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6 recruiting studies matching your filters

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Phase 3

A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 18 Years of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD

Autosomal Recessive Polycystic Kidney (ARPKD)

Estimated

~$50 – $2,100

Actual pay is set by the research site.

Otsuka Pharmaceutical Development & Commercialization, Inc.NCT04782258

Core A: The Hepato/Renal Fibrocystic Diseases Translational Resource (ARPKD Database Study)

Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and …

Hepato/Renal Fibrocystic DiseaseAutosomal Recessive Polycystic Kidney DiseaseJoubert Syndrome+7 more

Estimated

~$10 – $750

Actual pay is set by the research site.

Children's Hospital of PhiladelphiaNCT01401998

Brain Metabolism Observed at 3 Tesla or 7 Tesla in Health and Metabolic Disease

The goal is to develop methodology to monitor flux in the citric acid cycle in brain via 13C nuclear magnetic resonance (NMR) spectroscopy at 7 Tesla or 3 Tesl…

Glut1 Deficiency Syndrome 1Glucose Metabolism DisordersEpilepsy+4 more

Estimated

~$10 – $750

Actual pay is set by the research site.

Weill Medical College of Cornell UniversityNCT05085704

Phase 2

A Randomized, Double-Blind, Placebo-Controlled, Delayed-Start Study to Evaluate the Efficacy and Safety of Exaluren in Alport Syndrome Patients With Nonsense Mutations in COL4A3/4/5 Genes

This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutation…

Alport Syndrome, X-LinkedAlport Syndrome, Autosomal Recessive

Estimated

~$75 – $2,800

Actual pay is set by the research site.

Eloxx Pharmaceuticals, Inc.NCT07523581

Imaging Assessments of ARPKD Kidney Disease Progression

Autosomal Recessive Polycystic Kidney Disease (ARPKD) is a potentially lethal genetic disorder for which there are currently no disease specific treatments. Cl…

Autosomal Recessive Polycystic Kidney Disease

Estimated

~$10 – $750

Actual pay is set by the research site.

The Cleveland ClinicNCT07201025

Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, …

Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more