A Study of the Safety and Tolerability of Experimental WVE-120102 For Huntington's Disease

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Age: 25 to 65 years old
Healthy Volunteers: No
Keywords: Huntington’s Disease, HD
Type: Drug study, Phase [/1/,/ /2/]
8 Participants
Huntington's Disease (HD) is a rare progressive neurological disease. It affects thought processes, movement, personality, and/or mood. HD is caused by known mutations on a single gene, called the Huntingtin gene (HTT). This results in a repeat of 3 amino acids called the “CAG repeat”. All people carry the Huntingtin gene which makes Huntingtin protein. This protein is important for normal brain development. The CAG repeat on the HTT gene in people with HD leads to production of another type of protein known as the mutant Huntingtin (mHTT) protein. The study researchers believe if you can lower the amount of mHTT, you may be able to help treat Huntington’s Disease.

Although treatments for symptoms are available, there is currently no cure for HD. The Sponsor is developing an experimental drug, WVE-120102. It has been developed to treat patients with HD with a mutation at a single point in their DNA, called a “SNP” (pronounced “snip”). SNP2 is often found on the HTT gene of HD patients and WVE-120102, the drug that will be investigated in this study, targets SNP2. Because the investigational drug being tested in this study is only expected to act on SNP2, we do not think the drug will help you if you do not carry SNP2. If you do not have SNP2 you will not be eligible for entry into this study.

The purpose of this study is to determine if WVE-120102 is safe and well tolerated in the treatment of this disease when compared with a placebo control. WVE-120102 has not been tested previously in humans.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

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 Key

Exclusion criteria:

  • 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 halflives 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
Benefits and risks of participating

We cannot promise any benefits to you as a result of taking part in this research study.


The study doctor will discuss the risks associated with taking part in this research study.
If you agree to take part in this research study, we will compensate you for your time and effort. You will be paid $75 for completing the pre-screening visit, screening visit, Day 14, Day 28, and Day 56 visits. You will also receive $150 for completing both the Baseline and Day 2 visits. The total you could receive for completing the study visits is $675. You will receive this money in the form of a check mailed to your home on a quarterly basis (every 3 months).

In addition, you are eligible for mileage reimbursement for each study visit you complete that requires you to travel from your home to the clinical site at the following rates:

· Less than 25 miles round trip: $10.00
· 25-50 miles round trip: $20.00
· Greater than 50 miles round trip: $30.00

You will receive mileage reimbursement in the form of a check mailed to your home on a quarterly basis (every 3 months).

You may also be eligible for lodging reimbursement up to $175 per night if you and your caregiver are traveling more than one hour to the site for dosing visits and/or visits that require a lumbar puncture (LP) and pre-LP labs (Day prior to Day 1 visit, Day 1 and Day prior to Day 28 visit). This is for a maximum of up to 3 nights with an itemized receipt. You will receive reimbursement in the form of a check when you turn in your receipt.
Study duration and period
We expect that you will be in this research study for 56 days (8 weeks or a little less than 2 months).
Recruitment period
From Nov. 19, 2018
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Kelly Wallace
Research Topic
  • Huntington's Disease

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