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Kinase Inhibitor

Vemurafenib for Pediatric Brain Cancer

Phase < 1

Waitlist Available

Led By Sabine Mueller, MD

Research Sponsored by University of California, San Francisco

Eligibility Criteria Checklist

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

Must have

All skin lesions suspicious for keratoacanthomas/cutaneous squamous cell carcinoma (cSCC) found at baseline dermatology visit must have been excised

Patients with histologically confirmed diagnosis of glioma (WHO Grades I-IV) will be eligible

Must not have

Patients receiving any other anticancer or investigational drug therapy

Patients with uncontrolled seizures are not eligible for the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 6 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is to determine the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of vemurafenib in children with recurrent or refractory gliomas containing the BRAFV600E or BRAF Ins T mutation.

Who is the study for?

This trial is for children with recurrent or refractory gliomas that have a specific BRAFV600E mutation. They must be able to swallow tablets, not have had certain treatments recently, and their organs need to function well. Kids under 18 are eligible for the safety study, while those up to 25 can join other parts of the trial if they meet additional criteria.

What is being tested?

The trial is testing Vemurafenib's safety and proper dosing in kids with brain tumors that came back or didn't respond to treatment. It aims to find the maximum tolerable dose (MTD) and/or recommend a phase 2 dose (RP2D). The drug concentration in tumor tissue will also guide eligibility for later phases.

What are the potential side effects?

Possible side effects include liver problems, skin issues like rashes or lesions that may require removal before starting treatment, heart complications such as QT interval prolongation which affects heart rhythm, and allergic reactions similar to other compounds related to Vemurafenib.

Eligibility Criteria

Inclusion Criteria

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

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I have had all suspicious skin lesions removed.

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I have been diagnosed with a type of brain tumor called glioma.

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My tumor has the BRAFV600E mutation as confirmed by UCSF.

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I am under 18 years old.

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I have tried at least one treatment other than surgery for my condition without success.

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It has been over 6 months since my bone marrow transplant.

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My liver tests are within the normal range for my age.

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My kidney function is good based on tests.

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I am under 25 years old.

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I am under 25 years old and eligible for the pre-surgical group.

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I am not pregnant or breastfeeding.

Exclusion Criteria

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

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I am not on any other cancer treatments or experimental drugs.

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I do not have uncontrolled seizures.

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I have previously used a BRAF inhibitor like vemurafenib.

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My heart's electrical activity is normal and I don't have severe heart failure.

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I can attend all required follow-up visits and tests.

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My BRAFV600E test result from UCSF is negative.

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I am taking medication that can affect my heart's rhythm.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 6 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

Maximum tolerated dose (MTD)/Recommended Phase 2 Dose (RP2D)

Median concentrations of vemurafenib in the blood found through pharmacokinetic (PK) samples

Objective Response Rate

+1 more

Secondary study objectives

Median Intra-tumoral drug level concentration

Progression-free survival

Other study objectives

Mitogen-Activated Protein Kinases

Side effects data

From 2015 Phase 3 trial • 675 Patients • NCT01006980

56%

Arthralgia

48%

Alopecia

47%

Fatigue

43%

Rash

40%

Photosensitivity Reaction

39%

Nausea

37%

Diarrhoea

34%

Headache

29%

Hyperkeratosis

29%

Skin Papilloma

26%

Pruritus

24%

Dry Skin

23%

Decreased Appetite

23%

Pain In Extremity

22%

Vomiting

21%

Pyrexia

20%

Squamous cell carcinoma of skin

18%

Erythema

17%

Sunburn

16%

Dysgeusia

16%

Back Pain

15%

Constipation

15%

Oedema Peripheral

15%

Asthenia

15%

Myalgia

15%

Cough

14%

Seborrhoeic Keratosis

13%

Actinic Keratosis

13%

Musculoskeletal Pain

12%

Melanocytic Naevus

12%

Dizziness

12%

Skin Lesion

11%

Keratoacanthoma

11%

Abdominal Pain Upper

11%

Nasopharyngitis

11%

Insomnia

10%

Blood Alkaline Phosphatase Increased

10%

Anaemia

10%

Weight Decreased

10%

Dyspnoea

10%

Abdominal Pain

10%

Keratosis Pilaris

10%

Palmar-Plantar Erythrodysaesthesia Syndrome

10%

Rash Maculo-Papular

Influenza Like Illness

Blood Bilirubin Increased

Pain

Paraesthesia

Blood Creatinine Increased

Dermal Cyst

Chest Pain

Peripheral Swelling

Oropharyngeal Pain

Alanine Aminotransferase Increased

Folliculitis

Chills

Gamma-Glutamyltransferase Increased

Aspartate Aminotransferase Increased

Dyspepsia

Conjunctivitis

Upper Respiratory Tract Infection

Depression

Abdominal Distension

Dermatitis Acneiform

Influenza

Hyperaesthesia

Skin Exfoliation

Flushing

Acne

Rash erythematous

Basal cell carcinoma

Urinary tract infection

Lower respiratory tract infection

Malignant melanoma

Seizure

Abdominal pain

Pulmonary embolism

Thrombocytopenia

Dehydration

Pleuritic pain

Uveitis

Pancreatitis

Atrial fibrillation

Myocardial infarction

Chest pain

Pneumonia

Blood bilirubin increased

Gamma-glutamyltransferase increased

Acute kidney injury

Pleural effusion

Blood alkaline phosphatase increased

Gastrointestinal haemorrhage

Bowen's disease

Loss of consciousness

100%

80%

60%

40%

20%

Study treatment Arm

Vemurafenib

Vemurafenib After Crossover

Dacarbazine

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: VemurafenibExperimental Treatment1 Intervention

Vemurafenib should be swallowed whole with 8 oz (1 cup) of water. Pharmacokinetic studies will determine if vemurafenib can be "crushed". If patients receiving "crushed" tablets are felt to receive adequate exposure, then they will be allowed to participate in the expansion cohort. \[Patients approved to take "crushed" tablets should use a pill crusher and mix pill with 3-5 ml apple sauce\]. If not, then only patients able to swallow whole pills will be eligible. The patient will be requested to maintain a medication diary of each dose of medication. The medication diary will be returned to clinic staff at the end of each cycle.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vemurafenib

2015

Completed Phase 3

~3550

0 / 18Eligibility criteria met