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Kinase Inhibitor

Cobimetinib for Langerhans Cell Histiocytosis (NACHO-COBI Trial)

Phase 2

Recruiting

Research Sponsored by Carl Allen

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age at study entry: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment for Group 1, at least 6 months of age at the time of enrollment for Group 2, and at least 6 months of age and less than 21 years of age at the time of enrollment for Group 3. Participant must be 21 years of age or older at the time of enrollment for Group 4.

Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet which may be taken by mouth or other enteral route such as nasogastric or gastric tube.

Must not have

Other active malignancy or history of secondary malignancy.

Treatment with a long-acting hematopoietic growth factor within 14 days prior to initiation of study drug or a short-acting hematopoietic growth factor within 7 days prior to enrollment.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

No Placebo-Only Group

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.

Who is the study for?

This trial is for children and adults with Langerhans cell histiocytosis (LCH) or other histiocytic disorders that are resistant to treatment. Participants must have proper blood, kidney, liver, and heart function, not be pregnant or breastfeeding, agree to use contraception if applicable, and be able to take oral medication. Specific age groups are targeted for different parts of the study.

What is being tested?

The drug cobimetinib is being tested on patients with various histiocytic disorders including LCH. Cobimetinib works by blocking a protein involved in abnormal cell growth signals. The study will include four distinct patient groups based on their specific conditions.

What are the potential side effects?

While not explicitly listed here, common side effects of drugs like cobimetinib may include diarrhea, acneiform rash (skin eruptions similar to acne), increased liver enzymes indicating liver inflammation, muscle pain or spasms, fatigue, nausea and vomiting.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can take medicine by mouth or through a feeding tube.

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My blood counts meet the required levels without recent medical help.

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My kidney function is good based on tests.

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My liver functions within the normal range for my age.

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I can do most daily activities by myself.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have no other active cancer or a history of another cancer.

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I haven't taken any long-acting blood cell growth boosters in the last 14 days or short-acting ones in the last 7 days.

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I have a history of serious heart problems.

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I haven't taken drugs that affect liver enzyme levels in the last 14 days.

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I experience uncontrollable nausea and vomiting, have issues absorbing food, or have an external biliary shunt.

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I finished my last cancer treatment over 28 days ago and have recovered from side effects.

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I haven't had major surgery or significant injury in the last 28 days and don't expect to need major surgery during the study.

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I have been diagnosed with HIV.

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I have not had radiation therapy in the last 28 days.

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I am on blood thinners or have a bleeding disorder not caused by histiocytic disease.

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I have had a solid organ transplant.

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I am not pregnant or breastfeeding.

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I have not had a stem cell transplant in the last 3 months.

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I have not had a serious brain bleed or any brain bleed in the last 28 days.

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I have had an infection in the last 28 days that is not fully resolved.

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I have had major bowel surgery affecting my nutrient absorption.

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I have had pneumonitis before.

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I do not have serious eye conditions or risks for retinal vein blockage.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Overall Response Rates using modified RECiST criteria

Secondary study objectives

Nature and Severity of Adverse Events

Progression Free Survival

Other study objectives

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

Side effects data

From 2023 Phase 2 trial • 673 Patients • NCT02091141

36%

Diarrhoea

29%

Fatigue

22%

Nausea

18%

Anaemia

15%

Decreased appetite

14%

Infusion related reaction

13%

Chills

13%

Vomiting

12%

Cough

12%

Arthralgia

12%

Abdominal pain

11%

Dyspnoea

11%

Rash

10%

Blood alkaline phosphatase increased

10%

Pyrexia

10%

Pruritus

10%

Constipation

Back pain

Weight decreased

Insomnia

Headache

Aspartate aminotransferase increased

Hypokalaemia

Dry skin

Urinary tract infection

Alanine aminotransferase increased

Oedema peripheral

Hyponatraemia

Blood bilirubin increased

Muscle spasms

Blood creatinine increased

Abdominal distension

Hypoalbuminaemia

Dizziness

Myalgia

Rash maculo-papular

Dehydration

Small intestinal obstruction

Seizure

Pneumonia

Pleural effusion

Pneumonitis

Acute kidney injury

Device related infection

Respiratory failure

Sepsis

Obstruction gastric

Hypoxia

Urosepsis

Fall

Chronic obstructive pulmonary disease

100%

80%

60%

40%

20%

Study treatment Arm

Trastuzumab Plus Pertuzumab

Atezolizumab

Vismodegib

Alectinib

Vemurafenib Plus Cobimetinib

Erlotinib

Vemurafenib

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Patients ≥ 21 years with LCH/histiocytic disorders (Grp4)Experimental Treatment1 Intervention

Adults (≥21 years) with LCH or other histiocytic disorder with recurrent active lesions (may also have LCH-ND).

Group II: Patients of any age with LCH-ND (Grp2)Experimental Treatment1 Intervention

Patients of any age (≥ 6 months) with progressive LCH Neurodegenerative Disease (LCH-ND) without other sites of active LCH.

Group III: Patients <21 years with other histiocytic disorders (Grp3)Experimental Treatment1 Intervention

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.

Group IV: Patients < 21 years with recurrent LCH (Grp1)Experimental Treatment1 Intervention

Children (≥ 6 months) and young adults (\<21 years) with recurrent active LCH lesions (may also have LCH-ND).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cobimetinib

2017

Completed Phase 3

~3630

0 / 24Eligibility criteria met