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

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Carl Allen
Study ID
NCT04079179
Phase
PHASE2
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Cobimetinib — DRUG
    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).

Study Details

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.

Key Dates

Start date
Apr 19, 2021
Status verified
Sep 2025
Primary completion
Dec 31, 2025
Completion
Dec 31, 2029

Study Design

Enrollment
90 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Patients < 21 years with recurrent LCH (Grp1)
    Children (≥ 6 months) and young adults (\<21 years) with recurrent active LCH lesions (may also have LCH-ND).
  • Experimental: Patients of any age with LCH-ND (Grp2)
    Patients of any age (≥ 6 months) with progressive LCH Neurodegenerative Disease (LCH-ND) without other sites of active LCH.
  • Experimental: Patients <21 years with other histiocytic disorders (Grp3)
    Newly diagnosed or relapsed/refractory children (≥ 6 months) and young adults (\<21 years) with other histiocytic disorders including juvenile xanthogranuloma, Erdheim-Chester disease, histiocytic sarcoma and Rosai-Dorfman disease.
  • Experimental: Patients ≥ 21 years with LCH/histiocytic disorders (Grp4)
    Adults (≥21 years) with LCH or other histiocytic disorder with recurrent active lesions (may also have LCH-ND).

Primary Outcome Measure

Overall Response Rates using modified RECiST criteria [ Time Frame: 12 months ]

Central Contacts

Locations (12)

FacilityCityStateZIPSite coordinators
Phoenix Children's HospitalPhoenixArizona85016
Laura Retson, MD
[email protected]
(602) 933-0920
Arkansas Children's HospitalLittle RockArkansas72202
Kevin Bielamowicz
[email protected]
501-364-1494
Children's Hospital of Orange CountyOrangeCalifornia92868
Lilbeth Torno, MD
[email protected]
714-509-4348
UCSF Benioff Children's HospitalSan FranciscoCalifornia94158
Karina Wong
[email protected]
415-298-9434
Mahum Kudia
[email protected]
415-290-1053
Michelle L Hermiston, MD (PRINCIPAL_INVESTIGATOR)
Children's National HospitalWashington D.C.District of Columbia20010
Birte Wistinghausen, MD
[email protected]
2024762800
John Hopkins University School of MedicineBaltimoreMaryland21287
Elias T Zambidis, MD PhD
[email protected]
(410) 614-0123
Dana Farber Cancer Institute, Boston Children'sBostonMassachusetts02215
Barbara Degar, MD
[email protected]
617-632-5186
Sara Galinko
[email protected]
617-632-6644
Memorial Sloan Kettering Cancer CenterNew YorkNew York10065
Christopher Forlenza
[email protected]
212-639-5226
NACHO ConsortiumMemphisTennessee38105
Sara Clyburn
[email protected]
901-595-6042
Heidi Clough
[email protected]
901-595-0362
Children's Medical Center- UTSWDallasTexas75235
Erin Butler, MD
[email protected]
214-648-6332
Texas Children's HospitalHoustonTexas77030
Carl E. Allen, MD, PhD
[email protected]
832-826-0860
University of Wisconsin-American Family Children's HospitalMadisonWisconsin53792-

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