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(R)-1-(Tert-Butyl)-N-(8-(2-((1-Methyl-1H-Pyrazol-4-Yl)Amino)Pyrimidin-4-Yl)-2-(Oxetan-3-Yl)-2,3,4,5-Tetrahydro-1H-Benzo[C]Azepin-5-Yl)-1H-1,2,3-Triazole-4-Carboxamide

A new clinical trial is underway to investigate the safety and effectiveness of BIIB091, a potential treatment for relapsing forms of multiple sclerosis (MS). This study, known as FUSION, aims to evaluate BIIB091 both as a standalone therapy and in combination with diroximel fumarate (DRF). The trial focuses on assessing the drug’s impact on brain inflammation and its overall safety profile in adults with relapsing MS.

Table of Contents

What is BIIB091?

BIIB091 is a new medication being studied for the treatment of relapsing forms of multiple sclerosis (MS). Its full chemical name is (R)-1-(TERT-BUTYL)-N-(8-(2-((1-METHYL-1H-PYRAZOL-4-YL)AMINO)PYRIMIDIN-4-YL)-2-(OXETAN-3-YL)-2,3,4,5-TETRAHYDRO-1H-BENZO[C]AZEPIN-5-YL)-1H-1,2,3-TRIAZOLE-4-CARBOXAMIDE[1]. This long name describes its unique chemical structure. For simplicity, we’ll refer to it as BIIB091 throughout this article.

How BIIB091 Works

BIIB091 is classified as a small molecule drug[1]. Small molecule drugs are typically able to enter cells easily due to their size. In the case of BIIB091, it’s designed to target a specific protein in the body called Bruton’s tyrosine kinase (BTK)[1]. BTK is involved in the immune system’s response, and by targeting it, BIIB091 aims to reduce the harmful immune activity that occurs in multiple sclerosis.

Target Condition: Relapsing Forms of Multiple Sclerosis

BIIB091 is being developed to treat relapsing forms of multiple sclerosis (MS)[1]. This includes:

  • Relapsing-remitting multiple sclerosis (RRMS): The most common form of MS, characterized by periods of new symptoms or relapses, followed by periods of recovery or remission.
  • Active secondary progressive multiple sclerosis (SPMS): A form of MS that develops in some people with RRMS, where disability progressively worsens over time, with or without relapses.
These forms of MS involve inflammation in the central nervous system (brain and spinal cord) that causes damage to the protective covering of nerve fibers, leading to various neurological symptoms.

Clinical Trial: FUSION Study

BIIB091 is currently being studied in a clinical trial called FUSION[1]. This is a Phase 2 study, which means it’s designed to test the drug’s effectiveness and safety in a larger group of people with MS. The study has two parts:

  1. Part 1: Testing BIIB091 alone (monotherapy)
  2. Part 2: Testing BIIB091 in combination with another MS drug called Diroximel Fumarate (DRF)
This approach allows researchers to understand how BIIB091 works on its own and how it might work together with an existing MS treatment.

Eligibility Criteria

To participate in the FUSION study, patients must meet certain criteria[1]. Some key eligibility requirements include:

  • Diagnosis of relapsing MS (RRMS or active SPMS) according to specific diagnostic criteria
  • MS symptoms for less than 20 years
  • An Expanded Disability Status Scale (EDSS) score between 0 and 5.0 (This scale measures the degree of disability in MS)
  • Evidence of recent disease activity, such as relapses or new lesions on brain MRI scans
There are also several conditions that would exclude a person from participating, such as having primary progressive MS, recent MS relapse, or certain other health conditions.

Study Objectives

The FUSION study aims to answer several important questions about BIIB091[1]:

  • Is BIIB091 safe and well-tolerated when used alone?
  • How does BIIB091 affect brain inflammation when used alone?
  • How does the combination of BIIB091 and DRF compare to DRF alone in reducing brain inflammation?
  • Is the combination of BIIB091 and DRF safe and well-tolerated?
To answer these questions, researchers will use various measures, including:
  • MRI scans: To look for new or enlarging lesions in the brain, which are signs of active inflammation
  • Adverse event monitoring: To track any side effects or safety concerns
  • ECG measurements: To monitor heart activity and ensure the treatment doesn’t cause heart-related issues

Safety Considerations

As with any new medication, safety is a top priority in the BIIB091 study[1]. The researchers will closely monitor participants for any side effects or adverse reactions. Some specific safety considerations include:

  • Allergic reactions: Participants with known allergies to BIIB091, DRF, or similar drugs are not eligible for the study
  • Infections: The study excludes people with certain infections, including HIV and hepatitis B or C
  • Heart monitoring: Regular ECG tests will be performed to check for any effects on heart rhythm
It’s important to note that while BIIB091 shows promise, it is still an experimental drug. More research is needed to fully understand its effectiveness and safety profile.

