An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1
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|ClinicalTrials.gov Identifier: NCT03259633|
Expanded Access Status : Approved for marketing
First Posted : August 24, 2017
Last Update Posted : May 1, 2020
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
|Condition or disease||Intervention/treatment|
|NF type1 With Inoperable Plexiform Neurofibromas||Drug: Selumetinib|
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
|Study Type :||Expanded Access|
|Expanded Access Type :||Intermediate-size Population|
|Official Title:||An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN)|
- Drug: Selumetinib
open-label, single-arm, multicenter intermediate access protocol
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03259633
|United States, Louisiana|
|New Orleans, Louisiana, United States, 12345|
|Principal Investigator:||Miriam Bornhorst, MD||Investigator|