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Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders

NCT04079179 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 90

Last updated 2025-09-18

No results posted yet for this study

Summary

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.

Conditions

  • Langerhan's Cell Histiocytosis
  • Juvenile Xanthogranuloma
  • Erdheim-Chester Disease
  • Rosai Dorfman Disease
  • Neuro-Degenerative Disease
  • Histiocytic Sarcoma
  • Histiocytic Disorders, Malignant

Interventions

DRUG

Cobimetinib

Cobimetinib will be administered at a maximal dose of 60 mg daily for patients \<18 years old and a flat dose of 40 mg daily for patients ≥18 years for 21 days on, then 7 days off, in a 28-day treatment cycle for a total of 12 cycles (approximately 12 months).

Sponsors & Collaborators

  • Baylor College of Medicine

    collaborator OTHER

  • North American Consortium for Histiocytosis

    collaborator OTHER

  • Genentech, Inc.

    collaborator INDUSTRY

  • Carl Allen

    lead OTHER

Principal Investigators

  • Carl E Allen, MD, PhD · Baylor College of Medicine

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-04-19
Primary Completion
2025-12-31
Completion
2029-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04079179 on ClinicalTrials.gov