IRB00358575, PI: Dr. Jee Bang

Clinical Trial: Phase II GENERATION HD2 of Tominersen in Participants With Prodromal and Early Manifest Huntington's Disease

In this clinical trial, researchers in 15 countries are evaluating an investigational drug, Tominersen, designed to target mutant huntingtin (mHTT) – the build-up of this protein is found in the fluid surrounding the brain and spinal cord, and causes Huntington’s disease (HD) symptoms.

The goal of this study is to evaluate the safety, biomarkers and efficacy trends of different dose levels of investigational drug Tominersen compared with placebo in people aged 25 to 50 with prodromal (very early subtle signs of HD) or early manifest HD.

Who is eligible for the study?

For this study, you may be eligible if you:

• Are between 25 and 50 years old (at the start of the study).
• Have a CAP score of 400 to 500. A CAP score is a number which is calculated to provide a measurement of your HD progression. The study team can provide more information about this measure if you have not come across it before.
• Have a formal diagnosis of early Huntington’s disease or who carry the abnormal huntingtin gene and are starting to show very early, subtle signs of HD, which may only be apparent during a detailed examination by a physician.

View additional inclusion and exclusion criteria for eligibility

What will the study involve?

• At least 22 months: If you take part in GENERATION HD2, you will be involved for a minimum of 22 months. This will include a 1 month screening period, a minimum of 16 months in the treatment period (where you will receive either the investigational drug or placebo), and one follow-up appointment 5 months after the treatment period ends. You will need to come into the clinic approximately once every 4 months and you will also receive a telephone call in-between each clinic visit so that we can monitor your health.
• Study companion: To take part in the study, you will need a study companion to support you through GENERATION HD2. Your study companion will need to attend clinic visits with you in-person for screening, at the start of your treatment period and in month 16 (or if you leave the study early) and be available for telephone calls at other timepoints during the study.
• Extension study: If the main study is successful, you may also be able to take part in an extension study where all participants will receive the investigational drug.
• Please note that participation is entirely voluntary, and you can leave at any time without affecting your usual healthcare.

To learn more visit ClinicalTrials.gov or contact the Huntington's Disease Center at Johns Hopkins:
Etta Bernard, Sr. Research Program Coordinator
[email protected]
410-614-7671