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Alkylating Agent; Vinca Alkaloid
Selumetinib vs Carboplatin/Vincristine for Brain Cancer
Phase 3
Recruiting
Led By Jason R Fangusaro
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery
Patients must have the ability to swallow whole capsules
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 10 years
Awards & highlights
Study Summary
This trial is testing if selumetinib works as well or better than the standard treatment (carboplatin/vincristine) for patients with low-grade glioma associated with NF1.
Who is the study for?
This trial is for children and young adults aged 2 to 21 with Neurofibromatosis type 1 (NF1) and low-grade glioma (LGG), including tumors of the optic pathway. They must have measurable tumors, no prior tumor-directed therapy except surgery, stable blood pressure, adequate organ function, and not be pregnant or breastfeeding. Participants need to agree to use effective contraception during the study.Check my eligibility
What is being tested?
The trial compares Selumetinib—a drug that blocks enzymes needed by tumor cells—with standard chemotherapy drugs Carboplatin/Vincristine in treating NF1-associated LGG. It aims to determine if Selumetinib is as effective or better than standard treatment in improving vision for those with optic pathway tumors.See study design
What are the potential side effects?
Selumetinib may cause heart issues, high blood pressure, skin rash, fatigue, nausea and vomiting. Chemotherapy with Carboplatin/Vincristine can lead to hair loss, nerve damage causing numbness or tingling in hands/feet, hearing problems and increased risk of infections.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have an untreated NF-1 associated low-grade glioma, except for surgery.
Select...
I can swallow whole capsules.
Select...
I have been diagnosed with neurofibromatosis type 1.
Select...
I can take care of myself but may not be able to do heavy physical work.
Select...
My kidney function is normal or near normal.
Select...
My bilirubin levels are within the normal range for my age.
Select...
I have a newly-diagnosed low-grade glioma with symptoms or findings related to the tumor.
Select...
My blood pressure is 130/80 mmHg or lower.
Select...
My body surface area is at least 0.5 square meters.
Select...
I can understand and speak English or Spanish for assessments.
Select...
I am between 2 and 21 years old.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 10 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 10 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Event-free survival (EFS)
Number of participants with visual acuity (VA) improvement per arm
Secondary outcome measures
Change in VA using HOTV letter acuity testing
Change in executive function
Change in motor function
+4 moreOther outcome measures
Change in circumpapillary retinal nerve fiber layer (cpRNFL) thickness by treatment arm
Change in macular ganglion cell - inner plexiform layer (GCIPL) thickness by treatment arm
GCIPL thickness at baseline by visual acuity (VA) treatment response
+14 moreSide effects data
From 2020 Phase 2 trial • 8 Patients • NCT03040986100%
Aspartate aminotransferase increased
83%
Edema limbs
83%
Hypoalbuminemia
67%
Hypertension
67%
Fatigue
50%
Nausea
50%
Alkaline phosphatase increased
50%
Anorexia
50%
Anemia
50%
Abdominal pain
50%
Alanine aminotransferase increased
50%
Dyspnea
33%
Dizziness
33%
Generalized muscle weakness
33%
Hypocalcemia
33%
Hyponatremia
33%
Lymphocyte count decreased
33%
Rash maculo-papular
33%
Hypokalemia
33%
Bloating
33%
CPK increased
33%
Cough
33%
Creatinine increased
33%
Vomiting
17%
Alopecia
17%
Atelectasis
17%
Gallbladder infection
17%
Dysphagia
17%
Edema trunk
17%
Lipase increased
17%
Serum amylase increased
17%
Glucose intolerance
17%
Dry mouth
17%
Colonic obstruction
17%
Gallbladder obstruction
17%
Confusion
17%
Pancreatitis
17%
Diarrhea
17%
Dysgeusia
17%
Heart failure
17%
Ascites
17%
Hyperglycemia
17%
Weight loss
17%
White blood cell decreased
17%
Hypomagnesemia
17%
Hypotension
17%
Malaise
17%
Neck pain
17%
Pleural effusion
17%
Postnasal drip
17%
Renal and urinary disorders - Other, Dysuria
17%
Rash acneiform
17%
Neutrophil count decreased
17%
Paresthesia
17%
Peritoneal infection
17%
Back pain
17%
Biliary tract infection
17%
Blood bilirubin increased
17%
Fever
17%
Sore throat
17%
Urinary tract obstruction
17%
Musculoskeletal and connective tissue disorder - Other, muscle spasm
17%
Urine discoloration
100%
80%
60%
40%
20%
0%
Study treatment Arm
Dose Level 0: 75mg Selumetinib Sulfate Twice Daily
75mg Selumetinib Sulfate Twice Daily Follow/by 50mg TwiceDaily
Trial Design
2Treatment groups
Experimental Treatment
Active Control
Group I: Arm II (selumetinib sulfate)Experimental Treatment4 Interventions
Patients receive selumetinib sulfate PO BID on days 1-28. Treatment is continuous and repeats every 28 days for 27 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI throughout the trial.
Group II: Arm I (carboplatin, vincristine)Active Control5 Interventions
INDUCTION: Patients receive carboplatin IV over 60 minutes on days 1, 8, 15, 22, 43, 50, 57, and 64 and vincristine IV or IV push over 1 minute on days 1, 8, 15, 22, 29, 36, 43, 50, 57, and 64 in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI during screening and on study.
