45 Pharmacokinetics of Linezolid in Children With Cystic Fibrosis
Cystic Fibrosis
Primary: To Determine the Pharmacokinetic Profile of IV (Intravenous) and PO (Oral) Formulations of Linezolid Among Children With Cystic Fibrosis
Phase 2 N=36 Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
Inherited Mitochondrial Disease, Including Leigh Syndrome
Primary: Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV — -0.3; 0.1; -0.6; 0.0 units on a scale — p=0.1875
N/A N=10 Effects of Mycophenolate Mofetil in Cystic Fibrosis Lung Transplant Patients
Cystic Fibrosis · Lung Transplant
Primary: Steady-state Pharmacokinetics of Mycophenolic Acid and Mycophenolic Acid Glucuronide in Stable Cystic Fibrosis and Non-Cystic Fibrosis Lung Transplant Recipients…
N/A N=47 Supplementation of Oral Reduced Glutathione in Pediatric Cystic Fibrosis Patients
Cystic Fibrosis
Primary: Weight Percentile at 3 Months — 33.5; 31.0 Weight Percentile, sex and age adjusted
Phase 2 N=30 A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
Cystic Fibrosis
Primary: Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2 — 1.45; 0.66; -2.81; -2.69 millivolts (mV) — p=0.133
Phase 1 N=20 A Pharmacokinetic and Safety Study of IV Gallium Nitrate (Ganite) in Cystic Fibrosis Patients
Cystic Fibrosis
Primary: Pharmacokinetic Assessment of a 5 Day Infusion of Gallium Nitrate (IV Ganite®) — 503; 537 ug*hr/mL
Phase 3 N=279 Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
Cystic Fibrosis
Primary: Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48 — -1.396; -1.992 Percentage of predicted FEV1 — p=0.5336
Phase 2 N=63 Antioxidant Therapy in RYR1-Related Congenital Myopathy
Neuromuscular Disease
Primary: Urine 15-F2t Isoprostane Concentration — 2.7; 2.6 ng/mg Cr — p=0.88
Phase 2 N=89 A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations
Cystic Fibrosis
Primary: Change From Baseline in Sputum Bacterial Load — -2.1; -1.03; -1.28; -1.7 log 10 cfu/ml
Phase 2 N=4 Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C
Primary: Efficacy of Adrabetadex (VTS-270) to Reduce Plasma Levels of a Conjugated Bile Acid, Known as 5α-cholanic Acid-3β, 5α, 6β-triol N-(Carboxymethyl)-Amide — 32.6; 87.9 ng/ml