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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 6 months
Awards & highlights

Study 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.Check my eligibility
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.See study design
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.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
Median Intra-tumoral drug level concentration
Progression-free survival
Other outcome measures
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%
Oedema Peripheral
15%
Constipation
15%
Myalgia
15%
Asthenia
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%
Weight Decreased
10%
Dyspnoea
10%
Anaemia
10%
Abdominal Pain
10%
Keratosis Pilaris
10%
Palmar-Plantar Erythrodysaesthesia Syndrome
10%
Rash Maculo-Papular
9%
Influenza Like Illness
9%
Pain
9%
Blood Bilirubin Increased
9%
Paraesthesia
8%
Oropharyngeal Pain
8%
Dermal Cyst
8%
Peripheral Swelling
8%
Blood Creatinine Increased
8%
Chest Pain
8%
Alanine Aminotransferase Increased
8%
Folliculitis
7%
Gamma-Glutamyltransferase Increased
7%
Aspartate Aminotransferase Increased
7%
Chills
6%
Dyspepsia
6%
Conjunctivitis
6%
Upper Respiratory Tract Infection
6%
Depression
5%
Dermatitis Acneiform
5%
Abdominal Distension
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%
Thrombocytopenia
1%
Pleuritic pain
1%
Dehydration
1%
Seizure
1%
Pulmonary embolism
1%
Uveitis
1%
Abdominal pain
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

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
~3100

Find a Location

Who is running the clinical trial?

University of California, San FranciscoLead Sponsor
2,493 Previous Clinical Trials
11,931,758 Total Patients Enrolled
Genentech, Inc.Industry Sponsor
1,538 Previous Clinical Trials
567,640 Total Patients Enrolled
Sabine Mueller, MDPrincipal InvestigatorUniversity of California, San Francisco
2 Previous Clinical Trials
103 Total Patients Enrolled

Media Library

Vemurafenib (Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT01748149 — Phase < 1
Pediatric Glioma Research Study Groups: Vemurafenib
Pediatric Glioma Clinical Trial 2023: Vemurafenib Highlights & Side Effects. Trial Name: NCT01748149 — Phase < 1
Vemurafenib (Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01748149 — Phase < 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

For what conditions is Vemurafenib regularly prescribed?

"Vemurafenib is an accepted intervention for metastatic melanoma, non-small cell lung carcinoma and unreseptable cases of skin cancer."

Answered by AI

Are there any vacant slots available for potential participants in this experiment?

"Per clinicaltrials.gov, this medical trial is no longer open for recruitment as the most recent edit to the listing occurred on August 9th 2022. However, there are an abundance of other trials actively searching for participants at present (1360)."

Answered by AI

What other investigations involving Vemurafenib have been conducted?

"Currently, 44 investigations involving Vemurafenib are in progress. Of these studies, 3 have advanced to Phase 3 of the clinical trial process. Toronto, Ontario is a major centre for research into this medication; however there are 1626 other locations conducting trials with vemurafenib as well."

Answered by AI

How many healthcare institutions are facilitating this experiment?

"This research is being administered at Mattel Children's Hospital UCLA in Los Angeles, Dana-Farber Cancer Institute in Boston and UCSF Medical Center-Mount Zion in San Francisco along with another 18 sites."

Answered by AI

How many individuals are actively taking part in this research?

"This clinical trial is not actively recruiting right now; it was initially posted on April 29th, 2014 and the most recent update was recorded August 9th, 2022. For those seeking alternative studies, there are 1316 trials for relapse and 44 Vemurafenib-related investigations which require participants."

Answered by AI
~4 spots leftby Mar 2025