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MEK Inhibitor

AZD6244 for Neurofibromatosis

Phase 1 & 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
Performance status: Karnofsky performance level >70% for patients >16 years old, Lansky performance >70% for children <16 years old
Renal function: creatinine clearance or GFR >=60ml/min/1.73 m(2) or normal serum creatinine based on age
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at each response evaluation
Awards & highlights

Study Summary

This trial is testing a drug to see if it can prevent, slow down the growth, or shrink tumors in children and young adults with plexiform neurofibromas.

Who is the study for?
This trial is for children and young adults aged 2 to 18 with inoperable plexiform neurofibromas, a type of nerve tumor. Participants must be able to swallow capsules, have no severe allergies to the drug's ingredients, and not require surgery within the first three months. They should also have normal organ function and not be on certain other medications or treatments.Check my eligibility
What is being tested?
The study tests AZD6244 hydrogen sulfate's ability to stop growth or shrink inoperable plexiform neurofibromas in children. The treatment involves taking the drug twice daily over cycles of 28 days with regular health checks, blood tests, urine tests, and imaging studies.See study design
What are the potential side effects?
Potential side effects may include skin sensitivity requiring sun avoidance and sunscreen use; digestive issues that could affect absorption; increased risk if there are existing conditions like uncontrolled diseases or infections; possible impact on vision; and potential interaction with grapefruit products.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am mostly active and can care for myself.
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My kidney function is normal or only mildly impaired.
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I have been diagnosed with or tested positive for NF1.
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My blood pressure is within the normal range for my age, height, and gender.
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My liver tests are within the required range.
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My heart pumps well and my heart rhythm is normal.
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I am between 3 and 18 years old and can swallow capsules.
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I am between 2 and 18 years old, can swallow capsules, and my body surface area is at least 0.55 m2.
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I have NF1 with tumors that can't be removed and may cause serious health problems.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 treatment cycles
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 treatment cycles for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Ph1: Determine MTD and extended tolerability
Ph2: Evaluate the confirmed partial and complete response rate of selumetinib in children and young adults with NF1 and inoperable PN.
Secondary outcome measures
Define toxicities
Measure adherence of chronic dosing
Ph2: Evaluate confirmed partial and complete response rate, and duration of response.
+4 more

Side effects data

From 2020 Phase 2 trial • 334 Patients • NCT01248247
50%
Rash Acneiform
36%
Diarrhea
32%
Nausea
32%
Dry skin
27%
Anorexia
23%
Increased blood bilirubin
23%
Vomiting
23%
Increased AST
14%
Oral mucositis
14%
Weight loss
14%
Other skin and subcutaneous tissue disorders, specify
14%
Rash Maculopapular
14%
Epistaxis
9%
Fatigue
9%
Hyperglycemia
9%
Increased alkaline phosphatase
5%
Dry mouth
5%
Lung infection
5%
Pruritus
5%
Hyponatremia
5%
Dyspnea
5%
Palmar-plantar erythrodysesthesia syndrome
5%
Multi-organ failure
5%
Hypoalbuminemia
5%
Dysgeusia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm 1 - Erlotinib
Arm 3 - AZD6244 + MK-2206
Arm 4 - Sorafenib
Arm 2 - Erlotinib + MK-2206

Trial Design

2Treatment groups
Experimental Treatment
Group I: Arm 2Experimental Treatment1 Intervention
Phase 2: AZD6244 PO BID x 28 DAYS
Group II: Arm 1Experimental Treatment1 Intervention
Phase 1: AZD6244 PO BID x 28 DAYS
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AZD6244
2011
Completed Phase 2
~1570

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor
13,609 Previous Clinical Trials
40,915,547 Total Patients Enrolled
3 Trials studying Neurofibromatosis
284 Patients Enrolled for Neurofibromatosis
Brigitte C Widemann, M.D.Principal InvestigatorNational Cancer Institute (NCI)
20 Previous Clinical Trials
3,511 Total Patients Enrolled
1 Trials studying Neurofibromatosis
10 Patients Enrolled for Neurofibromatosis

Media Library

AZD6244 Hydrogen Sulfate (MEK Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT01362803 — Phase 1 & 2
Neurofibromatosis Research Study Groups: Arm 2, Arm 1
Neurofibromatosis Clinical Trial 2023: AZD6244 Hydrogen Sulfate Highlights & Side Effects. Trial Name: NCT01362803 — Phase 1 & 2
AZD6244 Hydrogen Sulfate (MEK Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01362803 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Could individuals aged 60 and above participate in the experiment?

"According to the official requirements for participation, this study only accepts patients aged between 2 and 18 years old."

Answered by AI

Is this experiment currently open to participants?

"Unfortunately, this medical trial is no longer recruiting participants. The clinical trial was initiated on September 21st 2011 and last updated November 19th 2022. If you are looking for similar studies, there are currently 58 trials researching watson syndrome and 34 investigating AZD6244 that have open enrollment periods."

Answered by AI

At which venues is this trial being administered?

"This clinical study is available at seven distinct medical sites, including Children's National Medical Center in Washington D.C., Cincinnati Children's Hospital Medical Centre in Ohio and the prestigious NIH Clinical Center located in Bethesda Maryland."

Answered by AI

Have any other investigations taken place involving AZD6244?

"In 2007, AZD6244 was initially studied at Research Site. 60 completed trials have taken place since then and 34 live studies are currently active - many of these located in the District of Columbia's capital, Washington DC."

Answered by AI

Which population is most suited to participate in this clinical study?

"This medical trial is seeking to enroll ninety-nine individuals between the ages of two and eighteen who suffer from Watson Syndrome. To qualify, candidates must possess at least six cafe au lait macules (with dimensions being 0.5cm in prepubertal participants or 1.5 cm for post pubertal patients), freckling within their armpits or groin, optic glioma, Lisch nodules, a distinctive bone abnormality in either their sphenoid or long bones as well as body surface area greater than 0.55 m2 if they are able to swallow whole capsules. This age range was specifically chosen"

Answered by AI

How does this research endeavor seek to benefit patients?

"This three-cycle clinical trial seeks to assess the efficacy of selumetinib in treating NF1 and inoperable PN among children and young adults. Additional aims include measuring long term safety, evaluating growth rate of PN, and determining objective response rates."

Answered by AI

Is there a cap on the number of participants in this medical trial?

"This research is no longer recruiting participants. It first appeared on September 21st 2011 and was last updated November 19th 2022. However, 58 clinical studies are accepting Watson Syndrome patients while 34 centres are enrolling candidates for AZD6244 trials."

Answered by AI

Is this research effort a pioneering endeavor?

"AZD6244 has been in clinical trials since 2007, when AstraZeneca initiated the first study with 58 participants. Thanks to positive results from that trial, AZD6244 was granted Phase 1 drug approval and is now being tested in 34 active studies across 33 different countries and 156 cities."

Answered by AI
Recent research and studies
~6 spots leftby Jan 2025