Study on Cobimetinib for Treating Resistant Langerhans Cell Histiocytosis and Other Histiocytic Disorders

Sponsor: Carl Allen

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT04079179

Provider View

This clinical trial is testing a drug called cobimetinib in children and adults with Langerhans cell histiocytosis (LCH) and other histiocytic disorders that have returned or do not respond to treatment. Cobimetinib works by blocking a protein involved in incorrect growth signals in these cells.

Patient Parameters

Program Overview

This study is exploring the use of cobimetinib, a drug that targets a protein called MEK, in patients with Langerhans cell histiocytosis (LCH) and other similar disorders that have not responded to previous treatments or have come back. These disorders are caused by immune cells that grow incorrectly due to gene mutations, leading to tissue damage. Cobimetinib aims to block these incorrect signals and help control the disease. The study will include four different groups of patients to see if the drug is safe and effective.

Description

Histiocytic disorders are conditions where certain immune cells, called histiocytes, malfunction or build up in the body. This can happen due to genetic changes that send wrong signals to these cells, causing them to grow abnormally and damage tissues. Some of these disorders include Langerhans cell histiocytosis (LCH), juvenile xanthogranuloma (JXG), Erdheim-Chester disease (ECD), and Rosai-Dorfman Disease (RDD). In some cases, LCH can lead to brain damage, known as neurodegeneration, when the cells affect the brain.

The purpose of this study is to determine if cobimetinib is safe and effective for patients with these disorders, especially those with a specific gene mutation called BRAF-V600E. This mutation can cause cells to grow uncontrollably. Cobimetinib is designed to block a protein called MEK, which is involved in this process, potentially helping to control the disease.

Eligibility Criteria

Inclusion Criteria

  • Age requirements vary by group:
    • Group 1: 6 months to under 21 years
    • Group 2: At least 6 months
    • Group 3: 6 months to under 21 years
    • Group 4: 21 years or older
  • Must be able to take the medication orally or through a feeding tube.
  • Must have a confirmed diagnosis of LCH or other histiocytic disorders.
  • Must have tried and not responded to at least one previous treatment.
  • Must have a certain level of physical health and organ function, including:
    • Blood cell counts and kidney, liver, and heart function within specified limits.
  • Women of childbearing age must have a negative pregnancy test and agree to use effective birth control during the study.

Exclusion Criteria

  • Cannot take certain medications that affect how cobimetinib works.
  • Must have completed any previous cancer treatments at least 28 days before starting the study.
  • Cannot have had certain treatments like stem cell transplants or specific medications recently.
  • Cannot have other active cancers or serious health conditions that could interfere with the study.
  • Cannot have significant heart problems or a history of certain eye conditions.
  • Cannot be pregnant or breastfeeding.

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