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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
Must not have
Patients with NF1 related tumors requiring treatment with chemotherapy or surgery
Uncontrolled intercurrent illness including ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, active bleeding diatheses or renal transplant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up rior to cycle 5, 9, 13, 17, 21, 25 then after every 6 cycles
Awards & highlights
No Placebo-Only Group

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.
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.
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 below
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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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have NF1 and need treatment with chemo or surgery.
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I do not have any uncontrolled illnesses like heart failure or active infections.
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I cannot swallow pills.
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I have previously been treated with a MEK 1/2 inhibitor.
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I have ongoing issues with nausea, vomiting, or serious gut problems.
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My high blood pressure is not under control.
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I have a known eye condition.
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I have a known heart condition.
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I have lasting side effects from previous NF1 treatment that are moderate or worse.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at each response evaluation visit
This trial's timeline: 3 weeks for screening, Varies for treatment, and at each response evaluation visit for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Determine objective response rate
Secondary study objectives
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 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%
Hyponatremia
19%
Hypocalcemia
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%
Anorexia
8%
Hypoglycemia
8%
Acute kidney injury
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%
Hypernatremia
6%
Metabolism and nutrition disorders - Other
6%
Blood and lymphatic system disorders - Other
6%
Renal and urinary disorders - Other
6%
Hypercalcemia
6%
Dehydration
6%
Musculoskeletal and connective tissue disorder - Other, Rhabdomyolysis
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%
Vaginal inflammation
3%
Confusion
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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active 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
~2080

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor
13,907 Previous Clinical Trials
41,012,108 Total Patients Enrolled
3 Trials studying Neurofibromatosis
290 Patients Enrolled for Neurofibromatosis
Brigitte C Widemann, M.D.Principal InvestigatorNational Cancer Institute (NCI)
20 Previous Clinical Trials
3,574 Total Patients Enrolled
1 Trials studying Neurofibromatosis
10 Patients Enrolled for Neurofibromatosis

Media Library

Selumetinib (MEK Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT02407405 — Phase 2
Neurofibromatosis Research Study Groups: 1
Neurofibromatosis Clinical Trial 2023: Selumetinib Highlights & Side Effects. Trial Name: NCT02407405 — Phase 2
Selumetinib (MEK Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02407405 — Phase 2
~0 spots leftby Jan 2025
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