This clinical trial is testing a new drug that selectively lowers the mutant Huntington’s disease protein, whilst leaving the normal huntingtin protein relatively untouched. This study is only for patients who have a positive HD genetic result and are confirmed to carry SNP1 (PRECISION-HD1) or SNP2 (PRECISION-HD2). The primary objective is to understand whether the drug is safe in a small number of volunteers, before testing in a larger population and collecting evidence that the drug may work. The medication will be injected into the CSF by lumbar puncture.
This study is currently recruiting.
PRECISION-HD1 will test the safety, tolerability, pharmacokinestics and pharmacodynamics of of single and multiple doses of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism (SNP) rs362307 (SNP1).
PRECISION-HD2 will test the tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism (SNP) rs362331 (SNP2).
Exclusion Criteria (eligibility that would exclude you from this study)
- Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years.
- Received investigational drug or implantable device in prior 3 months or investigational oligonucleotide in prior 6 months or 5 half-lives of the oligonucleotide, whichever is longer.
- Clinically significant medical condition, unstable psychiatric symptoms, substance abuse, or pregnancy.
- Inability to undergo brain MRI.
- Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture.