AstraZeneca

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

Official Title: 
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN)
Brief Summary: 
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
Detailed Description: 

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

Available
EA Type
Intermediate-size Population
NF type1 With Inoperable Plexiform Neurofibromas

Drug: Selumetinib
open-label, single-arm, multicenter intermediate access protocol

Eligibility Criteria: 

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

Eligibility Gender: 
All
Eligibility Age: 
Minimum: 2 Years
Maximum: 130 Years
Countries: 
United States
Locations: 

Children's of Alabama
Birmingham, Alabama, 35233

Available

Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234

Available

Loma Linda University
Loma Linda, California, 92354

Available

Kaiser Permanente Oakland Medical Center
Oakland, California, 94611

Available

CHOC Children's of Orange County
Orange, California, 92868

Available

Presbyterian/St. Luke's Medical Center
Denver, Colorado, 80218

Available

Alfred I. DuPont Hospital for Children
Wilmington, Delaware, 19803

Available

Children's National Medical Center
Washington, District of Columbia, 20010

Available

St. Luke's Health System
Boise, Idaho, 83712

Available

Lurie Children's Hospital
Chicago, Illinois, 60611

Available

Indiana University Health
Indianapolis, Indiana, 46202

Available

Our Lady of the Lake - St. Jude Affiliate Clinic
Baton Rouge, Louisiana, 70808

Available

Ochsner Medical Center
New Orleans, Louisiana, 70121

Available

Willis-Knight Cancer Center
Shreveport, Louisiana, 71103

Available

Maine Medical Center
Portland, Maine, 04101

Available

National Institute of Health
Bethesda, Maryland, 20892

Available

Tufts Medical Center
Boston, Massachusetts, 02111

Available

Michigan Medicine
Ann Arbor, Michigan, 48109

Available

Spectrum Health
Grand Rapids, Michigan, 49503

Available

Children's Minnesota
Minneapolis, Minnesota, 55404

Available

University of Mississippi Medical Center
Jackson, Mississippi, 39213

Available

SSM Cardinal Glennon Children's Hospital
Saint Louis, Missouri, 63104

Available

Washington University in St. Louis
Saint Louis, Missouri, 63110

Available

Albany Medical Center
Albany, New York, 12208

Available

Memorial Sloan Kettering
New York, New York, 10065

Available

Duke University
Durham, North Carolina, 27710

Available

University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106

Available

Jimmy Everest Cancer Center
Oklahoma City, Oklahoma, 73104

Available

The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Available

University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224

Available

PrismaHealth Upstate
Greenville, South Carolina, 29605

Available

Research Site
Memphis, Tennessee, 38105

Available

Vanderbilt University Medical Center
Nashville, Tennessee, 37232

Available

Dell Children's Medical Center
Austin, Texas, 78723

Available

Cook Children's Hospital
Fort Worth, Texas, 76104

Available

Center for Oncology and Blood Disorders
Houston, Texas, 77030

Available

Texas Children's Hospital
Houston, Texas, 77030

Available

Children's Hospital of San Antonio
San Antonio, Texas, 78207

Available

University of Utah
Salt Lake City, Utah, 84112

Available

Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226

Available

Contacts: 

AstraZeneca Clinical Study Information Center
1-877-240-9479
information.center@astrazeneca.com

Medicine Access Clinigen Group
medicineaccess@clinigengroup.com

Miriam Bornhorst, MD
Principal Investigator
Investigator

AstraZeneca
EA Number: 
Keywords: 
Neurofibromatosis type1, Plexiform Neurofibromas, inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options, mutation in the NF1 gene, Selumetinib, Early Access, Intermediate Access Protocol
MeSH Terms: 
Neurofibromatoses, Neurofibromatosis 1, Neurofibroma, Neurofibroma, Plexiform
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