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Mbf-015

Clinical trials are studying Mbf-015 in people with Huntington’s disease. The main goal is to check safety, tolerability, and early signs of benefit, as well as how the body handles the treatment. The trial data here describe adults with Huntington’s disease in a phase 2 study.

Table of contents

Trial overview

The available study investigated Mbf-015 in people with Huntington’s disease.[1] It was a phase 2, open-label, single-centre trial that enrolled 10 participants and was completed.[1]

Who participated

The trial included participants with Huntington’s disease, and the brief summary says the treatment was studied on top of standard of care.[1] This means people were not being taken off their usual care just to join the study.[1]

Study design and phase

This was an interventional study, which means the researchers gave the treatment and then watched what happened.[1] It was open label, so the study team and participants knew the treatment being used.[1] It was also single-centre, meaning it took place at one research site only.[1]

The study was in phase 2, a stage that usually looks closely at safety and early signs of benefit after initial testing.[1] The treatment was given orally, and the study period lasted 28 days with follow-up to day 43.[1]

What was measured

The primary endpoint was safety and tolerability of Mbf-015 from baseline to the end of follow-up at day 43.[1] In simple terms, the study wanted to see whether the treatment could be used without causing major problems and how well people could handle it.[1]

The study also tracked the number and severity of adverse events, which are unwanted medical problems that happen during a trial.[1] Other safety checks included changes in vital signs, physical examination findings, laboratory measurements, ECGs, and the Columbia-Suicide Severity Rating Scale (C-SSRS).[1]

What the trial was designed to learn

Besides safety, the study aimed to assess pharmacokinetics, pharmacodynamics, and preliminary efficacy.[1] Pharmacokinetics means how the body handles a treatment, while pharmacodynamics means what the treatment does in the body.[1] Preliminary efficacy means early signs that the treatment may help, but not proof of benefit yet.[1]

Because this was a small phase 2 study, the main value of the trial was to build early knowledge about Mbf-015 in Huntington’s disease rather than to give a final answer about effectiveness.[1]

References

[1] 2023-505241-10-00. A phase IIa, open label, single centre study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of orally dosed MBF-015 in Huntington’s disease patients.

Trial IDPhaseCondition studiedStatusEnrollment
2023-505241-10-00Phase 2Huntington’s diseaseCompleted10

FAQ

  • What is being studied in the Mbf-015 trial? The trial is studying Mbf-015 in people with Huntington’s disease. It looks at safety, tolerability, and early signs of benefit.
  • Who took part in the study? The study included participants with Huntington’s disease. It was a small single-centre trial with 10 enrolled participants.
  • What phase was the Mbf-015 trial? This was a phase 2 study. Phase 2 trials usually look more closely at safety and whether a treatment may help.
  • What did the study measure? The main measure was safety and tolerability over 28 days. The study also tracked side effects, vital signs, physical exams, lab tests, ECGs, and suicide risk screening.
  • What was the status of the trial? The trial was completed. It followed participants for 28 days, with follow-up until day 43.

Igangværende kliniske forsøg for Mbf-015

  • Test af ny medicin (MBF-015) til behandling af Huntingtons sygdom: Undersøgelse af sikkerhed og virkning

    Rekrutterer ikke

    1 1
    Undersøgte sygdomme:
    Undersøgte lægemidler:
    Spanien

Ordliste

  • Huntington’s disease: A brain disorder that gets worse over time and can affect movement, thinking, and mood.
  • Phase 2: A trial stage that checks safety more closely and looks for early signs that a treatment may work.
  • Open label: A study where both the researchers and the participants know which treatment is being given.
  • Single-centre: A study done at one hospital or research site only.
  • Safety: How well a treatment can be used without causing serious harm.
  • Tolerability: How well people can handle a treatment and its effects.
  • Pharmacokinetics: How the body absorbs, moves, and removes a treatment.
  • Pharmacodynamics: What the treatment does in the body and what effects it has.
  • Efficacy: How well a treatment works.
  • Adverse events: Unwanted medical problems that happen during a study.
  • ECG: A test that records the heart’s electrical activity.
  • C-SSRS: A questionnaire used to check for suicidal thoughts and behavior.

Referencer

  1. https://clinicaltrials.gov/study/2023-505241-10-00