Aspect Details
Drug Name BIIB091
Trial Name FUSION
Condition Relapsing Forms of Multiple Sclerosis
Trial Design 2-Part, Multicenter, Randomized, Blinded, Active-Controlled Phase 2 Study
Primary Objectives 1. Investigate safety and tolerability of BIIB091 monotherapy
2. Evaluate effects of BIIB091 + DRF combination therapy on MRI measures of CNS inflammation
Key Inclusion Criteria – Diagnosis of RMS
– MS symptom onset < 20 years
– EDSS score 0-5.0
– Recent relapses or new MRI lesions
Key Exclusion Criteria – Diagnosis of PPMS
– Recent MS relapse
– Severe allergic reactions to study treatments
– Certain infections (HIV, Hepatitis B/C)
Primary Endpoints 1. Number of participants with adverse events
2. Cumulative number of new T1 GdE lesions
Treatment Duration Up to 48 weeks
Maximum Daily Dose 700 mg

FAQ

  • What is the main purpose of the FUSION clinical trial? The main purpose of the FUSION clinical trial is to investigate the safety and effectiveness of BIIB091 in treating relapsing forms of multiple sclerosis. The study is conducted in two parts: Part 1 evaluates BIIB091 as a standalone therapy, while Part 2 assesses BIIB091 in combination with diroximel fumarate (DRF).
  • Who is eligible to participate in this clinical trial? Eligible participants include adults diagnosed with relapsing forms of multiple sclerosis (either relapsing-remitting MS or active secondary progressive MS) according to the 2017 Revised McDonald criteria. They must have had MS symptoms for less than 20 years and meet specific criteria regarding recent relapses or brain lesions.
  • What are the main outcomes being measured in this trial? The main outcomes being measured include the number of participants experiencing adverse events and serious adverse events, as well as the cumulative number of new T1 gadolinium-enhancing lesions on brain MRI scans. The trial also evaluates changes in various MRI measures of active central nervous system inflammation.
  • How long does the treatment period last in this clinical trial? The maximum treatment period for this clinical trial is 48 weeks, which is approximately 11 months.
  • What is BIIB091 and how is it administered? BIIB091 is the investigational drug being studied in this trial. It is a small molecule medication that is administered orally in tablet form. The maximum daily dose being tested is 700 mg.

Ongoing Clinical Trials on (R)-1-(Tert-Butyl)-N-(8-(2-((1-Methyl-1H-Pyrazol-4-Yl)Amino)Pyrimidin-4-Yl)-2-(Oxetan-3-Yl)-2,3,4,5-Tetrahydro-1H-Benzo[C]Azepin-5-Yl)-1H-1,2,3-Triazole-4-Carboxamide

  • Study on the Safety and Efficacy of BIIB091 and Diroximel Fumarate for Patients with Relapsing Multiple Sclerosis

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria Czechia Germany Italy Poland Romania +1

Glossary

  • Relapsing Multiple Sclerosis (RMS): A form of multiple sclerosis characterized by periods of new or worsening symptoms (relapses) followed by periods of recovery or remission.
  • Diroximel Fumarate (DRF): A medication used to treat relapsing forms of multiple sclerosis that is being studied in combination with BIIB091 in this trial.
  • Magnetic Resonance Imaging (MRI): A medical imaging technique that uses powerful magnets and radio waves to create detailed images of the body's internal structures, including the brain and spinal cord.
  • Gadolinium-Enhancing (GdE) Lesions: Areas of active inflammation in the brain or spinal cord that can be seen on MRI scans after the injection of a contrast agent called gadolinium.
  • Expanded Disability Status Scale (EDSS): A method of measuring disability in multiple sclerosis, ranging from 0 (normal neurological exam) to 10 (death due to MS).
  • T2 Hyperintense Lesions: Areas of damage in the brain or spinal cord that appear bright on certain types of MRI scans, indicating past or present inflammation or tissue damage.
  • Bruton's Tyrosine Kinase (BTK) Inhibitor: A type of medication that blocks the action of an enzyme called BTK, which is involved in the development and function of certain immune cells.
  • Central Nervous System (CNS): The part of the nervous system consisting of the brain and spinal cord, which is primarily affected in multiple sclerosis.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-efficacy-of-biib091-and-diroximel-fumarate-for-patients-with-relapsing-multiple-sclerosis/