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mTOR Inhibitor

Trametinib + Everolimus for Recurrent Brain Cancer

Phase 1
Recruiting
Led By Sabine Mueller
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
Creatinine clearance or radioisotope growth factor receptor (rGFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows: 1 to < 2 years: 0.6 (male), 0.6 (female); 2 to < 6 years: 0.8 (male), 0.8 (female); 6 to < 10 years: 1 (male), 1 (female); 10 to < 13 years: 1.2 (male), 1.2 (female); 13 to < 16 years: 1.5 (male), 1.4 (female); >= 16 years: 1.7 (male), 1.4 (female)
Participants must have histologically confirmed diagnosis of an LGG (WHO grade I-II) that is recurrent or progressive after prior treatment (biologic, chemotherapy or radiation therapy) or must have a histologically confirmed diagnosis of a high grade glioma (HGG) (WHO grade III-VI)
Must not have
Women of childbearing potential who are pregnant or breast-feeding
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial studies the side effects and best dose of two drugs, trametinib and everolimus, in treating pediatric and young adult patients with low grade gliomas that has come back (recurrent).

Who is the study for?
This trial is for pediatric and young adult patients with recurrent gliomas, including those with low grade (WHO I-II) or high grade (III-VI) tumors. It's open to participants who've had prior treatments and have stable neurological deficits. They must not be pregnant, agree to use contraception, and meet specific health criteria like adequate blood counts and organ function.
What is being tested?
The trial is testing the combination of two drugs: Trametinib targets a protein called MEK to stop tumor growth, while Everolimus blocks a different pathway that helps tumors grow. The goal is to see if using both drugs together is more effective than using them separately in treating gliomas.
What are the potential side effects?
Potential side effects include skin rash, diarrhea, fatigue, mouth sores, infections due to weakened immune system, liver problems indicated by yellowing of the skin or eyes (jaundice), shortness of breath from lung issues, abnormal blood tests reflecting kidney or pancreas problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My kidney function is within the required range for my age and gender.
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My brain tumor is low-grade and has returned or worsened after treatment, or it is high-grade.
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My cancer started in the spinal cord or has spread widely.
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I am over 18 and my blood pressure is below 140/90 mm Hg.
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It's been over 6 months since my allogeneic bone marrow transplant or over 3 months since my autologous transplant.
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My cancer can be measured or observed.
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My child's blood pressure is within the normal range for their age, height, and gender.
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I had my last radiation treatment more than 12 weeks ago and my cancer has progressed since then.
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I can provide tissue samples or previous test results for genomic testing.
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I received my last monoclonal antibody treatment over four weeks ago.
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My bilirubin levels are within the normal range for my age.
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I have had treatments other than just surgery for my low-grade glioma.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Area Under the Curve (AUC) of trametinib and everolimus
Dose limiting toxicities (DLTs) of the combination for both continuous and intermittent dosing schedules
Incidence of adverse events for both continuous and intermittent dosing schedules
+3 more

