About the PH1 Study
The PH1 Study is currently evaluating an investigational medication, ALN-GO1, for the treatment of
Primary Hyperoxaluria Type 1.
Adults and children may be able to take part if they are 6–64 years old, have been diagnosed with PH1, and have not had a liver and/or kidney transplant (other criteria will apply). Interested individuals would first be assessed to determine if the study is right for them.
Primary Hyperoxaluria Type 1 is a rare, inherited condition that occurs when the liver makes too much of a substance called oxalate. This can result in painful, recurrent kidney stones, and eventually cause kidney failure. Symptoms of PH1 can start during infancy, childhood, or adulthood.
The investigational medication, ALN-GO1, may reduce or prevent the overproduction of oxalate in people with PH1, using a new approach called RNA interference (RNAi). Researchers at Alnylam Pharmaceuticals are also using RNAi therapeutics to research and develop medicines for other rare, inherited conditions.
In clinical research studies, also called clinical trials, people volunteer to receive an investigational medication under closely monitored conditions. The studies are designed to find out if the investigational medication is safe and if it works.