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Report for Clinical Trial NCT04079179

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

A Phase 2 Study to Assess the Safety and Efficacy of Cobimetinib in Refractory Langerhans Cell Histiocytosis, LCH-Associated Neurodegenerative Disease, and Other Histiocytic Disorders.

This is a research study of a drug called cobimetinib in children and adults diagnosed with Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or does not respond to treatment. Cobimetinib blocks activation of a protein called Mitogen-activated protein kinase (MEK) that is part of incorrect growth signals in histiocytosis cells. Four different groups of patients will be enrolled.

NCT04079179 Langerhan's Cell Histiocytosis Juvenile Xanthogranuloma Erdheim-Chester Disease Rosai Dorfman Disease Neuro-Degenerative Disease Histiocytic Sarcoma Histiocytic Disorders, Malignant
MeSH:Histiocytic Sarcoma Histiocytic Disorders, Malignant Histiocytosis, Langerhans-Cell Neurodegenerative Diseases Histiocytosis Erdheim-Chester Disease Xanthogranuloma, Juvenile Histiocytosis, Sinus
HPO:Histiocytosis Neurodegeneration

1 Interventions

Name: Cobimetinib

Description: All groups will receive the same intervention. Cobimetinib will be given orally at a maximal dose of 60mg daily for 21 days on, then 7 days off, in a 28-day treatment cycle for a total of 12 cycles (approximately 12 months).
Type: Drug
Group Labels: 8

Patients Patients < Patients < 30 years with recurrent LCH (Grp1) Patients Patients < Patients <30 years with other histiocytic disorders (Grp3) Patients of any age with LCH-ND (Grp2) Patients ≥ 30 years with LCH/histiocytic disorders (Grp4)

Primary Outcomes

Description: Proportion of participants with (complete response, partial response, stable disease, progressive disease) by 1 year of therapy with Cobimetinib. It is assumed that at each protocol-specified timepoint, a response assessment occurs. Status calculation will occur at each timepoint for patients who have measurable disease at baseline per the criteria defined in the protocol.

Measure: Overall Response Rates using modified RECiST criteria

Time: 12 months

Secondary Outcomes

Description: Progression Free Survival defined as the length of time during and after the treatment of a disease, that a patient lives with the disease but it does not get worse.

Measure: Progression Free Survival

Time: 12 months

Description: Assessment of the nature and severity of adverse events in patients treated with Cobimetinib for histiocytic disorders.

Measure: Nature and Severity of Adverse Events

Time: 12 months

Other Outcomes

Description: Response assessment using modified RECIST criteria for target lesions with specific mutations (e.g. BRAF-V600E) . Best response rate by 1 year of therapy.

Measure: Response assessment (Modified RECIST) of histiocytic lesions with specific mutations

Time: 12 months

Purpose: Treatment

Allocation: Non-Randomized

Parallel Assignment

There is one SNP


1 V600E

BRAF-V600E) . --- V600E ---

The purpose of this research study is to learn whether cobimetinib is safe and effective in subjects diagnosed with LCH, LCH-ND, RDD, JXG and ECD which may have a specfic mutation called BRAF-V600E. --- V600E ---

BRAF-V600E is a specific change in a gene that may cause cancer cells to grow and spread by sending constant signals to the MEK protein. --- V600E ---

HPO Nodes