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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 below
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I am between 12 and 17 years old.
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My body surface area is between 1.3 and 2.5 square meters.
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I have NF1 with inoperable PN and meet another NF1 diagnostic criterion.

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.
This trial's timeline: 3 weeks for screening, Varies for treatment, and 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. for reporting.

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 more
Secondary 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 more

Side effects data

From 2012 Phase 2 trial • 37 Patients • NCT01085214
75%
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
~2140

Find a Location

Who is running the clinical trial?

AstraZenecaLead Sponsor
4,287 Previous Clinical Trials
288,620,126 Total Patients Enrolled
Merck Sharp & Dohme LLCIndustry Sponsor
3,907 Previous Clinical Trials
5,065,994 Total Patients Enrolled
Study physician Study physician, MDStudy DirectorAstraZeneca
1 Previous Clinical Trials
36 Total Patients Enrolled

Media Library

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