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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 belowSelect...
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.
Select...
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:Select...
I am not on any other cancer treatments or experimental drugs.
Select...
I do not have uncontrolled seizures.
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I have previously used a BRAF inhibitor like vemurafenib.
Select...
My heart's electrical activity is normal and I don't have severe heart failure.
Select...
I can attend all required follow-up visits and tests.
Select...
My BRAFV600E test result from UCSF is negative.
Select...
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
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 moreSecondary 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 • NCT0100698056%
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%
Dyspnoea
10%
Anaemia
10%
Weight Decreased
10%
Abdominal Pain
10%
Keratosis Pilaris
10%
Palmar-Plantar Erythrodysaesthesia Syndrome
10%
Rash Maculo-Papular
9%
Influenza Like Illness
9%
Blood Bilirubin Increased
9%
Pain
9%
Paraesthesia
8%
Blood Creatinine Increased
8%
Oropharyngeal Pain
8%
Dermal Cyst
8%
Chest Pain
8%
Peripheral Swelling
8%
Alanine Aminotransferase Increased
8%
Folliculitis
7%
Chills
7%
Gamma-Glutamyltransferase Increased
7%
Aspartate Aminotransferase Increased
6%
Dyspepsia
6%
Conjunctivitis
6%
Upper Respiratory Tract Infection
6%
Depression
5%
Abdominal Distension
5%
Dermatitis Acneiform
5%
Influenza
5%
Hyperaesthesia
5%
Skin Exfoliation
5%
Flushing
4%
Acne
3%
Rash erythematous
3%
Basal cell carcinoma
3%
Urinary tract infection
2%
Malignant melanoma
2%
Lower respiratory tract infection
1%
Abdominal pain
1%
Seizure
1%
Pleuritic pain
1%
Thrombocytopenia
1%
Dehydration
1%
Pulmonary embolism
1%
Uveitis
1%
Pancreatitis
1%
Atrial fibrillation
1%
Myocardial infarction
1%
Chest pain
1%
Pneumonia
1%
Blood bilirubin increased
1%
Gamma-glutamyltransferase increased
1%
Acute kidney injury
1%
Pleural effusion
1%
Blood alkaline phosphatase increased
1%
Gastrointestinal haemorrhage
1%
Bowen's disease
1%
Loss of consciousness
100%
80%
60%
40%
20%
0%
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
~3560
Find a Location
Who is running the clinical trial?
University of California, San FranciscoLead Sponsor
2,591 Previous Clinical Trials
14,901,601 Total Patients Enrolled
Genentech, Inc.Industry Sponsor
1,564 Previous Clinical Trials
570,192 Total Patients Enrolled
Sabine Mueller, MDPrincipal InvestigatorUniversity of California, San Francisco
2 Previous Clinical Trials
103 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had all suspicious skin lesions removed.My last radiation treatment was over 12 weeks ago.It has been over 6 months since my bone marrow transplant.I am not on any other cancer treatments or experimental drugs.I do not have uncontrolled seizures.I have previously used a BRAF inhibitor like vemurafenib.My heart's electrical activity is normal and I don't have severe heart failure.My neurological symptoms have been stable for at least a week.I can care for myself but may not be able to do active work.My blood tests show enough neutrophils, platelets, and hemoglobin.My liver tests are within the normal range for my age.My kidney function is good based on tests.I am under 25 and eligible for a specific part of the study based on earlier results.I can attend all required follow-up visits and tests.My BRAFV600E test result from UCSF is negative.I have been diagnosed with a type of brain tumor called glioma.My tumor has the BRAFV600E mutation as confirmed by UCSF.I am under 18 years old.I have had an MRI within the last two weeks.I have tried at least one treatment other than surgery for my condition without success.I had my last dose of strong chemotherapy 3 weeks ago, or 6 weeks ago if it was a specific type (nitrosourea).I've had no recent side effects from biologic therapy, last dose was over a week ago.I have been on a stable or decreasing dose of dexamethasone for at least a week.I am under 25 years old.I am under 25 years old and eligible for the pre-surgical group.I am taking medication that can affect my heart's rhythm.I am not pregnant or breastfeeding.My organ function tests are recent.I do not have any serious illnesses that could affect the study.
Research Study Groups:
This trial has the following groups:- Group 1: Vemurafenib
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.