These trials aim to specifically target and reduce the amount of mutant huntingtin protein produced, whilst leaving the healthy huntingtin protein untouched
Wave Life Sciences are conducting two Phase 1b/2a clinical trials called PRECISION-HD1 and PRECISION-HD2.
These trials aim to specifically target and reduce the amount of mutant huntingtin protein produced, whilst leaving the healthy huntingtin protein untouched.
The treatments will be administered into the fluid in the spinal canal using a needle and syringe. The trials will also analyse the safety and tolerability of each drug. Researchers are also interested to understand how each drug affects the levels of huntingtin protein produced, and their effectiveness against the disease.
This is a placebo-controlled trial, meaning that trial participants will receive placebo (non-treatment) and others will receive the drug. Participants are randomly assigned to receive placebo or drug, and cannot choose which condition they are in. Furthermore, during the study, neither the participant nor the clinical staff will know whether they have received placebo or drug.
Both trials involve the same procedures, however participants in PRECISION-HD1 will be administered a drug called WVE-120101 or placebo, whilst those participating in PRECISION-HD2 will receive the drug WVE-120102 or placebo.