Sponsored by WAVE Life Sciences.
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 a limited number of patients.
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).
Canadian Locations and Contact Information:
Wave Life Sciences: NCT03225846 PRECISION-HD1
Wave Life Sciences: NCT03225833 PRECISION-HD2
Status: Limited recruitment
Location: Centre for Movement and Disorders at 1 Valleybrook Drive, Suite 400, Toronto, ON M3B 2S7
Coordinator: Teena Kailasanathan
Phone: 416-847-7084 ext. 235
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.
For more detailed information on this study and others and for the most recent updates, please go to clinicaltrials.gov ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.