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Kinase Inhibitor
Selumetinib for Neurofibromatosis
Phase 2
Waitlist Available
Led By Bruce R Korf
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
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to up to 1 year
Awards & highlights
Study Summary
This trial looks at whether selumetinib can stop the growth of tumors in neurofibromatosis type 1 patients by blocking enzymes needed for cell growth.
Eligible Conditions
- Neurofibromatosis Type 1
- Optic Nerve Glioma
- Neurofibroma
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to up to 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to up to 1 year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Median Best Response of Cutaneous Neurofibromas in Participants With at Least One Restaging Evaluation
Secondary outcome measures
Change in the Number of Cutaneous Neurofibromas
Other outcome measures
Changes in Levels of pERK and pAKT Assessed by Quantitative Enzyme-linked Immunosorbent Assay
Changes in Pathway Biomarkers Assessed by Kinome Analysis
Changes in Skin Related Morbidity
Side 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
1Treatment groups
Experimental Treatment
Group I: Treatment (selumetinib sulfate)Experimental Treatment2 Interventions
Patients receive selumetinib sulfate PO twice daily (BID) on days 1-28. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients who experience a volume decrease in the target cutaneous neurofibromas may continue treatment for 12 additional cycles.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Selumetinib Sulfate
2017
Completed Phase 2
~80
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)Lead Sponsor
13,658 Previous Clinical Trials
40,924,476 Total Patients Enrolled
3 Trials studying Neurofibromatosis
284 Patients Enrolled for Neurofibromatosis
Bruce R KorfPrincipal InvestigatorUniversity of Alabama at Birmingham Cancer Center
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have not taken any medications that help increase your platelet or white cell count in the past week, and you cannot take them during the study.Your blood and liver function levels must be within specific ranges.You have a lot of skin bumps from neurofibromatosis that bother you because they itch or make you feel uncomfortable.You cannot have taken experimental drugs or biological treatments before.You have a serious ongoing illness, such as a severe infection, heart problems, or mental health issues, that could make it hard for you to follow the study rules.You have been treated with a drug like selumetinib before.You have certain eye conditions like retinal pigment epithelial detachment, retinal vein occlusion, or uncontrolled glaucoma, or your eye exam shows significant problems.You have a tumor related to NF1 that needs treatment with chemotherapy, radiation, or surgery.You are taking strong medications that affect the liver.You must have a confirmed genetic mutation or a diagnosis of neurofibromatosis 1 based on specific criteria.You cannot swallow pills.You have ongoing severe nausea and vomiting, long-term stomach or bowel problems, or have had major surgery on your stomach or intestines that would affect how your body absorbs medication.You have a history of serious heart problems, including recent heart attack, uncontrolled chest pain, severe heart failure, or certain heart conditions that affect the heart's pumping function.You have a strong allergic reaction to selumetinib or any of its ingredients.You have ongoing severe side effects from previous NF1 treatment.You have many skin neurofibromas and specific signs related to NF1, like café-au-lait spots, freckling, optic glioma, Lisch nodules, bony lesions, and a close family member with NF1, and have had tumors confirmed by a biopsy.You should be able to perform daily activities without being restricted by your health condition.You can participate in the trial even if you haven't had treatment for your plexiform neurofibromas before.You must wait at least 6 weeks after any radiation treatment before joining the study, and the neurofibromas to be treated should not be in the same area where you had radiation before.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment (selumetinib sulfate)
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.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are we currently recruiting people for this clinical trial?
"This study is not seeking participants at the moment. It was originally posted on April 11th, 2017 and updated for the last time on September 9th, 2022. 59 other trials are currently looking for patients with optic nerve glioma and 105 studies involving Selumetinib Sulfate are actively recruiting."
Answered by AI
What is the FDA's decision on Selumetinib Sulfate?
"Selumetinib Sulfate has been given a safety rating of 2. This is due to the fact that, while there is evidence suggesting Selumetinib Sulfate is safe, there is no clinical data yet supporting its efficacy."
Answered by AI
Are there any other notable examples of Selumetinib Sulfate research?
"Currently, there are 105 different clinical trials being run that involve Selumetinib Sulfate. Out of those active investigations, 34 have reached Phase 3. However, the majority of these tests are conducted in Sioux Falls, South dakota. In total, 10462 locations across the world are running some form of trial for this medication."
Answered by AI
How many people are eligible to participate in this research?
"This particular trial is no longer recruiting patients. However, as of right now, there are 59 other trials for optic nerve glioma and 105 for Selumetinib Sulfate that still need participants."
Answered by AI
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