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(3R)-N-[3-[5-(2-Cyclopropylpyrimidin-5-Yl)-1H-Pyrrolo[2,3-B]Pyridine-3-Carbonyl]-2,4-Difluorophenyl]-3-Fluoropyrrolidine-1-Sulfonamide

This article discusses a Phase 2 clinical trial investigating the effectiveness and safety of Plixorafenib (FORE8394), a drug designed to inhibit BRAF class 1 and class 2 alterations in cancer patients. The study focuses on patients with various types of tumors harboring BRAF alterations, including solid tumors and primary central nervous system (CNS) tumors. The trial aims to assess the drug’s efficacy, safety, and other important factors in specific subgroups of patients.

Table of Contents

What is Plixorafenib?

Plixorafenib, also known as FORE8394 or PLX8394, is an investigational drug being developed to treat various types of cancer[1]. It is a tablet taken by mouth that targets specific genetic changes in cancer cells[1].

How Does Plixorafenib Work?

Plixorafenib is designed to inhibit BRAF alterations, which are genetic changes that can drive cancer growth. Specifically, it targets both class 1 and class 2 BRAF alterations[1]. By blocking these altered genes, plixorafenib aims to slow down or stop the growth of cancer cells.

What Conditions Does Plixorafenib Target?

Plixorafenib is being studied in patients with various types of cancer that have BRAF alterations, including:

  • Solid tumors
  • Primary central nervous system (CNS) tumors
  • Gliomas (brain tumors), including:
    • Glioblastoma
    • Astrocytoma
    • Oligodendroglioma

The drug is particularly focused on cancers with BRAF V600E mutations and BRAF fusions, which are specific types of genetic changes[1].

Clinical Trial Details

Plixorafenib is currently being studied in a Phase 2 clinical trial. This trial is designed to evaluate how well the drug works and how safe it is for patients[1]. The study is divided into two main parts:

  1. Subprotocol A: Focuses on patients with solid tumors or primary CNS tumors that have BRAF fusions.
  2. Subprotocol B: Targets patients with recurrent high-grade gliomas (HGG) that have the BRAF V600E mutation.

Who is Eligible for the Trial?

The trial is open to both adults and children (10 years and older) who meet specific criteria[1]. Key eligibility factors include:

  • Having a confirmed BRAF alteration in their tumor
  • Having cancer that has progressed or not responded to standard treatments
  • Being in overall good health, apart from their cancer

Some patients may not be eligible if they have certain other medical conditions or have received specific prior treatments[1].

Potential Benefits of Plixorafenib

While the effectiveness of plixorafenib is still being studied, researchers hope it may offer several potential benefits:

  • Shrinking tumors or slowing their growth
  • Improving symptoms caused by the cancer
  • Potentially offering a treatment option for patients who have limited choices

The trial will measure outcomes such as how many patients’ tumors shrink (overall response rate) and how long the benefits last (duration of response)[1].

Important Considerations

Patients considering participation in this trial should be aware of the following:

  • As an investigational drug, the full risks and benefits of plixorafenib are not yet known.
  • Participants will need to undergo regular tests and check-ups as part of the study.
  • Some medications may interact with plixorafenib and need to be avoided during the trial[1].
  • The trial involves a placebo-controlled design, meaning some participants may receive a placebo instead of the active drug.

It’s crucial for patients to discuss all aspects of the trial with their healthcare team to determine if participation is right for them.

Aspect Details
Drug Name Plixorafenib (FORE8394)
Trial Phase Phase 2
Target Population Patients with tumors harboring BRAF alterations
Age Range 10 years and older
Primary Objectives Evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics
Primary Endpoint Overall Response Rate (ORR)
Secondary Endpoints Duration of Response, Progression-Free Survival, Disease Control Rate
Tumor Types Solid tumors, primary CNS tumors, high-grade gliomas
Administration Oral tablet

FAQ

  • What is Plixorafenib (FORE8394)? Plixorafenib (FORE8394) is an investigational drug designed to inhibit BRAF class 1 and class 2 alterations in cancer patients. It is being studied for its potential effectiveness in treating various types of tumors that have BRAF gene mutations.
  • What types of cancer does this clinical trial focus on? This clinical trial focuses on patients with tumors harboring BRAF alterations, including locally advanced or metastatic solid tumors, recurrent or progressive primary CNS tumors, and high-grade gliomas (HGG) with BRAF V600E mutations.
  • Who is eligible to participate in this clinical trial? Eligibility varies depending on the subprotocol, but generally includes patients aged 10 years and older, weighing at least 30 kg, with specific tumor types and BRAF alterations. Detailed eligibility criteria are provided for each subprotocol in the trial.
  • What are the main objectives of this clinical trial? The main objectives are to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Plixorafenib in defined subgroups of patients with tumors harboring BRAF alterations. The trial also aims to assess additional measures of clinical benefit and characterize the drug's plasma pharmacokinetic profile.
  • How is the effectiveness of Plixorafenib measured in this trial? The primary measure of effectiveness is the Overall Response Rate (ORR) as determined by specific criteria (RECIST v1.1 or RANO) appropriate for each tumor type. This is assessed by a Blinded Independent Central Review (BICR). Secondary measures include duration of response, progression-free survival, and disease control rate.

Ongoing Clinical Trials on (3R)-N-[3-[5-(2-Cyclopropylpyrimidin-5-Yl)-1H-Pyrrolo[2,3-B]Pyridine-3-Carbonyl]-2,4-Difluorophenyl]-3-Fluoropyrrolidine-1-Sulfonamide

  • Study on the Effectiveness and Safety of Plixorafenib (FORE8394) for Patients with Cancer Having BRAF Alterations

    Recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy The Netherlands Norway Spain +1

Glossary

  • BRAF: A gene that provides instructions for making a protein involved in cell signaling and growth. Mutations in this gene can lead to various types of cancer.
  • CNS: Central Nervous System, which includes the brain and spinal cord.
  • HGG: High-Grade Glioma, a type of fast-growing brain tumor that forms from glial cells (cells that support and protect nerve cells).
  • LGG: Low-Grade Glioma, a slower-growing type of brain tumor that also forms from glial cells.
  • RANO: Response Assessment in Neuro-Oncology, a set of criteria used to evaluate treatment response in brain tumors.
  • RECIST: Response Evaluation Criteria in Solid Tumors, a standard way to measure how well a cancer patient responds to treatment.
  • Pharmacokinetics: The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Pharmacodynamics: The study of how a drug affects the body, including its mechanism of action and relationship between drug concentration and effect.
  • Overall Response Rate (ORR): The proportion of patients whose cancer shrinks or disappears after treatment.
  • Progression-Free Survival (PFS): The length of time during and after treatment that a patient lives with cancer without it worsening.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-plixorafenib-fore8394-for-patients-with-cancer-having-braf-alterations/