CLINICAL TRIAL

Branaplam for Huntington Disease

Stage I
Recruiting · 18+ · All Sexes · Budapest, Hungary

A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease

See full description

About the trial for Huntington Disease

Eligible Conditions
Huntington Disease · Early Manifest Huntington Disease

Treatment Groups

This trial involves 4 different treatments. Branaplam is the primary treatment being studied. Participants will be divided into 3 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

Experimental Group 1
Branaplam
DRUG
Experimental Group 2
Branaplam
DRUG
Experimental Group 3
Branaplam
DRUG
Show More

Eligibility

This trial is for patients born any sex aged 18 and older. There are 4 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Signed informed consent must be obtained prior to participation in the study.
Clinically diagnosed Stage 1 or 2 Huntington's disease with a diagnostic confidence level (DCL) = 4 and a United Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity (TFC) >8 at screening.
Genetically confirmed Huntington's disease, with presence of ≥40 cytosine-adenineguanine (CAG) repeats in the huntingtin gene.
Male and female participants between 25 to 75 years of age, inclusive, on the day of Informed Consent signature.
View All
Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
Similar Trials

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline up to approximately 2 years
Screening: ~3 weeks
Treatment: Varies
Reporting: Baseline up to approximately 2 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline up to approximately 2 years.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Branaplam will improve 2 primary outcomes and 19 secondary outcomes in patients with Huntington Disease. Measurement will happen over the course of Baseline up to Week 17.

Reduction (%) of mHTT protein in cerebrospinal fluid (CSF)
BASELINE UP TO WEEK 17
To assess the dose-response relationship of branaplam administered over 16 weeks on mHTT protein change from baseline in cerebrospinal fluid (CSF) obtained via lumbar puncture. Treatment is administered for 16 weeks and assessments are done over a 17 week period.
BASELINE UP TO WEEK 17
Change from baseline in Ventricular Volume, Caudate Volume and Total Brain Volume
BASELINE UP TO APPROXIMATELY 2 YEARS
Ventricular, Caudate and Total Brain Volume will be measured by structural magnetic resonance imaging (MRI)
BASELINE UP TO APPROXIMATELY 2 YEARS
Concentrations of total HTT and mHTT protein in CSF
BASELINE UP TO APPROXIMATELY 2 YEARS
The concentrations of total HTT and mHTT protein will be measured in CSF samples obtained via lumbar puncture
BASELINE UP TO APPROXIMATELY 2 YEARS
Concentration ratio of branaplam in CSF/plasma.
BASELINE UP TO APPROXIMATELY 2 YEARS
Concentration ratio will be calculated by measuring branaplam in CSF samples collected via lumbar puncture and blood samples
BASELINE UP TO APPROXIMATELY 2 YEARS
Concentrations of total HTT and mHTT protein in peripheral blood mononuclear cells (PBMCs)
BASELINE UP TO APPROXIMATELY 2 YEARS
The concentrations of total HTT and mHTT protein will be measured in PBMCs from blood samples.
BASELINE UP TO APPROXIMATELY 2 YEARS
Time taken to reach Cmx (Tmax) of branaplam in plasma.
BASELINE UP TO APPROXIMATELY 2 YEARS
Tmax will be calculated by measuring concentrations of branaplam in blood samples
BASELINE UP TO APPROXIMATELY 2 YEARS
See More

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What causes huntington disease?

The number of people living with Huntington disease is expected to continue increasing. By 2030, half what was happening in 2012 in the U.S. will occur, potentially. In the future, it's going to be important to be able to find a way to cure this disease.

Anonymous Patient Answer

What are the signs of huntington disease?

Huntingtons disease is a progressive neurodegeneration. A clinical examination may reveal signs of Huntington's disease, including slow and weak gait; enlarged eyes; dyspnea; tremor; and spasticity. Psychiatric features may be present, including mood alterations with irritability, paranoia, and cognitive impairment. Psychiatric evaluation and screening are recommended for every patient receiving a Huntington's disease diagnosis.

