Study on the Safety and Effects of MBF-015 for Patients with Huntington’s Disease

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What is this study about?

This clinical trial is focused on studying Huntington’s disease, a genetic disorder that affects the brain and leads to the gradual loss of movement control, thinking ability, and emotional stability. The trial will test a new treatment called MBF-015, which is taken orally in the form of hard capsules. The purpose of the study is to evaluate the safety and tolerability of MBF-015 in patients with Huntington’s disease over a period of 28 days, with a follow-up until day 43.

Participants in the study will receive MBF-015 in addition to their standard care. The study will monitor how the body processes the medication and its effects on the disease. This includes checking the levels of MBF-015 in the blood and cerebrospinal fluid (CSF), which is the fluid surrounding the brain and spinal cord. The study will also look at changes in certain biological markers that can indicate the progression of the disease, as well as any changes in the participants’ ability to perform daily activities and their overall mental and physical health.

The trial will involve regular visits to the study center for assessments, including blood tests, physical examinations, and possibly MRI scans, which are imaging tests that help visualize the brain. Participants will be closely monitored for any side effects or changes in their condition throughout the study. The goal is to gather information that could lead to better treatment options for people living with Huntington’s disease.

1 beginning of the trial

Upon joining the study, the participant will begin taking the medication MBF-015 in the form of hard capsules. This medication is taken orally.

The primary goal is to assess the safety and tolerability of MBF-015 over a period of 28 days, with a follow-up extending to day 43.

2 medication administration

The participant will take MBF-015 daily for 28 days. The specific dosage and frequency will be provided by the study team.

The participant will continue their standard care for Huntington’s disease alongside the study medication.

3 monitoring and assessments

Throughout the study, the participant will undergo various assessments to monitor safety and effectiveness. These include physical examinations, laboratory tests, and vital sign measurements.

Additional assessments will include electrocardiograms (ECGs) and the Columbia-Suicide Severity Rating Scale (C-SSRS) to monitor any changes in health status.

4 pharmacokinetic and pharmacodynamic evaluations

Blood samples will be collected to measure the concentration of MBF-015 in plasma on days 1-2, day 28, and a single-point concentration on day 43.

Cerebrospinal fluid (CSF) samples will be collected on day 28 to measure the concentration of MBF-015.

5 biomarker and cognitive assessments

Changes in neuronal biomarkers, such as Nfl levels, will be measured in CSF and/or blood at day 28.

Cognitive assessments will be conducted to evaluate changes in motor and functional capacity, including tests like the UHDRS total motor score and the Stroop word reading test.

6 end of treatment and follow-up

The treatment phase concludes on day 28, followed by a final assessment on day 43 to evaluate any lasting effects of the medication.

The study will conclude with a comprehensive review of all collected data to determine the safety and tolerability of MBF-015.

Who Can Join the Study?

Must be a male or a nonpregnant, nonlactating female, aged between 25 and 60 years old.
Males and females who can have children must agree to use proper birth control during the study. Females must have a negative pregnancy test before starting the study and use birth control until 60 days after the last dose of the study drug. Females who cannot have children must have proof of this, like a past surgery or being postmenopausal (not having a period for at least 1 year).
Must have a specific genetic marker called CAG triplet repeats of 39 or more in the HTT gene. Must show certain movement symptoms of Huntington’s Disease, with a specific score on a test called UHDRS-TMS greater than 5 and a Diagnostic Confidence Score of 4.
Must have a score between 7 and 13 on a test called UHDRS Total Functional Capacity (TFC), with mild to moderate thinking difficulties, shown by a MoCA score between 10 and 25.
The study doctor must believe the patient can handle all study procedures and follow all study rules.
Must be able to have MRI scans and tolerate them, meaning no metal implants that are not safe for MRI, or severe movement issues that make MRI scans impossible.
Must be able to handle blood tests and a procedure called a lumbar puncture (LP).
If taking medication for Huntington’s Disease, the dose must be stable for at least 3 months before starting the study. Acceptable medications include certain types of drugs like Dopamine D2 receptor blockers, vesicular monoamine transporter 2 blockers, or GABA agonists.
Must be able to fully participate in all parts of the clinical trial, as judged by the study doctor. Must fully understand the study information and give written consent to participate.

Who Cannot Join the Study?

Participants who are not diagnosed with Huntington’s disease cannot join the study. Huntington’s disease is a condition that affects the brain and causes problems with movement, thinking, and behavior.
Participants who are under 18 years old cannot join the study.
Participants who are part of a vulnerable population, such as those who cannot make decisions for themselves, cannot join the study.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Hsijgdwy Ds Lj Sjgeg Cadh I Socn Pfp	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Spain	Not recruiting	20.09.2023

Trial locations

Investigated drugs:

Mbf-015

MBF-015 is an experimental medication being studied for its potential effects on patients with Huntington’s disease. The trial aims to evaluate how safe and tolerable this medication is when taken by mouth over a period of 28 days. Researchers are also looking at how the body processes the medication and its initial effectiveness in treating symptoms of Huntington’s disease. This study is conducted alongside the standard care that patients are already receiving.

Investigated diseases:

Huntington's disease

Huntington’s disease – Huntington’s disease is a genetic disorder that affects the brain, leading to the progressive breakdown of nerve cells. It typically manifests in adulthood and is characterized by a combination of movement disorders, cognitive decline, and psychiatric symptoms. Individuals with Huntington’s disease may experience involuntary jerking or writhing movements, known as chorea, as well as difficulties with coordination and balance. Cognitive symptoms can include problems with planning, organizing, and focusing, while psychiatric symptoms may involve depression, irritability, and mood swings. As the disease progresses, these symptoms become more pronounced, affecting daily functioning and quality of life. The progression of Huntington’s disease varies among individuals, but it generally leads to increasing disability over time.

Trial ID:

2023-505241-10-00

Protocol code:

MBF-015CT-02

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effects of MBF-015 for Patients with Huntington’s Disease

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