Ignite Creation Date:
2025-12-25 @ 2:49 AM
Ignite Modification Date:
2026-03-11 @ 12:38 PM
Study NCT ID:
NCT03259633
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2020-05-01
First Post:
2017-08-21
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1
Sponsor:
AstraZeneca
Organization:
Study Overview
Official Title:
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 With Inoperable, Progressive/Symptomatic Plexiform Neurofibromas (PN)
Status:
APPROVED_FOR_MARKETING
Status Verified Date:
2020-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
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 Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| D1346R00002 | OTHER | Astra Zeneca | View |