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5,7-Dimethyl-N-((1R,4R)-4-(Pentyloxy)Cyclohexyl)Pyrazolo[1,5-A]Pyrimidine-3-Carboxamide

A new clinical trial is underway to evaluate the effectiveness, safety, and tolerability of a drug called BIA 28-6156 in patients with Parkinson’s disease who have a specific genetic variant in the GBA1 gene. This study aims to assess how well the drug can delay the progression of motor symptoms and improve overall function in these patients. The trial involves testing two different doses of the medication against a placebo over a 78-week period.

Table of Contents

Introduction

Parkinson’s disease (PD) is a progressive neurological disorder that affects movement, balance, and coordination. Recent research has shown that certain genetic variations, particularly in the GBA1 gene, can increase the risk of developing Parkinson’s disease. A new medication called BIA 28-6156 is being studied as a potential treatment for people with Parkinson’s disease who have these specific genetic variations.[1]

What is BIA 28-6156?

BIA 28-6156 is an experimental drug developed by BIAL – R&D Investments, S.A. It is classified as an allosteric activator of the enzyme beta-glucocerebrosidase (GCase). This means it helps to boost the activity of an important enzyme in the body. The medication comes in the form of a film-coated tablet and is taken orally (by mouth).[1]

How Does It Work?

In people with certain GBA1 gene variants, the GCase enzyme doesn’t work as well as it should. This can lead to the buildup of certain substances in brain cells, which may contribute to the development of Parkinson’s disease. BIA 28-6156 is designed to enhance the activity of the GCase enzyme, potentially slowing down the progression of Parkinson’s disease in people with these genetic variations.[1]

Target Population

This medication is specifically being studied for people who meet the following criteria:

  • Have been diagnosed with Parkinson’s disease for at least 1 year but no more than 7 years
  • Are between 35 and 80 years old
  • Have a specific genetic variation in the GBA1 gene
  • Have mild to moderate Parkinson’s symptoms (a modified Hoehn and Yahr score of 2.5 or less)
  • Have a score of 22 or higher on the Montreal Cognitive Assessment (MoCA), which indicates relatively preserved cognitive function

The study excludes people with more advanced Parkinson’s disease, those with certain other medical conditions, and those taking specific medications that might interfere with the study results.[1]

Clinical Trial Details

The ongoing clinical trial, known as the ACTIVATE study, is designed to evaluate the effectiveness, safety, and tolerability of BIA 28-6156. Here are some key details about the trial:

  • It’s a Phase 2 study, which means it’s testing the drug in a larger group of people with Parkinson’s disease to see if it’s effective and to further evaluate its safety.
  • The study is randomized, double-blind, and placebo-controlled. This means that participants are randomly assigned to receive either the actual drug or a placebo (a pill with no active ingredient), and neither the participants nor the researchers know who is receiving which until the study is completed.
  • There are three groups in the study: one group receives 10 mg of BIA 28-6156 per day, another receives 60 mg per day, and the third group receives a placebo.
  • The treatment period lasts up to 78 weeks (about 1.5 years), followed by a 30-day follow-up period.
  • The study aims to include about 237 participants, with 79 people in each group.

The main goal of the study is to see if BIA 28-6156 can delay the progression of motor symptoms in people with Parkinson’s disease who have the GBA1 gene variant.[1]

Potential Benefits

If successful, BIA 28-6156 could potentially:

  • Slow down the progression of motor symptoms in Parkinson’s disease
  • Improve daily living activities for people with Parkinson’s
  • Enhance overall quality of life for patients
  • Provide a targeted treatment option for people with Parkinson’s disease who have specific GBA1 gene variants

However, it’s important to note that these potential benefits are still being studied and have not yet been proven.[1]

Safety and Side Effects

As BIA 28-6156 is still in the testing phase, its full safety profile and potential side effects are not yet known. The clinical trial is designed to carefully monitor participants for any adverse effects. Some general safety measures in the study include:

  • Regular check-ups and assessments throughout the study period
  • Monitoring of liver and kidney function
  • Heart rhythm checks
  • Assessment of mental health and suicide risk

Participants in the study are also asked to use effective birth control methods, as the effects of the drug on pregnancy are not known.[1]

Conclusion

BIA 28-6156 represents a promising new approach to treating Parkinson’s disease, especially for those with specific genetic variations. While the results of the clinical trial are not yet known, this research could potentially lead to more personalized and effective treatments for Parkinson’s disease in the future. As always, it’s important for patients to discuss any new treatments or clinical trials with their healthcare providers to determine the best course of action for their individual situation.

