← Back to Search

Tominersen for Huntington's Disease

Phase 2
Recruiting
Research Sponsored by Hoffmann-La Roche
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2
Huntington's disease (HD) gene expansion mutation carrier status with a CAP score of 400-500 inclusive
Must not have
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 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, apixaban, and heparin
Current or previous use of an ASO (including small interfering RNA) or any HTT lowering therapy (including tominersen)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 16 months

Summary

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.

Who is the study for?
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.
What is being tested?
The study tests Tominersen (at two different doses: 60 mg and 100 mg) against a placebo to check its safety and effectiveness in slowing down Huntington's Disease progression. Participants will be randomly assigned to receive either the drug or placebo.
What are the potential side effects?
While the side effects aren't specified here, similar drugs can cause reactions at injection sites, flu-like symptoms, potential liver issues, nervous system problems like dizziness or seizures, and could affect blood clotting.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I weigh more than 40 kg and my BMI is between 18-32.
Select...
I carry the Huntington's disease gene with a CAP score between 400-500.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I haven't taken any blood thinners, except possibly low-dose aspirin, in the last 14 days.
Select...
I have used gene-silencing drugs for my condition.
Select...
I have had gene therapy, cell transplantation, or brain surgery.
Select...
I have hydrocephalus.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 16 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 16 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from baseline in Total Functional Capacity (TFC) Scores (U.S. sites) at 16 months
Change from baseline in composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores (non-U.S. sites) at 16 months

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: Tominersen 60 mgExperimental Treatment1 Intervention
Group II: Tominersen 100 mgExperimental Treatment1 Intervention
Group III: PlaceboPlacebo Group1 Intervention

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Huntington's Disease (HD) include VMAT2 inhibitors like tetrabenazine, deutetrabenazine, and valbenazine, which reduce chorea by depleting monoamines such as dopamine. This is significant for HD patients as it helps manage motor symptoms that impair daily functioning. Additionally, antisense oligonucleotides (ASOs) like tominersen target the mutant huntingtin (mHTT) protein by binding to its mRNA, thereby reducing its production. This mechanism is particularly important as it aims to lower the levels of the toxic protein directly involved in HD progression, offering a potential disease-modifying approach.

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor
2,450 Previous Clinical Trials
1,095,244 Total Patients Enrolled

Media Library

Placebo Clinical Trial Eligibility Overview. Trial Name: NCT05686551 — Phase 2
Huntington's Disease Research Study Groups: Tominersen 100 mg, Tominersen 60 mg, Placebo
Huntington's Disease Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT05686551 — Phase 2
Placebo 2023 Treatment Timeline for Medical Study. Trial Name: NCT05686551 — Phase 2
~133 spots leftby Feb 2026