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Study on the Safety and Efficacy of Tominersen for Patients with Prodromal and Early Manifest Huntington's Disease

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical study focuses on examining the safety and effectiveness of a medication called tominersen for people in the early stages of Huntington's disease, a condition that affects the brain and can lead to movement, thinking, and psychiatric problems. The study includes individuals who are either in the very early phases (prodromal) or have just started showing symptoms (early manifest) of Huntington's disease.

The purpose of the study is to assess how safely and effectively tominersen, administered as a solution for injection, works in comparison with a placebo. The study is designed to understand if tominersen can help reduce certain signs related to Huntington's disease that are measured by changes in biomarker levels and overall patient ability to perform daily activities. Participants will undergo evaluations using clinical tests and procedures such as MRI to track changes over time.

Tominersen belongs to a group known as antisense oligonucleotides, which are designed to target and reduce specific proteins in the body, like the mutant huntingtin protein in this case. Throughout the trial, researchers will measure various aspects, including safety indicators and biomarker levels, to determine if tominersen provides any benefits for individuals with early signs of Huntington's disease.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying the presence of the Huntington's disease gene expansion mutation and other health criteria.

    The assessment ensures that the participant meets the age, weight, and health requirements specified for the study.

  2. Step 2

    Randomization and treatment assignment

    Participants are randomly assigned to receive either the active medication, tominersen, or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

    The medication is administered as a solution for injection directly into the intrathecal space, which is the area surrounding the spinal cord.

  3. Step 3

    Treatment administration

    The treatment involves regular injections of tominersen or placebo. The frequency and dosage are determined by the study protocol and are designed to evaluate the safety and efficacy of the treatment.

    Participants will receive these injections over a period of 16 months, with specific intervals as outlined in the study schedule.

  4. Step 4

    Monitoring and assessments

    Throughout the study, participants undergo regular monitoring to assess the safety and effects of the treatment. This includes clinical laboratory tests, magnetic resonance imaging (MRI), and evaluations of cerebrospinal fluid (CSF) protein levels.

    Additional assessments include changes in vital signs, electrocardiogram (ECG) parameters, and cognitive function tests.

  5. Step 5

    Final evaluation

    At the end of the 16-month treatment period, a final evaluation is conducted to assess the overall impact of the treatment on the participant's condition.

    This includes a comprehensive review of all collected data, including any changes in the participant's functional capacity and any adverse events experienced during the study.

Who can join the trial?

6 criteria

  • You must have a specific change in your genes related to Huntington's disease. This is confirmed by a CAP score between 400 and 500.
  • You should either be in the early stages of Huntington's disease, known as prodromal HD, or in the early noticeable stages, known as early manifest HD. This is determined by specific scales that measure your independence and ability to function.
  • Your kidney function should be at a certain level, measured by something called the estimated glomerular filtration rate, which should be at least 60 mL/min/1.73 m² in at least one of two tests.
  • Your total body weight should be more than 40 kg, and your body mass index (BMI) should be between 18 and 32.
  • You need to be between 25 and 50 years old when you agree to participate in the study.
  • You must have a study companion, someone who can support you during the study.

Who cannot join the trial?

8 criteria

  • Patients with any other serious medical condition that might interfere with the study.
  • Patients who have had a recent surgery or are planning to have surgery during the study period.
  • Patients who are currently participating in another clinical trial.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a known allergy to the study medication or its ingredients.
  • Patients who have a significant mental health disorder that could affect their ability to participate.
  • Patients who are unable to comply with the study procedures or follow-up visits.
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Investigated drugs

Tominersen is a medication being studied for its potential to treat individuals with prodromal and early manifest Huntington's Disease. The trial aims to assess the safety of tominersen by monitoring adverse events and changes in clinical laboratory results, such as cerebrospinal fluid white blood cell and protein levels. Additionally, the study evaluates the medication's effect on mutant huntingtin protein levels in the cerebrospinal fluid and its efficacy in improving symptoms and functional capacity in patients over a period of 16 months.

What is already known about the treatment

Tominersen – Tominersen is administered through an injection into the spinal canal, a method known as intrathecal administration. It is currently being studied in clinical trials for its potential use in treating prodromal and early manifest Huntington's disease. The medication works by reducing the levels of mutant huntingtin protein, which is believed to play a role in the progression of Huntington's disease. Tominersen is classified as an antisense oligonucleotide, a type of drug designed to target specific genetic material.

Investigated diseases

Huntington's Disease – Huntington's Disease is a genetic disorder that affects the brain, leading to the progressive breakdown of nerve cells. It typically begins with subtle changes in mood, cognition, and movement, which gradually worsen over time. As the disease progresses, individuals may experience involuntary movements, difficulty with coordination, and cognitive decline. Early stages might include irritability, depression, and small involuntary movements. Over time, these symptoms become more pronounced, affecting daily activities and quality of life. The disease is caused by a genetic mutation and is inherited in an autosomal dominant pattern.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-503928-10-00Protocol codeBN42489Estimated enrolment300 patientsSponsorF. Hoffmann-La Roche AG

sourced from the EU Clinical Trials Register and site verification

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