Huntington DiseasePhase 2NCT06469853
Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of MBF-015 in Huntington's Disease Patients
A Phase 2 study testing MBF-015 16 mg oral capsules in people with Huntington Disease, run by Medibiofarma S.L. at 1 site in Spain.
Trial summary
This is a Phase IIa (proof of concept), single center clinical trial to evaluate the safety and efficacy of daily MBF-015 oral treatment during 28 days in Huntington's Disease patients on top of standard of care. Read more about this study
- Who is it for?
- Ages 25 to 60 years
- Conditions the study is for
- Huntington Disease
- What is being tested?
- MBF-015 16 mg oral capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteSpain
- Study dates
- 1 Feb 2024to 4 Dec 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 25 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Ambulatory male or nonpregnant, nonlactating females, age ≥25 to ≤60 years old.
- Males and females of childbearing potential must agree to use adequate birth control measures during the study. Females of childbearing potential must have a negative serum pregnancy test prior to Visit 2 and either be sexually abstinent or must use a hormonal (oral, implantable, or injectable) or double barrier method of birth control throughout the study, and until 60 days after the last dose of study drug. Females unable to bear children must have documentation of such in the source records (i.e., tubal ligation, hysterectomy, or postmenopausal [defined as a minimum of 1 year since the last menstrual period]).
- Documented CAG triplet repeats ≥39 in the HTT gene. Clinical diagnostic motor features of HD, defined as UHDRS-TMS > 5 with Diagnostic Confidence Score = 4
- UHDRS Total Functional Capacity (TFC) scores ≥7 and ≤13 with moderate cognitive impairment based on clinical assessment.
- In the opinion of the Investigator, the patient can tolerate all study procedures and is willing to comply with all other protocol requirements.
- Able to undergo MRI scans and able to tolerate them (e.g., no metal implants including MRI incompatible IUDs, chorea of a severity that precludes MRI scans or any condition that renders testing intolerable for the patient.
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- Able to tolerate blood draws and lumbar puncture (LP).
- If participants are using a treatment for HD, they should be on a stable dose for at least 3 months prior to study commencement. Acceptable treatments include Dopamine D2 receptor blockers or reverse agonists, vesicular monoamine transporter 2 blockers or γ-aminobutyric acid (GABA) agonists.
- Ability to participate fully, in the opinion of the Investigator, in all aspects of this clinical trial. Full comprehension of consent language and written informed consent must be obtained from the participant and documented.
Who cannot join
- Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.
- Clinically significant laboratory abnormality at Screening.
- Clinically significant abnormality at Screening electrocardiogram (ECG), including but not necessarily limited to a confirmed QT interval corrected for heart rate (QTc) ≥450 msec for males or ≥470 msec for females. Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the opinion of the Investigator, precludes the patient's safe participation in the study or would interfere with the study assessments. Mental status, psychiatric medical history, and eligibility for the study must be documented in the screening questionnaire.
- Pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening Visit, or plans to become pregnant during the course of the study.
- Deemed to be at significant risk for suicidal behaviour based on any the following criteria:
- The opinion of the Investigator
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- Answers "yes" to Actual Suicide Attempts or Suicidal Behaviors in the Suicidal Behaviors section of the Columbia-Suicide Severity Rating Scale (C-SSRS) with reference to a 2-year period prior to the Screening Visit
- Answers "yes" on any items in the Suicidal Ideation section of the C-SSRS with reference to a 6-month period prior to the Screening Visit.
- Answers "yes" on any items in the Suicidal Ideation section of the C-SSRS at the Baseline Visit since the last visit (Screening Visit).
- Positive for Hepatitis B virus (HBV) or Hepatitis C virus (HCV).
- Known to be positive for human immunodeficiency virus (HIV).
- Evidence of Clostridioides difficile toxin or treatment for C. difficile infection, or other intestinal bacterial pathogen, within 30 days prior to Screening.Any condition that increases risk of meningitis unless patient is receiving appropriate prophylactic treatment.