MAINTENANCE: Patients receive carboplatin IV over 60 minutes on days 1, 8, 15, and 22 and vincristine IV or IV push over 1 minute on days 1, 8, and 15. Treatment repeats every 6 weeks for 8 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI on study and during follow-up.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Magnetic Resonance Imaging
2017
Completed Phase 3
~1190
Selumetinib Sulfate
2017
Completed Phase 2
~80
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)Lead Sponsor
13,664 Previous Clinical Trials
40,925,843 Total Patients Enrolled
3 Trials studying Neurofibromatosis
284 Patients Enrolled for Neurofibromatosis
Jason R FangusaroPrincipal InvestigatorChildren's Oncology Group
2 Previous Clinical Trials
249 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have an untreated NF-1 associated low-grade glioma, except for surgery.I can swallow whole capsules.I have been diagnosed with neurofibromatosis type 1.I can take care of myself but may not be able to do heavy physical work.I've had a recent MRI of my brain or spine with contrast.My kidney function is normal or near normal.My bilirubin levels are within the normal range for my age.I haven't had surgery in the last 2 weeks and don't take high doses of vitamin E.I have cancer that has spread or I have more than one primary low-grade glioma.I have a newly-diagnosed low-grade glioma with symptoms or findings related to the tumor.My child's blood pressure is within the normal range for their age, height, and gender.My blood pressure is 130/80 mmHg or lower.I've had an MRI of my brain or spine before and after surgery on my tumor within the last 4 weeks.You must have a tumor that can be measured and is at least 1 square centimeter in size.My body surface area is at least 0.5 square meters.Your SGPT (ALT) level in the blood is not more than 3 times the upper limit of normal, which is 135 U/L.Your heart's pumping ability is normal, as shown in an echocardiogram.I have a newly-diagnosed low-grade glioma not in the optic pathway and it's causing symptoms.I have a newly-diagnosed optic pathway glioma with symptoms or findings related to the tumor.You have enough infection-fighting white blood cells in your body.You have enough platelets in your blood (at least 100,000 per microliter).Your corrected QT (QTc) interval is less than or equal to 450 milliseconds on your EKG.I can understand and speak English or Spanish for assessments.I have no history of serious eye conditions and my glaucoma is under control.I am not pregnant.I have a heart condition that could increase my risk for heart disease or have had serious heart issues.You need to have a hemoglobin level of at least 8 grams per deciliter.I have a newly-diagnosed or worsening optic pathway glioma with symptoms or tumor growth.I haven't been treated for another cancer besides surgery in the past year.I have not had any treatments like chemotherapy, radiation, immunotherapy, or bone marrow transplant for my cancer.I am between 2 and 21 years old.I do not have any infections that are not under control.My seizures have been stable with no increase in frequency in the last 2 weeks.Your albumin level in your blood is at least 2 grams per deciliter.
Research Study Groups:
This trial has the following groups:- Group 1: Arm I (carboplatin, vincristine)
- Group 2: Arm II (selumetinib sulfate)
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
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.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How can I sign up for the chance to be a part of this research?
"This clinical study is looking for 290 participants that have visual pathway glioma and meet the age criteria of being between 2 and 21 years old."
Answered by AI
What is the projected amount of people that will enroll in this clinical trial?
"In order to satisfy the requirements of this 290-person clinical trial, Carolinas Medical Center/Levine Cancer Institute in Charlotte, North carolina and East Carolina University in Greenville, California are both actively recruiting patients that meet the specified inclusion criteria."
Answered by AI
Could you please list any other similar clinical trials that have been conducted using Selumetinib Sulfate?
"Selumetinib Sulfate was first studied 19 years ago at the UCSF Helen Diller Family Comprehensive Cancer Center. Out of the 1518 completed studies, 977 are currently ongoing; many of which are being done in Charlotte, North carolina."
Answered by AI
Can you tell me how many staff are needed to manage this study at each site?
"This study has a Carolinas Medical Center/Levine Cancer Institute in Charlotte, North carolina as its base of operations. Additionally, East Carolina University in Greenville, California and Children's Hospital of Orange County in Orange, Arizona are participating locations. There are 100 other medical facilities taking part in this research project."
Answered by AI
Are middle-aged adults welcome in this research project?
"According to the age requirements specified in this study's inclusion criteria, patients must be aged 2-21."
Answered by AI
What is Selumetinib Sulfate's most popular use?
"Selumetinib Sulfate is not only an effective treatment for advanced sarcoma, but also initial treatment, acquired immunodeficiency syndrome, and advanced thymoma."
Answered by AI
Is this trial currently looking for participants?
"As of today, this clinical trial is still recruiting patients. It was first posted on October 4th, 2019 and the most recent update was November 17th, 2022."
Answered by AI
Are there long-term repercussions to prescription Selumetinib Sulfate?
"Selumetinib Sulfate has received a 3 for safety from our team at Power. This is due to the fact that it is a Phase 3 trial, which suggests that not only is there evidence supporting its efficacy, but also that there have been multiple rounds of data collected regarding Selumetinib Sulfate's safety."
Answered by AI
What are the goals that this group of patients hopes to achieve by participating in this clinical trial?
"The primary outcome of this study will be the number of participants with visual acuity (VA) improvement per arm, monitored over a period of up to 3 years. Secondary outcomes include change in motor function, as measured by the Vineland-3 Motor Scale from the Comprehensive Parent Rating Form; change in neurocognitive function, as measured by Cogstate composite score; and change in executive function, as measured by BRIEF Cognitive Regulation Index (CRI)."
Answered by AI
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