Side effects data

From 2021 Phase 2 trial • 206 Patients • NCT02034110
47%
Pyrexia
36%
Fatigue
33%
Anaemia
33%
Nausea
33%
Decreased appetite
28%
Rash
22%
Headache
22%
Constipation
22%
Pneumonia
22%
Chills
22%
Dyspnoea
19%
Dizziness
19%
Hypoalbuminaemia
19%
Vomiting
19%
Diarrhoea
19%
Hyponatraemia
17%
Dysphagia
17%
Blood alkaline phosphatase increased
17%
Back pain
14%
Aspartate aminotransferase increased
14%
Hypocalcaemia
14%
Dry mouth
14%
Hyperglycaemia
14%
Arthralgia
14%
Oedema peripheral
14%
White blood cell count decreased
14%
Insomnia
14%
Hypotension
11%
Hypokalaemia
11%
Haemoptysis
11%
Alanine aminotransferase increased
11%
Dry skin
11%
Hypothyroidism
11%
Pruritus
11%
Visual impairment
11%
Weight decreased
11%
Cough
8%
Productive cough
8%
Upper respiratory tract infection
8%
Rash maculo-papular
8%
Mucosal inflammation
8%
Hypercalcaemia
8%
Gastrooesophageal reflux disease
8%
Pleural effusion
8%
Night sweats
8%
Asthenia
8%
Ejection fraction decreased
8%
Electrocardiogram QT prolonged
8%
Gamma-glutamyltransferase increased
8%
Neutrophil count decreased
8%
Neck pain
6%
Rhinorrhoea
6%
Seborrhoeic keratosis
6%
Haematochezia
6%
Skin lesion
6%
Acute kidney injury
6%
Pulmonary embolism
6%
Thrombocytopenia
6%
Atrial fibrillation
6%
Stomatitis
6%
Rhinitis allergic
6%
Tachycardia
6%
Abdominal pain upper
6%
Hyperuricaemia
6%
Leukopenia
6%
Sinusitis
6%
Urinary tract infection
6%
Polyneuropathy
6%
Haematuria
6%
Neutropenia
6%
Ear pain
6%
Abdominal pain
6%
Feeling cold
6%
Non-cardiac chest pain
6%
Pain
6%
Fungal infection
6%
Nasopharyngitis
6%
Blood creatinine increased
6%
Blood urea increased
6%
Neutrophil count increased
6%
Hypomagnesaemia
6%
Myalgia
6%
Neuropathy peripheral
6%
Proteinuria
6%
Nasal congestion
6%
Pneumonitis
6%
Palmar-plantar erythrodysaesthesia syndrome
3%
Malaise
3%
Pelvic infection
3%
Rhabdomyolysis
3%
Tinnitus
3%
Sepsis
3%
Cataract
3%
Dermatitis acneiform
3%
Erythema nodosum
3%
Femoral neck fracture
3%
Hyperglycaemic hyperosmolar nonketotic syndrome
3%
Aortic thrombosis
3%
Pollakiuria
3%
Hypophosphataemia
3%
Flushing
3%
Oesophageal stenosis
3%
Atrioventricular block first degree
3%
Photophobia
3%
Dehydration
3%
Toothache
3%
Oral candidiasis
3%
Urinary retention
3%
Alopecia
3%
Skin mass
3%
Aspiration
3%
Vision blurred
3%
Oedema
3%
Depression
3%
Folliculitis
3%
Staphylococcal infection
3%
Clavicle fracture
3%
Aphasia
3%
Cardiac ventricular thrombosis
3%
Stress cardiomyopathy
3%
Oral pain
3%
Clostridium difficile infection
3%
Diverticulitis
3%
Pneumonia aspiration
3%
Pneumonia necrotising
3%
Wound infection
3%
Hyperkalaemia
3%
Rib fracture
3%
Bladder transitional cell carcinoma
3%
Facial nerve disorder
3%
Hypertension
3%
Paralysis recurrent laryngeal nerve
3%
Syncope
3%
Hallucination
3%
Pulmonary haematoma
3%
Sinus bradycardia
3%
Dry eye
3%
Abdominal distension
3%
Dyspepsia
3%
Gait disturbance
3%
Nodule
3%
Xerosis
3%
Conjunctivitis
3%
Tooth infection
3%
Procedural pain
3%
Blood creatine phosphokinase increased
3%
Platelet count decreased
3%
Weight increased
3%
Flank pain
3%
Muscular weakness
3%
Musculoskeletal chest pain
3%
Pain in extremity
3%
Hypoaesthesia
3%
Paraesthesia
3%
Anxiety
3%
Sleep disorder
3%
Dysphonia
3%
Epistaxis
3%
Upper-airway cough syndrome
3%
Wheezing
3%
Erythema
100%
80%
60%
40%
20%
0%
Study treatment Arm
Anaplastic Thyroid Cancer (ATC) (On-Treatment)
Biliary Tract Cancer (BTC) (On-Treatment)
Gastrointestinal Stromal Tumor (GIST) (On-Treatment)
Low Grade (WHO G1/G2) Glioma (LGG) (On-Treatment)
Anaplastic Thyroid Cancer (ATC) (Post-treatment Survival Follow-up)
Gastrointestinal Stromal Tumor (GIST) (Post-treatment Survival Follow-up)
Low Grade (WHO G1/G2) Glioma (LGG) (Post-treatment Survival Follow-up)
Adenocarcinoma of the Small Intestine (ASI) (Post-treatment Survival Follow-up)
Hairy Cell Leukemia (HCL) (Post-treatment Survival Follow-up)
High Grade (WHO G3/G4) Glioma (HGG) (On-Treatment)
Hairy Cell Leukemia (HCL) (On-Treatment)
Multiple Myeloma (MM) (On-Treatment)
High Grade (WHO G3/G4) Glioma (HGG) (Post-treatment Survival Follow-up)
Adenocarcinoma of the Small Intestine (ASI) (On-Treatment)
Biliary Tract Cancer (BTC) (Post-treatment Survival Follow-up)
Multiple Myeloma (MM) (Post-treatment Survival Follow-up)

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: Treatment (trametinib, everolimus)Experimental Treatment2 Interventions
Patients receive dosing per their assigned dose level. Treatment repeats every 28 days for up to 26 cycles in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Trametinib
2014
Completed Phase 2
~1630
Everolimus
2010
Completed Phase 4
~1510

Find a Location

Who is running the clinical trial?

University of California, San FranciscoLead Sponsor
2,593 Previous Clinical Trials
14,887,900 Total Patients Enrolled
Novartis PharmaceuticalsIndustry Sponsor
2,920 Previous Clinical Trials
4,254,637 Total Patients Enrolled
Pediatric Brain Tumor FoundationUNKNOWN
1 Previous Clinical Trials
30 Total Patients Enrolled
The Lilabean Foundation for Pediatric Brain Cancer ResearchUNKNOWN
Sabine Mueller5.01 ReviewsPrincipal Investigator - University of California, San Francisco
University of California, San Francisco
2 Previous Clinical Trials
124 Total Patients Enrolled

Media Library

Everolimus (mTOR Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04485559 — Phase 1
Brain Tumor Research Study Groups: Treatment (trametinib, everolimus)
Brain Tumor Clinical Trial 2023: Everolimus Highlights & Side Effects. Trial Name: NCT04485559 — Phase 1
Everolimus (mTOR Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04485559 — Phase 1
~21 spots leftby Dec 2027