- A medical history of brain or spinal disease that would interfere with lumbar puncture, CSF circulation or safety assessment. History of post-lumbar-puncture headache of moderate or severe intensity and/or blood patch.
- Contraindication for MRI (claustrophobia, pacemaker, aneurism clips, cardiac mechanical valve).
- Contraindication for lumbar puncture (anticoagulation, coagulation disease): Must not be taking anticoagulant treatment.
- Concurrent or previous participation in another clinical trial and received investigational therapy within 4 weeks or 5 half-lives (whichever is longer) prior to Screening.
- Any major surgery, in the investigator's opinion, performed within 8 weeks prior to randomization or planned during the study (i.e., any surgical procedure requiring general anesthesia).
- Unwillingness to withhold protocol-prohibited medications during the trial.
- Started or changed dose for concomitant medication for the treatment of HD symptoms or psychiatric disorders within 30 days prior to the Screening Visit (concomitant medications that have been administered on a stable regimen for ≥30 days are permitted).
- History of excessive alcohol or drug abuse that, in the opinion of the investigator, may interfere with the participant's ability to comply with the study procedures.
- Positive for opioids (unprescribed), cannabinoids, cocaine, amphetamines, methadone, barbiturates, methamphetamine, or phencyclidine at the Screening Visit.
- Known or suspected allergy, anaphylaxis, hypersensitivity or intolerance to the study drug excipients.
- Prior enrolment in the current study and had received study treatment.
- The participant is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in conduct of this study (e.g., spouse, parent, child, sibling).
Trial locations and contacts
1 site in 1 country.
Spain1 site
Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Barcelona, Barcelona, 08025
About this study
Official title: 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
This is a Phase IIa (proof of concept), single center clinical trial to evaluate the safety and efficacy of daily MBF-015 oral treatment during 28 days in Huntington's Disease patients on top of standard of care.
Read the detailed description
This is a Phase 2a study to evaluate the safety, tolerability and exploratory efficacy of daily oral treatment with either 16 mg or 32 mg MBF-015 during 28 days in Huntington's disease patients with cognitive impairment, in order to provide reliable safety, tolerability, and PK data that will guide drug dose and therapeutic regimen choices in subsequent clinical studies.
The study design is a single-center, open-label group consisting of two cohorts, one dosed with 16 mg MBF-015 daily for 28 days and one dosed with 32 mg MBF-015 daily for 28 days, in approximately 5 subjects per cohort without placebo control. No randomisation is required.
For each subject, the study duration will last a total of approximately 7-9 weeks consisting of a screening period of 1-3 weeks, a treatment period of 4 weeks and a follow-up period of 2 weeks.
- Conditions
- Huntington Disease
- Being tested
- MBF-015 16 mg oral capsules
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MBF-015 16 mg oral capsulesDrug
MBF-015 oral capsules HDAC inhibitor
| Study group | Role | What they receive |
|---|---|---|
| MBF-015 16 mg oral multiple dose | Experimental | Drug: MBF-015 16 mg oral capsules. Single daily dose. One hard gelatin capsule during 28 days. Receives: Drug: MBF-015 16 mg oral capsules |
| MBF-015 32 mg oral multiple dose | Experimental | Drug: MBF-015 16 mg oral capsules. Single daily dose. Two hard gelatin capsules during 28 days. Receives: Drug: MBF-015 16 mg oral capsules |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Evaluate safety and tolerability of MBF-015 in participants with Huntington's Disease (HD) on top of standard of care over 28 days, with follow-up to day 43.
