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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03259633
Expanded Access Status : Available
First Posted : August 24, 2017
Last Update Posted : November 9, 2018
Information provided by (Responsible Party):

August 21, 2017
August 24, 2017
November 9, 2018
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1

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

Expanded Access
Intermediate-size Population
Drug: Selumetinib
open-label, single-arm, multicenter intermediate access protocol
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Contact: AstraZeneca Clinical Study Information Center 1-877-240-9479
Contact: Medicine Access Clinigen Group
United States
Not Provided
Principal Investigator: Miriam Bornhorst, MD Investigator
November 2018