Cobimetinib for Langerhans Cell Histiocytosis

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Langerhans Cell Histiocytosis+6 MoreCobimetinib - Drug
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a drug called cobimetinib to see if it can help kids and adults with Langerhans cell histiocytosis (LCH) who have relapsed or who haven't responded to other treatments.

Eligible Conditions
  • Langerhans Cell Histiocytosis
  • Juvenile Xanthogranuloma
  • Neurodegenerative Disease
  • Rosai-Dorfman Disease
  • Erdheim-Chester Disease
  • Histiocytic Sarcoma
  • Malignant Histiocytic Disorders

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 2 Secondary · Reporting Duration: 12 months

12 months
Nature and Severity of Adverse Events
Overall Response Rates using modified RECiST criteria
Progression Free Survival
Response assessment (Modified RECIST) of histiocytic lesions with specific mutations

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Side Effects for

Atezolizumab
27%FATIGUE
23%DECREASED APPETITE
21%NAUSEA
19%DIARRHOEA
14%VOMITING
14%ABDOMINAL PAIN
14%BACK PAIN
13%ASTHENIA
13%DYSPNOEA
13%COUGH
13%PYREXIA
12%CONSTIPATION
12%HEADACHE
9%ARTHRALGIA
9%RASH
9%BLOOD ALKALINE PHOSPHATASE INCREASED
9%OEDEMA PERIPHERAL
8%WEIGHT DECREASED
8%ABDOMINAL PAIN UPPER
7%INSOMNIA
7%PAIN IN EXTREMITY
7%ASPARTATE AMINOTRANSFERASE INCREASED
7%UPPER RESPIRATORY TRACT INFECTION
6%ANAEMIA
6%ALANINE AMINOTRANSFERASE INCREASED
4%CHILLS
4%HYPERTENSION
4%MUCOSAL INFLAMMATION
3%HYPOPHOSPHATAEMIA
3%BLOOD THYROID STIMULATING HORMONE INCREASED
3%PRURITUS
3%DRY SKIN
3%MYALGIA
2%BLOOD BILIRUBIN INCREASED
2%DYSPEPSIA
2%HYPOCALCAEMIA
2%MUSCULOSKELETAL PAIN
2%DERMATITIS ACNEIFORM
2%OROPHARYNGEAL PAIN
2%CHEST PAIN
2%DRY MOUTH
2%URINARY TRACT INFECTION
1%SMALL INTESTINAL OBSTRUCTION
1%HYPOKALAEMIA
1%AUTOIMMUNE HEPATITIS
1%ABDOMINAL HERNIA INFECTION
1%MUSCULAR WEAKNESS
1%DELIRIUM
1%PNEUMONITIS
1%DYSPHONIA
1%RASH MACULO-PAPULAR
1%COLITIS
1%PLEURAL EFFUSION
1%RECTAL HAEMORRHAGE
1%HYPERSENSITIVITY
1%LIPASE INCREASED
1%PALMAR-PLANTAR ERYTHRODYSAESTHESIA SYNDROME
1%PELVIC VENOUS THROMBOSIS
1%BILE DUCT OBSTRUCTION
1%STOMA SITE HAEMORRHAGE
1%PULMONARY SEPSIS
This histogram enumerates side effects from a completed 2018 Phase 3 trial (NCT02788279) in the Atezolizumab ARM group. Side effects include: FATIGUE with 27%, DECREASED APPETITE with 23%, NAUSEA with 21%, DIARRHOEA with 19%, VOMITING with 14%.

Trial Design

4 Treatment Groups

Patients <30 years with other histiocytic disorders (Grp3)
1 of 4
Patients < 30 years with recurrent LCH (Grp1)
1 of 4
Patients ≥ 30 years with LCH/histiocytic disorders (Grp4)
1 of 4
Patients of any age with LCH-ND (Grp2)
1 of 4

Experimental Treatment

90 Total Participants · 4 Treatment Groups

Primary Treatment: Cobimetinib · No Placebo Group · Phase 2

Patients <30 years with other histiocytic disorders (Grp3)
Drug
Experimental Group · 1 Intervention: Cobimetinib · Intervention Types: Drug
Patients < 30 years with recurrent LCH (Grp1)
Drug
Experimental Group · 1 Intervention: Cobimetinib · Intervention Types: Drug
Patients ≥ 30 years with LCH/histiocytic disorders (Grp4)
Drug
Experimental Group · 1 Intervention: Cobimetinib · Intervention Types: Drug
Patients of any age with LCH-ND (Grp2)
Drug
Experimental Group · 1 Intervention: Cobimetinib · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cobimetinib
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 12 months

Who is running the clinical trial?

North American Consortium for HistiocytosisOTHER
4 Previous Clinical Trials
1,687 Total Patients Enrolled
Baylor College of MedicineOTHER
923 Previous Clinical Trials
5,987,923 Total Patients Enrolled
Genentech, Inc.Industry Sponsor
1,497 Previous Clinical Trials
564,589 Total Patients Enrolled
Carl AllenLead Sponsor
Carl E Allen, MD, PhDStudy ChairBaylor College of Medicine

Eligibility Criteria

Age Any Age · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
ANC > 0.75 x 10^9/L (unsupported/without growth factor stimulant) (WHO criteria).
Patients must be aged 18 years or older at the time of study entry.
You are 21 years of age or older at the time of enrollment.
Participants must be able to take an enteral dose and formulation of medication