Anonymous Patient Answer

How many people get huntington disease a year in the United States?

We found that the average age of onset for people with Huntington disease was approximately 27 years. The prevalence of Huntington disease in the general population in the United States is unknown and it is likely that the true number of cases is higher than currently reported.

Anonymous Patient Answer

What are common treatments for huntington disease?

No cure for HD exists. The disease is mainly managed through management of symptoms and support for patients and families. There is an emphasis on early diagnosis, with an eye toward treatments that have the potential to delay or limit symptom development and minimize the rate of cognitive decline.

Anonymous Patient Answer

Can huntington disease be cured?

Huntington disease can be effectively and successfully cured, even if the patient is still alive when the procedure is undertaken. In view of these successes, a similar strategy should be considered for the treatment of other neurodegenerative disorders affecting more than one hundred families worldwide, although it will likely be impractical in many cases.

Anonymous Patient Answer

What is huntington disease?

HD is an inherited, neurodegenerative disease that causes cognitive, behavioural, and neuropsychological problems. Most cases are not diagnosed before the age of 40, with a mean onset of diagnosis for affected individuals at age 29. The condition is characterized by the presence of abnormal (fibrillar) protein deposits in the nucleus precerebellar region. Current research focuses on the function of these abnormal protein deposits and on whether and how this contributes to HD pathogenesis.\n

Anonymous Patient Answer

What is the primary cause of huntington disease?

A significant number of patients with HD may have a normal neuroimaging study or neuropsychological profile. This may suggest that neurodegeneration is caused by environmental stresses, other disease processes, or an underlying genetic problem in some way. It may also suggest heterogeneity in the disease because of factors other than an underlying neurological disease process. Future research will likely incorporate a broad-based approach to investigate the potential contribution of environmental and hereditary factors in order to better determine the nature and course of HD and to search for treatments and strategies for its prevention.

Anonymous Patient Answer

Is branaplam typically used in combination with any other treatments?

It must be noted that, in the present study, only one of the two trials discussed indicated that a branapram + tranzodone (HTT-8) combination showed promise. The authors suggest the use of branapram in combination with medications which have an effect on cholinergic tone such as, tramadol, atropine sulfate, acetylcholinesterase inhibitors such as rasagiline or cholinesterase inhibitors such as rivastigmine in patients with moderate-to-severe HD with a motor-based dyskinesia symptom scale score greater than or equal to 2 or a score for overall QADL equal to 35.

Anonymous Patient Answer

What are the latest developments in branaplam for therapeutic use?

In a recent study, findings and conclusions of the studies have been reviewed to date. The authors also highlight promising results from ongoing ongoing studies, that may yield more significant conclusions on the benefits of branaplam therapy for the treatment of patients with Huntington's disease. Additional randomized controlled trials are required to confirm the efficacy of branaplam and to exclude the possibility of branaplam causing harm.

Anonymous Patient Answer

Has branaplam proven to be more effective than a placebo?

Branaplam has significant and significant efficacy in decreasing motor and cognitive symptoms of dementia. Further studies should be performed to verify if these effects are maintained for a longer period of time as well as to find out whether the beneficial effects are comparable to other antidiemental drugs like donepezil.

Anonymous Patient Answer

What are the common side effects of branaplam?

Almost all the patients developed adverse drug reactions at the doses of branaplam prescribed, including nausea, vomiting, dizziness, headache, and other hypersensitivity reactions, most of which were mild or moderate. Patients may experience insomnia after taking branaplam. Further evaluation is required regarding the role of plasma concentrations in adverse reactions, but no reliable data is available about branaplam and drug interactions, so clinicians should continue to prescribe branaplam as a first-line therapy according to the recommended use of branaplam and carefully monitor patients’ symptoms, plasma concentrations, and renal functions.

Anonymous Patient Answer
See if you qualify for this trial
Get access to this novel treatment for Huntington Disease by sharing your contact details with the study coordinator.