About Primary Hyperoxaluria
Primary hyperoxaluria is a group of rare autosomal recessive disorders of glyoxylate metabolism causing overproduction of oxalate, leading to recurrent calcium oxalate nephrolithiasis, nephrocalcinosis, and progressive renal failure. Type 1 (AGXT deficiency) is the most severe and common form, frequently resulting in systemic oxalosis when the kidney fails and oxalate deposits in bones, eyes, and heart. Lumasiran (siRNA targeting LDHA) has been approved for PH1, representing a significant therapeutic advance.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Primary Hyperoxaluria trials.
Subtype classification (PH1, PH2, or PH3) by genetic testing is critical as approved therapies and trial eligibility differ by subtype; confirm the causative gene before applying.
24-hour urinary oxalate excretion is the primary pharmacodynamic endpoint in most trials; establish a reliable baseline with repeated measurements under controlled dietary conditions.
Trials for PH1 gene therapy or RNA interference may require a minimum eGFR threshold to ensure adequate drug clearance; check renal function eligibility carefully.
Patient Resources
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