Your session is about to expire
← Back to Search
Kinase Inhibitor
Selumetinib for Plexiform Neurofibroma
Phase 1
Waitlist Available
Research Sponsored by AstraZeneca
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male and female participants aged ≥ 12 to < 18 years at the time of signing the informed consent
Participants must have a BSA ≥ 1.3 and ≤ 2.5 m2
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (cycle 1 day 8 of each treatment period 1, 2 and 3); each treatment period 1 and 2 has 1 cycle (each cycle is 28 days). if needed, treatment period 3 will be started approximately 5 cycles later.
Awards & highlights
Study Summary
This trial is testing whether it's safe to give the drug selumetinib to teens with NF1 who have inoperable PN, and whether doing so under fasting or fed conditions affects GI toxicity. The goal is to find a dosing recommendation that maintains efficacy while being safe.
Who is the study for?
Adolescents aged 12-17 with Neurofibromatosis Type 1 (NF1) and inoperable Plexiform Neurofibromas (PN), who have symptoms or risk of complications. They must not have had recent major surgery, abnormal eye conditions, significant heart disease, poor liver or kidney function, previous MEKi treatment issues, unresolved toxicity from past treatments for NF1-PN, or any life-threatening illness.Check my eligibility
What is being tested?
The trial is testing the drug Selumetinib to see how taking it with a low-fat meal affects its absorption and gastrointestinal side effects in young patients. The goal is to find an effective dose that can be taken with food while maintaining safety and efficacy.See study design
What are the potential side effects?
Selumetinib may cause gastrointestinal issues like nausea or diarrhea when taken without food. Other potential side effects include skin changes, vision problems due to optic nerve damage, fatigue, rash, muscle pain and heart dysfunction.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 12 and 17 years old.
Select...
My body surface area is between 1.3 and 2.5 square meters.
Select...
I have NF1 with inoperable PN and meet another NF1 diagnostic criterion.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (cycle 1 day 8 of each treatment period 1, 2 and 3); each treatment period 1 and 2 has 1 cycle (each cycle is 28 days). if needed, treatment period 3 will be started approximately 5 cycles later.
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (cycle 1 day 8 of each treatment period 1, 2 and 3); each treatment period 1 and 2 has 1 cycle (each cycle is 28 days). if needed, treatment period 3 will be started approximately 5 cycles later.
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Assessing change of Gastrointestinal toxicity diary: Modified Bristol Stool Form Scale for Children (mBSFS-C)
Assessing change of Gastrointestinal toxicity diary: Nausea and Vomiting Symptom Rating Scale (adapted from the Children's Cancer and Leukaemia Group)
Gastrointestinal Adverse Events graded by CTCAE Ver 5.0 (Grade 1 to 5)
+3 moreSecondary outcome measures
Adverse events(AEs) graded by CTCAE Version 5.0
Area under the concentration-time curve from time zero to time of last measurable concentration (AUClast) of selumetinib and N-desmethyl selumetinib
Maximum Peak plasma concentration (Cmax) of selumetinib and N-desmethyl selumetinib
+2 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: selumetinib single armExperimental Treatment1 Intervention
This is a sequential study consisting of a screening period lasting up to 28 days, a 28 day (1 cycle) treatment period (T1) in a fed state, a 7 day washout period, a further 1 cycle treatment period (T2) in a fasted state and an extension to T2 until results from the primary analysis are available. During Treatment Period 1 and 2 all participants will receive selumetinib (25 mg/m2 bid). If a third treatment period (T3) is required, participants will enter a 7 day washout period followed by a treatment period in a fed state at an adjusted dose for 3 cycles.
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?
AstraZenecaLead Sponsor
4,262 Previous Clinical Trials
288,595,218 Total Patients Enrolled
Merck Sharp & Dohme LLCIndustry Sponsor
3,887 Previous Clinical Trials
5,054,950 Total Patients Enrolled
Study physician Study physician, MDStudy DirectorAstraZeneca
1 Previous Clinical Trials
44 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have previously been treated with a MEK inhibitor.I have been diagnosed with NF1 and have a tumor that cannot be surgically removed.I stopped or lowered my MEKi treatment due to side effects.I have no active cancer treatments, except for certain skin cancers or cancers I've been free from for 2+ years.I have ongoing side effects from previous NF1-PN treatment, but no severe stomach issues.I need treatment for NF1 and inoperable PN due to symptoms or risk of serious complications.I haven't had major surgery in the last 4 weeks, except for vascular access, and don't plan any during the treatment.I am between 12 and 17 years old.My body surface area is between 1.3 and 2.5 square meters.I will stop taking multivitamins with vitamin E 7 days before starting selumetinib.I may have a brain tumor or another cancer needing chemo or radiation.I have NF1 with inoperable PN and meet another NF1 diagnostic criterion.I need treatment for NF1 and inoperable PN due to symptoms or risk of serious complications.I have a serious heart condition as listed in the study details.I have previously been treated with a MEK inhibitor.My liver tests are within normal limits, except for Gilbert syndrome.I don't have any health issues that could risk my safety in the study.My kidney function is reduced, with specific creatinine levels based on my age.I am between 12 and 17 years old and can give informed consent.
Research Study Groups:
This trial has the following groups:- Group 1: selumetinib single arm
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 FDA?
"Selumetinib is a relatively new drug and has thus received a score of 1. This implies that there is only scant evidence to back up its efficacy as well as any claims about safety."
Answered by AI
What are the objectives of this experimental trial?
"This medical trial will survey the number of patients who take gastrointestinal medications, from their initial screening to 30 days after their last dose. Secondary goals include evaluating selumetinib and N-desmethyl selumetinib's time to maximum concentration (tmax), time to last measurable concentration (tlast) as well as area under the curve from zero to tlast (AUClast). All results are compared between fed and fasted conditions."
Answered by AI
Are individuals aged 50 and above being recruited for participation in this clinical trial?
"This clinical trial has set the lower bound of eligibility at 12 years old and an upper limit of 17 years."
Answered by AI
Are there opportunities for me to join this medical experiment?
"In order to be applicable, subjects that wish to enrol must have a diagnosis of neurofibroma and range in age from 12 years old up until 17. The research team is seeking 24 total participants for the trial."
Answered by AI
To what extent has Selumetinib been explored in biomedical research?
"Presently, there are 34 active clinical trials for Selumetinib with 5 in the third Phase. While most of these tests are conducted in Philadelphia, Pennsylvania; a total of 1005 medical centres around the country offer this trial."
Answered by AI
What is the maximum capacity of participants for this experiment?
"The deadline to participate in this trial has passed. The study was posted on July 21, 2021 and last updated November 10th 2022. Currently, there are 56 clinical trials researching neurofibroma and 34 studies looking into Selumetinib that are accepting participants."
Answered by AI
Are there any openings left for prospective participants in this experiment?
"Data posted to clinicaltrials.gov reveals that this medical study is not currently recruiting participants as of November 10th, 2022; however, 90 other trials are open for enrolment at present. This trial was initially announced on July 21st 2021."
Answered by AI
Does this research represent a pioneering effort in its field?
"Since 2007, Selumetinib has been the focus of considerable research. The first investigation was sponsored by AstraZeneca and included 58 participants. Subsequently, it obtained its Phase 1 drug approval which paved the way for 34 current studies across 156 cities in 33 countries."
Answered by AI
Who else is applying?
What state do they live in?
Georgia
How old are they?
18 - 65
What portion of applicants met pre-screening criteria?
Did not meet criteria
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger