An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1



Status:Available
Conditions:Other Indications
Therapuetic Areas:Other
Healthy:No
Age Range:2 - Any
Updated:3/15/2019
Contact:AstraZeneca Clinical Study Information Center
Email:information.center@astrazeneca.com
Phone:1-877-240-9479

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An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN)

This will be an open-label, single-arm, multicenter intermediate access protocol which
provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1
(NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any
alternative therapeutic options. All patients will continue to receive drug while they are
deriving clinical benefit .

Approximately 100 patients in the US will be treated as part of this protocol

Patients must have received a clinical diagnosis of NF1 and have inoperable,
progressive/symptomatic PN, where inoperable is defined as PN that cannot be surgically
completely removed without risk of substantial morbidity.

The population are patients with NF1 who have inoperable, progressive/symptomatic PN aged ≥
2years with onset of disease before they were 18 years and who have demonstrated an ability
to swallow whole capsules, who have no further treatment options and are not eligible for
clinical trials.

There is no maximum duration for selumetinib treatment. Patients may continue to receive
selumetinib as long as they continue to show clinical benefit, as judged by the treating
physician, and in the absence of unacceptable toxicity.

Once patients have been discontinued from treatment, other available treatment options will
be at the discretion of the physician

Inclusion Criteria:

1. Diagnosis of neurofibromatosis type 1 (NF1) and have inoperable,
progressive/symptomatic plexiform neurofibromas (PN)

2. Presence of inoperable PN , defined as a PN that cannot be surgically completely
removed without risk for substantial morbidity

3. Patients aged ≥2 with onset of disease before they were 18 years and a BSA ≥ 0.55 m2
who are able to swallow whole capsules. (approx. length 15.4 mm, diameter 5.4 mm). A
swallow test must be performed before requesting drug

4. Normal cardiac function defined as normal ejection fraction (ECHO, MUGA or cardiac
MRI) as per institutional normal and absence of prior heart disease

5. Adequate blood pressure as defined in line with local practice.

6. The patient has exhausted all available approved therapies as appropriate for NF1 with
inoperable progressive/symptomatic PN

7. Provision of a signed informed consent prior to any protocol specific procedures.
Patients already receiving selumetinib through single patient access who enroll in
this protocol must be reconsented and sign the consent form for this intermediate
access protocol.

8. For female patients of childbearing potential, have evidence of a post-menopausal
status, or a negative urinary or serum pregnancy test.

Exclusion Criteria:

1. Unresolved chronic toxicity ≥ CTCAE Grade 2 from previous therapy

2. Patients eligible for any ongoing clinical trials with selumetinib in the indication
in question

3. Ophthalmological conditions: Current or past history of retinal pigment epithelial
detachment (RPED)/central serous retinopathy (CSR) or retinal vein occlusion
Intraocular pressure (IOP) should not be > 21 mmHg for adults or outside the range of
normal for children or uncontrolled glaucoma (irrespective of IOP)

4. Male or female patients of reproductive potential and, as judged by the investigator,
are not employing an effective method of birth control.

5. Female patients who are breast-feeding.

6. Have evidence of any other significant clinical disorder or laboratory finding that,
as judged by the treating physician, makes it undesirable for the patient to
participate in the study.

7. Have any evidence of a severe or uncontrolled systemic disease (e.g. unstable or
uncompensated respiratory, cardiac, hepatic, or renal disease, active infection
(including hepatitis B, hepatitis C, HIV), active bleeding diatheses or renal
transplant

8. Have refractory nausea and vomiting, chronic gastrointestinal diseases (e.g.,
inflammatory bowel disease), or significant bowel resection that would adversely
affect the absorption / bioavailability of the orally administered study medication
We found this trial at
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Indianapolis, Indiana 46202
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800 Washington St
Boston, Massachusetts 02111
(617) 636-5000
Tufts Medical Center Tufts Medical Center is an internationally-respected academic medical center – a teaching...
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1719 East 19th Avenue
Denver, Colorado 80218
(303) 839-6000
Presbyterian - St. Luke's Medical Center Presbyterian/St. Luke's Medical Center and the Rocky Mountain Hospital...
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Durham, North Carolina 27710
(919) 684-8111
Duke University Younger than most other prestigious U.S. research universities, Duke University consistently ranks among...
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262 Danny Thomas Pl
Memphis, Tennessee 38105
(901) 495-3300
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
University of Utah Research is a major component in the life of the U benefiting...
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1600 Rockland Road
Wilmington, Delaware 19803
(302) 651-4200
Alfred I. duPont Hospital for Children Nemours began more than 70 years ago with the...
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Albany, New York 12208
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1500 East Medical Center Drive
Ann Arbor, Michigan 48109
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Bethesda, Maryland 20892
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Birmingham, Alabama 35233
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Boise, Idaho 83712
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Chicago, Illinois 60611
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Grand Rapids, Michigan 49503
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6621 Fannin St
Houston, Texas 77030
(832) 824-1000
Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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Minneapolis, Minnesota 55404
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1514 Jefferson Hwy.
New Orleans, Louisiana 70121
504-842-3000
Ochsner Medical Center Ochsner Medical Center is located near uptown New Orleans and includes acute...
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New York, New York 10065
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Oakland, California 94611
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Oklahoma City, Oklahoma 73104
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Saint Louis, Missouri 63104
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San Antonio, Texas 78207
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Shreveport, Louisiana 71103
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111 Michigan Ave NW
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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