Aspect Details
Study Type Phase 2, randomized, double-blind, placebo-controlled
Medication BIA 28-6156 (10 mg/day and 60 mg/day doses)
Target Population Parkinson’s disease patients with GBA1 gene variant
Primary Objective Assess efficacy in delaying motor progression
Duration 78 weeks of treatment + 30-day follow-up
Key Inclusion Criteria Age 35-80, PD diagnosis 1-7 years, Hoehn and Yahr ≤2.5, MoCA ≥22
Key Exclusion Criteria Gaucher’s disease, atypical parkinsonism, significant psychosis
Main Outcome Measure Time to ≥2-point increase in MDS-UPDRS Part II score

FAQ

  • What is BIA 28-6156 and how does it work? BIA 28-6156 is a new drug that acts as an allosteric activator of the enzyme beta-glucocerebrosidase (GCase). It is designed to target Parkinson's disease in patients with a specific genetic variant in the GBA1 gene, which is associated with an increased risk of developing the condition.
  • Who is eligible to participate in this clinical trial? Eligible participants are adults aged 35-80 with a clinical diagnosis of Parkinson's disease for 1-7 years, a modified Hoehn and Yahr score ≤2.5, and a confirmed GBA1 gene variant. They must also have stable Parkinson's medication for at least 30 days prior to screening and a score of ≥22 on the Montreal Cognitive Assessment (MoCA).
  • What are the main goals of this clinical trial? The primary goal is to assess how well BIA 28-6156 can delay meaningful clinical motor progression in Parkinson's disease patients with the GBA1 gene variant. Secondary goals include evaluating its effect on slowing disease progression, improving functional impairment, and assessing safety and tolerability.
  • How long does the trial last and what does it involve? The trial lasts up to 78 weeks of treatment, followed by a 30-day safety follow-up period. Participants will be randomly assigned to receive either 10 mg/day of BIA 28-6156, 60 mg/day of BIA 28-6156, or a matching placebo. They will continue their usual Parkinson's medications throughout the study.
  • Are there any specific exclusion criteria for this trial? Yes, some key exclusions include having Gaucher's disease, atypical or secondary parkinsonism, moderate motor complications, clinically significant psychosis, or certain medical conditions that could interfere with the study. Additionally, participants cannot be using strong CYP3A4 modulators or certain other medications that might interact with the study drug.

Ongoing Clinical Trials on 5,7-Dimethyl-N-((1R,4R)-4-(Pentyloxy)Cyclohexyl)Pyrazolo[1,5-A]Pyrimidine-3-Carboxamide

  • Study on BIA 28-6156 for Parkinson’s Disease in Patients with GBA1 Gene Variant

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy The Netherlands Poland Portugal +2

Glossary

  • Parkinson's Disease (PD): A progressive neurological disorder that affects movement, often including tremors, stiffness, and difficulty with balance and coordination.
  • GBA1 Gene: A gene that provides instructions for making an enzyme called beta-glucocerebrosidase. Mutations in this gene are associated with an increased risk of developing Parkinson's disease.
  • Allosteric Activator: A substance that binds to an enzyme at a site other than the active site, enhancing the enzyme's activity.
  • Beta-glucocerebrosidase (GCase): An enzyme that breaks down certain fatty substances in cells. Reduced activity of this enzyme is associated with Parkinson's disease in some individuals.
  • MDS-UPDRS: Movement Disorder Society – Unified Parkinson's Disease Rating Scale, a comprehensive assessment tool used to evaluate the severity of Parkinson's disease symptoms.
  • Hoehn and Yahr Scale: A widely used system for describing how the symptoms of Parkinson's disease progress, ranging from stage 1 (mildest) to stage 5 (most severe).
  • Montreal Cognitive Assessment (MoCA): A rapid screening instrument for mild cognitive dysfunction, used to assess various cognitive domains including attention, memory, and language.
  • Pharmacodynamics: The study of how a drug affects the body, including its mechanism of action and the relationship between drug concentration and effect.
  • Pharmacokinetics: The study of how the body processes a drug, including its absorption, distribution, metabolism, and excretion.
  • Double-blind Study: A type of clinical trial where neither the participants nor the researchers know who is receiving the experimental treatment and who is receiving a placebo or standard treatment.

References

  1. http://clinicaltrials.eu/trial/study-on-bia-28-6156-for-parkinsons-disease-in-patients-with-gba1-gene-variant/