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MEK Inhibitor
Selumetinib for Neurofibromatosis
Phase 2
Waitlist Available
Led By Brigitte C Widemann, M.D.
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 have a PN that is inoperable and causes morbidity or has the potential to cause morbidity
Patients must be able to undergo serial MRI scans for response evaluation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up rior to cycle 5, 9, 13, 17, 21, 25 then after every 6 cycles
Awards & highlights
Study Summary
This trial is testing if the drug selumetinib helps treat neurofibromatosis type 1 (NF1) by causing plexiform neurofibromas (PNs) to shrink or slowing down their growth.
Who is the study for?
Adults over 18 with Neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs), which are tumors along nerves causing health issues or growing. Participants must not be on other investigational drugs, have certain NF1-related tumors needing treatment, uncontrolled illnesses, or be pregnant/breastfeeding without contraception. They should be able to swallow capsules and undergo MRI scans.Check my eligibility
What is being tested?
The trial is testing Selumetinib's effectiveness on shrinking or slowing the growth of PNs in NF1 patients. It involves taking Selumetinib capsules twice daily for multiple 28-day cycles, as long as it's tolerated and the disease doesn't progress. The study includes regular visits for medical tests to monitor effects.See study design
What are the potential side effects?
Potential side effects of Selumetinib may include fatigue, nausea, vomiting, diarrhea, skin rash, hypertension (high blood pressure), vision changes due to eye inflammation, muscle pain and heart problems. Side effects can vary from person to person.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My tumor cannot be removed by surgery and is causing or could cause health problems.
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I can undergo multiple MRI scans for monitoring.
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I am 18 years old or older.
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I can take care of myself and am up and about more than half of my waking hours.
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My organ and bone marrow functions are normal.
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I have recovered from side effects of my previous treatments.
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I have a confirmed diagnosis of NF1 either through genetic testing or clinical criteria.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at each response evaluation visit
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at each response evaluation visit
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Determine objective response rate
Secondary outcome measures
Analyze bone marrow derived precursor cells and cytokines before and after treatment
Analyze paired biopsies with mechanisms of response and resistance
Evaluate immune infiltrate of PN and Peripheral blood for circulating tumor cells
+5 moreSide effects data
From 2012 Phase 2 trial • 37 Patients • NCT0108521475%
Diarrhea
50%
Fatigue
47%
Anemia
47%
Rash acneiform
44%
Hypoalbuminemia
44%
Edema, limbs
39%
Aspartate aminotransferase increased
33%
Neutrophil count decreased
33%
White blood cell decreased
31%
Nausea
31%
Vomiting
28%
Platelet count decreased
25%
CPK increased
25%
Hypomagnesemia
22%
Hypertension
19%
Hypophosphatemia
19%
Hypocalcemia
19%
Hyponatremia
19%
Edema, face
17%
Dry skin
17%
Alanine aminotransferase increased
14%
Skin and subcutaneous tissue disorders - Other
14%
Hypokalemia
14%
Creatinine increased
14%
Back pain
14%
Dyspnea
14%
Lymphocyte count decreased
11%
Pain
11%
Fever
11%
Localized edema
11%
Peripheral sensory neuropathy
11%
Hyperkalemia
11%
Dizziness
11%
Abdominal pain
8%
Hypoglycemia
8%
Acute kidney injury
8%
Anorexia
8%
Death, NOS
8%
Periorbital edema
8%
Skin hypopigmentation
8%
Pain in extremity
8%
Cough
8%
Insomnia
8%
Alkaline phosphatase increased
8%
Dry mouth
8%
Sepsis
6%
Renal and urinary disorders - Other
6%
Dehydration
6%
Musculoskeletal and connective tissue disorder - Other, Rhabdomyolysis
6%
Hypernatremia
6%
Blood and lymphatic system disorders - Other
6%
Hypercalcemia
6%
Metabolism and nutrition disorders - Other
6%
Chills
6%
Hypotension
6%
Myalgia
6%
Arthralgia
6%
Upper respiratory infection
6%
Headache
6%
Sinusitis
6%
Generalized muscle weakness
6%
Gastrointestinal disorders - Other
6%
Gastroesophageal reflux disease
3%
Confusion
3%
Vaginal inflammation
3%
Pruritus
3%
Febrile neutropenia
3%
Flu like symptoms
3%
Hepatic failure
3%
Skin infection
3%
Fall
3%
Fracture
3%
Skin and subcutaneous tissue disorders - Other, Angular cheilitis, unilateral
3%
Adult respiratory distress syndrome
3%
Renal and urinary disorders - Other, Acute renal failure
3%
INR increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
AZD6244 (Selumetinib) Treatment
Trial Design
1Treatment groups
Experimental Treatment
Group I: 1Experimental Treatment1 Intervention
Selumetinib 50 mg BID daily
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Selumetinib
2010
Completed Phase 2
~2050
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)Lead Sponsor
13,662 Previous Clinical Trials
40,925,845 Total Patients Enrolled
Brigitte C Widemann, M.D.Principal InvestigatorNational Cancer Institute (NCI)
20 Previous Clinical Trials
3,574 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My tumor cannot be removed by surgery and is causing or could cause health problems.I am willing to have needle biopsies of my tumor before and during treatment.I can undergo multiple MRI scans for monitoring.I am 18 years old or older.I can take care of myself and am up and about more than half of my waking hours.My organ and bone marrow functions are normal.I have NF1 and need treatment with chemo or surgery.I do not have any uncontrolled illnesses like heart failure or active infections.I have previously been treated with a MEK 1/2 inhibitor.I have ongoing issues with nausea, vomiting, or serious gut problems.My high blood pressure is not under control.I have a known eye condition.I haven't had major surgery in the last 4 weeks.I have recovered from side effects of my previous treatments.I have not had any treatment for my peripheral neuropathy.I cannot swallow pills.I have a known heart condition.I have lasting side effects from previous NF1 treatment that are moderate or worse.I have a confirmed diagnosis of NF1 either through genetic testing or clinical criteria.
Research Study Groups:
This trial has the following groups:- Group 1: 1
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.
Has Selumetinib received regulatory clearance from the US Food and Drug Administration?
"Due to the Phase 2 nature of this trial, Selumetinib's safety was assigned a grade of 2 based on our internal evaluation. This indicates that there is evidence backing its security but no support for efficacy as yet."
Answered by AI
Has Selumetinib been evaluated in other clinical experiments?
"34 clinical trials examining Selumetinib are currently in progress, with 5 entering Phase 3. Philadelphia is one of many sites hosting these trials, and there are over a thousand locations offering this medication to patients worldwide."
Answered by AI
Is this investigation innovative in nature?
"At present, there are 34 ongoing studies on Selumetinib in 156 cities and 33 nations. This drug was first tested back in 2007 when AstraZeneca initiated an experiment with 58 volunteers that achieved its Phase 1 approval. Since then, 60 additional research projects have been completed."
Answered by AI
Is this trial actively recruiting new participants?
"Clinicaltrials.gov's records suggest that this medical trial is no longer seeking participants, with the initial post dating back to July 1st 2016 and the last update on November 24th 2022. Fortunately, there are 90 other studies currently enrolling patients."
Answered by AI
What is the current enrolment rate for this research trial?
"At present, the trial is not accepting new recruits. The initial posting was on July 1st 2016 and it was last updated November 24th 2022. If you are seeking alternative studies, there are currently 56 clinical trials actively recruiting patients with neurofibroma and 34 for Selumetinib."
Answered by AI
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