NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease

NCT05686551

Active Not RecruitingPhase 2

Sponsor: Hoffmann-La Roche

Conditions: Huntington Disease

Interventions: Tominersen, Placebo

Countries: United States, Argentina, Australia, Austria, Canada, Denmark, France, Germany

This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).

Eligibility overview

Sex: ALL

Age: 25 Years to 50 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

Inclusion Criteria:

DB Period:

* HD gene expansion mutation carrier status with a cytosine-adenine-guanine-age product (CAP) score of 400-500 inclusive
* Either:

  * Prodromal HD (defined as Diagnostic Confidence Level (DCL) 2 to 3, Independence Scale (IS) ≥70, and TFC ≥8); Or
  * Early manifest HD (defined as DCL 4, IS ≥70, and TFC ≥8)
* Total body weight \> 40 kilograms (kg) and a body mass index (BMI) within the range of 18-32 kilograms per meter square (kg/m\^2)
* Study companion

OLE Period:

* Participants must have completed the DB treatment period
* Participants must remain in the DB Safety follow-up period until OLE period starts

Exclusion Criteria:

DB Period:

* Current or previous use of an antisense oligonucleotide (ASO) (including small interfering ribonucleic acid \[RNA\]) or any HTT lowering therapy (including tominersen)
* Anti-platelet or anticoagulant therapy within 14 days prior to screening or anticipated use during the study, including, but not limited to, aspirin (unless ≤ 81 milligrams per day \[mg/day\]), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, apixaban, and heparin
* History of gene therapy, cell transplantation, or brain surgery
* Hydrocephalus
* Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug
* History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening

OLE Period:

* Early discontinuation from the DB treatment and the safety follow-up (SFU) periods
* Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required
* Current or previous use of an ASO other than tominersen (including small interfering RNA) or any other HTT-lowering therapy
* Hydrocephalus
* Received any active investigational treatment other than tominersen during or since completion of the DB treatment period

Key inclusions/exclusion criteria are listed here. Other protocol-defined I/E criteria may apply.

Locations (70)

Birmingham, Alabama, United States
Phoenix, Arizona, United States
Sacramento, California, United States
Englewood, Colorado, United States
Washington D.C., District of Columbia, United States
Gainesville, Florida, United States
Tampa, Florida, United States
Chicago, Illinois, United States
Iowa City, Iowa, United States
Baltimore, Maryland, United States
Boston, Massachusetts, United States
Detroit, Michigan, United States
Amherst, New York, United States
Pittsburgh, Pennsylvania, United States
Nashville, Tennessee, United States
Houston, Texas, United States
Kirkland, Washington, United States
Spokane, Washington, United States
Buenos Aires, Argentina
CABA, Argentina
Capital Federal, Argentina
Ciudad Autonoma Buenos Aires, Argentina
Clayton, Victoria, Australia
Parkville, Victoria, Australia
Nedlands, Western Australia, Australia
Innsbruck, Austria
Edmonton, Alberta, Canada
Montreal, Quebec, Canada
København Ø, Denmark
Angers, France
+ 40 more on CT.gov