Measured: 43 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2024
- Study start1 Feb 2024
- Primary completion4 Dec 2024
- Study completion4 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medibiofarma S.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06469853
- Sponsor's protocol ID
- MBF-015CT-02
- Status last verified
- Feb 2025
- First submitted
- 17 Jun 2024
- Last updated
- 3 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06469853
- Acronym / sponsor ID
- MBF-015CT-02
- Conditions
- Huntington Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Medibiofarma S.L.Industry (pharma, biotech, devices)
- Study start
- 1 Feb 2024 (actual)
- Primary completion
- 4 Dec 2024 (actual)
- Study completion
- 4 Dec 2024 (actual)
- First posted
- 24 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase IIa (proof of concept), single center clinical trial to evaluate the safety and efficacy of daily MBF-015 oral treatment during 28 days in Huntington's Disease patients on top of standard of care.
Detailed description
This is a Phase 2a study to evaluate the safety, tolerability and exploratory efficacy of daily oral treatment with either 16 mg or 32 mg MBF-015 during 28 days in Huntington's disease patients with cognitive impairment, in order to provide reliable safety, tolerability, and PK data that will guide drug dose and therapeutic regimen choices in subsequent clinical studies.
The study design is a single-center, open-label group consisting of two cohorts, one dosed with 16 mg MBF-015 daily for 28 days and one dosed with 32 mg MBF-015 daily for 28 days, in approximately 5 subjects per cohort without placebo control. No randomisation is required.
For each subject, the study duration will last a total of approximately 7-9 weeks consisting of a screening period of 1-3 weeks, a treatment period of 4 weeks and a follow-up period of 2 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MBF-015 16 mg oral multiple dose | Experimental | Drug: MBF-015 16 mg oral capsulesDrug: MBF-015 16 mg oral capsules. Single daily dose. One hard gelatin capsule during 28 days. |
| MBF-015 32 mg oral multiple dose | Experimental | Drug: MBF-015 16 mg oral capsulesDrug: MBF-015 16 mg oral capsules. Single daily dose. Two hard gelatin capsules during 28 days. |
Who may qualify
- Ages
- 25 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Ambulatory male or nonpregnant, nonlactating females, age ≥25 to ≤60 years old.
- Males and females of childbearing potential must agree to use adequate birth control measures during the study. Females of childbearing potential must have a negative serum pregnancy test prior to Visit 2 and either be sexually abstinent or must use a hormonal (oral, implantable, or injectable) or double barrier method of birth control throughout the study, and until 60 days after the last dose of study drug. Females unable to bear children must have documentation of such in the source records (i.e., tubal ligation, hysterectomy, or postmenopausal [defined as a minimum of 1 year since the last menstrual period]).
- Documented CAG triplet repeats ≥39 in the HTT gene. Clinical diagnostic motor features of HD, defined as UHDRS-TMS > 5 with Diagnostic Confidence Score = 4
- UHDRS Total Functional Capacity (TFC) scores ≥7 and ≤13 with moderate cognitive impairment based on clinical assessment.
- In the opinion of the Investigator, the patient can tolerate all study procedures and is willing to comply with all other protocol requirements.
Key exclusion criteria (24 listed)
- Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.
- Clinically significant laboratory abnormality at Screening.
- Clinically significant abnormality at Screening electrocardiogram (ECG), including but not necessarily limited to a confirmed QT interval corrected for heart rate (QTc) ≥450 msec for males or ≥470 msec for females. Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the opinion of the Investigator, precludes the patient's safe participation in the study or would interfere with the study assessments. Mental status, psychiatric medical history, and eligibility for the study must be documented in the screening questionnaire.
- Pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening Visit, or plans to become pregnant during the course of the study.
- Deemed to be at significant risk for suicidal behaviour based on any the following criteria:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MBF-015 16 mg oral capsulesMBF-015 oral capsules HDAC inhibitor | Drug | MBF-015 16 mg oral multiple dose, MBF-015 32 mg oral multiple dose |
Primary outcome
Adverse Events
Time frame: 43 days
Evaluate safety and tolerability of MBF-015 in participants with Huntington's Disease (HD) on top of standard of care over 28 days, with follow-up to day 43.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MBF-015 16 mg oral capsules in people with Huntington Disease, run by Medibiofarma S.L. at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: 43 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2025.