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VOTOPLAM

Clinical trials are investigating VOTOPLAM in people with Huntington’s disease. The study is looking at long-term safety and whether the treatment lowers a blood marker linked to the disease. It is a Phase 2 trial in adult participants.

Table of Contents

Trial overview

The available clinical trial is a Phase 2b, double-blind, randomized extension study of VOTOPLAM in people with Huntington’s disease.[1] The study is designed to evaluate long-term safety and efficacy, with a focus on blood total huntingtin (tHTT) levels.[1]

Study design and phase

This is an interventional study, which means participants receive a study treatment and researchers observe the results.[1] It is described as double-blind, meaning participants and study staff do not know which treatment is being given during the blinded part of the study.[1]

The trial is listed as Phase 2 and also described in the title as Phase 2b, which is a later part of Phase 2 research.[1] Phase 2 studies are usually done to look more closely at safety and early signs that a treatment may help.[1]

Who can participate

The trial is for participants with Huntington’s disease.[1] The source data do not give more details about age limits, disease stage, or other entry rules, so those cannot be confirmed here.[1]

What is measured in the study

The main safety measure is the safety profile of VOTOPLAM, which is checked through treatment-emergent adverse events (new or worsening health problems during the study), laboratory abnormalities, vital signs, physical examinations, and the C-SSRS.[1] The C-SSRS is a screening tool used to check for suicidal thoughts or behavior.[1]

The study also measures blood total huntingtin (tHTT) levels over time.[1] This is a pharmacodynamic measure, meaning it helps show whether the treatment is changing a disease-related marker in the body.[1]

The brief summary says the study aims to evaluate long-term safety and the reduction of blood tHTT levels, which is the main sign of possible efficacy in the source data.[1]

Trial status and size

The trial status is listed as Authorised, meaning it has been approved to proceed.[1] The planned enrollment is 250 participants.[1]

The interventions listed include VOTOPLAM tablets and placebo tablets, which shows that some participants may receive an inactive look-alike treatment for comparison.[1] The source data do not provide the full treatment schedule or the exact randomization groups, so those details are not described here.[1]

Trial ID Phase Condition studied Status Enrollment
NCT06254482 Phase 2 Huntington’s disease Authorised 250

FAQ

  • What is being studied in the VOTOPLAM trial? The trial is studying the long-term safety of VOTOPLAM and whether it can lower blood total huntingtin (tHTT) levels. tHTT is a protein linked to Huntington’s disease.
  • Who can take part in this trial? The trial is for participants with Huntington’s disease. The data provided do not list more detailed entry rules.
  • What phase is the VOTOPLAM study? This is a Phase 2 trial. Phase 2 studies usually look more closely at safety and early signs that a treatment may help.
  • What does the study measure? The main measures are safety and blood tHTT levels over time. Safety is checked with side effects, lab tests, vital signs, physical exams, and a suicide risk scale called C-SSRS.
  • Is the study ongoing? The trial status is listed as Authorised. This means the study has been approved to move forward.

Ongoing Clinical Trials on VOTOPLAM

  • Study on Long-Term Safety and Effects of PTC518 for Patients with Huntington’s Disease

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Austria France Germany Italy The Netherlands Spain

Glossary

  • Huntington’s disease: A brain disease that slowly affects movement, thinking, and mood.
  • Phase 2: A mid-stage clinical trial that looks at safety and early signs of benefit in a group of participants.
  • Randomized: Participants are assigned by chance to different study groups.
  • Double-blind: Neither the participant nor the study team knows who is getting the active treatment or placebo during the study period.
  • Extension study: A follow-up study that continues after an earlier trial to collect longer-term data.
  • Placebo: A look-alike treatment with no active study drug.
  • Safety profile: A summary of how safe a treatment appears, based on side effects and test results.
  • TEAEs: Treatment-emergent adverse events, meaning health problems that appear or get worse during the study.
  • Laboratory abnormalities: Test results that are outside the normal range.
  • Vital signs: Basic body measurements such as blood pressure, pulse, and temperature.
  • C-SSRS: A questionnaire used to check for suicidal thoughts or behavior.
  • tHTT: Total huntingtin, a blood protein being measured in the study as a marker of disease-related change.

References