Tominersen for Huntington's Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing a drug called tominersen to see if it is safe and effective for people in the early stages of Huntington's Disease. The drug aims to reduce a harmful protein that causes the disease, potentially slowing its progression. Tominersen targets and reduces the mutant huntingtin protein, which is implicated in Huntington's Disease.
Do I have to stop taking my current medications?
The trial requires you to stop taking anti-platelet or anticoagulant medications 14 days before screening and during the study, unless it's aspirin at 81 mg/day or less. Other medications are not specified, so check with the study team.
Will I have to stop taking my current medications?
The trial requires that you stop taking anti-platelet or anticoagulant medications (blood thinners) like aspirin (unless 81 mg/day or less), clopidogrel, and warfarin at least 14 days before starting the study. Other medications are not specifically mentioned, so it's best to discuss with the study team.
What safety data is available for tominersen in treating Huntington's Disease?
Safety data for tominersen, also known as IONIS-HTTRx, RG6042, RO7234292, ISIS-443139, and HTT ASO, has been gathered from multiple clinical studies. A population pharmacokinetic model was developed using data from 750 participants across five clinical studies, which included 6302 cerebrospinal fluid (CSF) and 5454 plasma samples. The studies identified significant covariates affecting tominersen pharmacokinetics, such as baseline total CSF protein, age, antidrug antibodies, body weight, and sex. This model helps guide dose selection for future trials, indicating a focus on understanding the drug's behavior in the body, which is crucial for assessing safety. Additionally, the GENERATION HD2 trial and other ongoing studies continue to evaluate tominersen's safety and efficacy.12345
What safety data exists for tominersen in humans?
Tominersen has been studied in several clinical trials for Huntington's disease, where it was administered to 750 participants. The studies focused on how the drug moves through the body and identified factors that affect its clearance, but they did not report specific safety concerns in the provided abstracts.12345
Is the drug tominersen a promising treatment for Huntington's Disease?
How is the drug tominersen unique for treating Huntington's disease?
Tominersen is unique because it is an antisense oligonucleotide (a type of genetic material) that is administered directly into the cerebrospinal fluid to target and reduce the levels of mutant huntingtin protein, which is the cause of Huntington's disease. This approach is different from other treatments that may focus on managing symptoms rather than addressing the underlying genetic cause.12356
What data supports the idea that the drug Tominersen for Huntington's Disease is an effective treatment?
The available research shows that Tominersen can lower the levels of a harmful protein in the fluid around the brain and spine in people with Huntington's Disease. This suggests it might help manage the disease. However, the research does not provide direct evidence of improvements in symptoms or compare it to other treatments. More studies are needed to confirm its effectiveness in treating Huntington's Disease.12578
What data supports the effectiveness of the drug tominersen for Huntington's disease?
Who Is on the Research Team?
Clinical Trials
Principal Investigator
Hoffmann-La Roche
Are You a Good Fit for This Trial?
This trial is for adults over 40 kg with a BMI of 18-32 who have Huntington's disease and a specific genetic mutation score. It's open to those in the early stages or just before symptoms start, but not to anyone who has used gene therapy, brain surgery, certain medications recently, or women who are pregnant or planning pregnancy.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive tominersen or placebo for 16 months to evaluate safety, biomarkers, and efficacy
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Placebo
- Tominersen
Find a Clinic Near You
Who Is Running the Clinical Trial?
Hoffmann-La Roche
Lead Sponsor
Dr. Levi Garraway
Hoffmann-La Roche
Chief Medical Officer since 2019
MD from the University of Basel
Dr. Thomas Schinecker
Hoffmann-La Roche
Chief Executive Officer since 2023
PhD in Molecular Biology from New York University