Sponsored by WAVE Life Sciences.
WAVE PRECISION-HD1 and PRECISION-HD2 was discontinued on March 29, 2021. For more information please click here.
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.
Status
Study Closed – Not Recruiting
Formal/Alternative Name
PRECISION-HD1: WVE-120101
PRECISION-HD2: WVE-120102
Study Type
Interventional
Purpose
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:
Edmonton, AB
Wave Life Sciences: NCT03225846 PRECISION-HD1
Wave Life Sciences: NCT03225833 PRECISION-HD2
Status: Recruiting
Location: University of Alberta, Edmonton
Principal Investigator: Oksana Suchowersky, MD
Toronto, ON
Wave Life Sciences: NCT03225846 PRECISION-HD1
Wave Life Sciences: NCT03225833 PRECISION-HD2
Status: Recruiting
Location: Centre for Movement and Disorders at 1 Valleybrook Drive, Suite 400, Toronto, ON M3B 2S7
Principal Investigator: Mark Guttman, MD
Montréal, QC
Wave Life Sciences: NCT03225846 PRECISION-HD1
Wave Life Sciences: NCT03225833 PRECISION-HD2
Status: Recruiting
Location: Centre Hospitalier de l-Universite de Montreal, Montreal, QC H2X 019
Principal Investigator: Sylvain Chouinard, MD
Inclusion Criteria
- Prescreened with targeted SNP on the same allele as the pathogenic CAG expansion
- Ambulatory, male or female patients aged ≥25 – ≤65 years
- Clinical diagnostic motor features of HD, defined as Unified Huntington’s Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
- Early manifest HD, Stage I or Stage II based on UHDRS Total Functional Capacity Scores ≥7 and ≤13
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.
Source
ClinicalTrials.gov A service of the U.S. National Institutes of Health
This study is currently recruiting participants. (see Contacts and Locations)
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.