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Tominersen

Tominersen is an investigational drug being studied for its potential in treating Huntington’s Disease, a genetic disorder that affects movement, thinking, and behavior. This article explores the ongoing clinical trial, GENERATION HD2, which aims to assess the safety, effectiveness, and biological effects of Tominersen in people with early-stage Huntington’s Disease.

Table of Contents

What is Tominersen?

Tominersen is an investigational drug being studied as a potential treatment for Huntington’s Disease. It is currently undergoing clinical trials to evaluate its safety and effectiveness in patients with early stages of the disease.[1]

Target Condition: Huntington’s Disease

Huntington’s Disease is a genetic disorder that causes progressive brain damage. It affects movement, thinking, and emotions. The disease typically appears in adulthood and worsens over time. Tominersen is being tested in people with prodromal (early signs) and early manifest (beginning stages) Huntington’s Disease.[1]

The GENERATION HD2 Clinical Trial

The GENERATION HD2 study is a clinical trial designed to test Tominersen. Here are some key points about the trial:[1]

  • Phase II study: This means it’s testing the drug in a larger group of people after initial safety studies.
  • Randomized: Participants are randomly assigned to different groups.
  • Double-blind: Neither the participants nor the researchers know who is receiving the actual drug or a placebo.
  • Placebo-controlled: Some participants receive a placebo (a substance with no active ingredients) for comparison.
  • Dose-finding: The study is testing different doses of Tominersen to find the most effective and safe dose.

How is Tominersen Administered?

Tominersen is given through an intrathecal injection, which means it’s injected directly into the fluid surrounding the brain and spinal cord. The study is testing two different doses:[1]

  • 60 mg of Tominersen every 16 weeks
  • 100 mg of Tominersen every 16 weeks

Some participants will receive a placebo injection that looks the same but contains no active drug.

Safety Monitoring

The study closely monitors the safety of Tominersen. This includes:[1]

  • Tracking adverse events (side effects) and their severity
  • Monitoring changes in cerebrospinal fluid (the fluid around the brain and spinal cord), including white blood cell count and protein levels
  • Regular brain scans (MRI) to check for any changes or abnormalities
  • Assessing suicide risk using a tool called the C-SSRS (Columbia-Suicide Severity Rating Scale)

Efficacy Measures

To determine if Tominersen is effective, the study uses several tests and scales:[1]

  • Composite Unified Huntington’s Disease Rating Scale (cUHDRS): A combined measure of various Huntington’s Disease symptoms
  • Total Functional Capacity (TFC) Scale: Measures a person’s ability to perform daily activities
  • Montreal Cognitive Assessment (MoCA): A test of cognitive function
  • Symbol Digit Modalities Test (SDMT): Assesses processing speed and working memory
  • Stroop Word Reading (SWR) Test: Measures attention and cognitive flexibility
  • Total Motor Score (TMS): Evaluates movement-related symptoms

Biomarker Assessments

The study also looks at biological markers (biomarkers) to understand how Tominersen affects the disease at a molecular level:[1]

  • CSF mHTT protein levels: Measures the amount of mutant huntingtin protein, which is believed to cause Huntington’s Disease
  • CSF Neurofilament Light Chain (NfL) levels: An indicator of nerve cell damage
  • Anti-drug antibodies (ADAs): Checks if the body is developing an immune response to the drug
Aspect Details
Study Name GENERATION HD2
Drug Tominersen
Condition Huntington’s Disease (prodromal and early manifest)
Administration Intrathecal, every 16 weeks
Dosages 60 mg and 100 mg
Study Duration Approximately 24 months
Primary Outcomes Safety, CSF composition, brain structure changes, mHTT levels, functional capacity
Secondary Outcomes Cognitive function, motor skills, CSF NfL levels, anti-drug antibodies

FAQ

  • What is the purpose of the GENERATION HD2 study? The GENERATION HD2 study aims to evaluate the safety, biomarkers, and efficacy of Tominersen compared to a placebo in people with prodromal (early signs) and early manifest Huntington's Disease.
  • How is Tominersen administered in the trial? Tominersen is administered intrathecally (into the spinal fluid) every 16 weeks. The study includes two dosage groups: 60 mg and 100 mg of Tominersen.
  • What are the main outcomes being measured in the study? The study measures several outcomes, including the incidence and severity of side effects, changes in cerebrospinal fluid composition, brain structure changes through MRI, levels of mutant huntingtin protein, and changes in functional capacity and cognitive abilities.
  • How long does the GENERATION HD2 study last? The study lasts for approximately 24 months, with various assessments conducted at different time points throughout this period.
  • What is the placebo used in this study? The placebo used in this study is a substance that matches Tominersen in appearance but does not contain the active drug. It is also administered intrathecally every 16 weeks.

Ongoing Clinical Trials on Tominersen

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

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Austria Denmark France Germany Italy Poland +2

Glossary

  • Huntington's Disease: A genetic disorder that causes progressive brain damage, affecting movement, thinking, and behavior.
  • Tominersen: An investigational drug being studied for its potential in treating Huntington's Disease.
  • Intrathecal: A method of drug administration where the medication is injected directly into the spinal fluid.
  • Prodromal: The early stage of a disease when symptoms are just beginning to appear.
  • Biomarkers: Measurable indicators in the body that can show the presence or progress of a disease or the effects of treatment.
  • Placebo: A substance that looks like the real drug but contains no active ingredient, used to compare the effects of the actual drug.
  • MRI: Magnetic Resonance Imaging, a technique used to create detailed images of the body's internal structures.
  • Cerebrospinal fluid (CSF): The clear liquid that surrounds the brain and spinal cord, protecting them and helping to remove waste products.
  • Mutant huntingtin protein (mHTT): The abnormal protein produced in Huntington's Disease that causes damage to brain cells.
  • Neurofilament light Chain (NfL): A protein released when nerve cells are damaged, used as a marker of neurological disease progression.

References

  1. https://clinicaltrials.gov/study/NCT05686551