Ignite Creation Date:
2024-10-26 @ 3:41 PM
Last Modification Date:
2024-10-26 @ 3:41 PM
Study NCT ID:
NCT06621082
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-20
Brief Title:
The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate Capsule
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Prospective One-arm Phase II Clinical Study of Smeitinib Hydrosulfate Capsules for the Treatment of Patients With Type I Neurofibromatosis After Surgery
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
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 study focused on patients with type I neurofibromatosis undergoing surgical treatment who currently lack effective drug therapy and have a high recurrence rate after surgical resection For patients with small solid tumors limited space and no invasion of the brain spine and other important organs surgical treatment is the main treatment As a MEK inhibitor Smetinib bisulfate capsule can induce tumor shrinkage and reduce postoperative recurrence by selectively binding mitogen-activated protein kinase MEK 12 protein to block the mitogen-activated protein kinaseextracellular signal regulatory kinase signaling pathway that regulates key cell responses The purpose of this study was to treat patients with type I neurofibromatosis with indications of surgery with the drug smetinib bisulfate after surgical treatment observe the therapeutic effect of the drug in stages consolidate the postoperative effect and reduce the recurrence rate In this study progression-free survival PFS after postoperative drug treatment was used as the main outcome index and duration of remission DOR and objective response rate ORR were used as secondary outcome indicators to investigate the efficacy of the use of Smetinib hydrosulfate capsule on tumor control reduction of recurrence rate and stability of efficacy in patients with type I neurofibromatosis after surgery
Detailed Description:
Neurofibromatosis NF has been included in the list of rare diseases in many countries including China of which 96 is NF1 subtype NF1 clinical manifestations are diverse involve multiple systems can cause respiratory obstruction spinal cord compression motor dysfunction and other serious complications Plexiform neurofibroma PN occurs in 30-50 of patients with NF1 PN progresses rapidly is associated with severe physical defects is highly disabling and is at risk of malignancy According to the 2023 edition of the Multidisciplinary Guidelines for the Diagnosis and Treatment of type I neurofibromatosis NF1 patients are more likely than the normal population to develop a variety of benign and malignant tumors including pNF CNF MPNST and OPG Attention should be paid to the early identification and monitoring of these tumors The possibility of MPNST should be highly vigilant for neurofibromas with growth acceleration pain and texture hardening At the same time systemic evaluation should be performed and early surgery should be performed as far as possible for patients without signs of distant metastasis while radiotherapy chemotherapy and targeted therapy can be selected for patients with distant metastasis
neurofibromatosis type 1 NF1 is an autosomal dominant disorder in which 50 of patients have familial inherited mutations and 50 have sporadic mutations NF1 gene encodes neurofibrin down-regulates the activity of Ras-Raf pathway and inhibits cell proliferation Neurofibrin defects can lead to overactivation of the RAS pathway resulting in uncontrolled cell proliferation in patients with NF1 At present surgery is the most commonly used and most important treatment for neurofibromatosis and neurofibroma has the characteristic of growing along the nerve root so it is difficult to solve all the lesions through surgery The lesions consist of a wide range of nerve and vascular tissues mixed with normal tissues and the surgical resection is difficult and bleeding is frequent and the recurrence after incomplete resection is as high as 50 For NF1 in the head and neck some patients require a second operation one year after surgery and the proportion of patients with partial resection undergoing a second operation is higher than that of patients with subtotal resection Five years after surgery more than 50 of patients needed a second operation
As a MEK inhibitor Smetinib bisulfate capsule can induce tumor shrinkage by selectively binding mitogen-activated protein kinase MEK 12 protein to block the mitogen-activated protein kinaseextracellular signal regulatory kinase signaling pathway that regulates key cellular responses To create conditions for disease control radical surgical resection reducing postoperative recurrence and reducing complications Based on the targeted therapy of smeitinib bisulfate capsule this study evaluated the treatment of NF1 patients after surgical treatment and evaluated the effect of tumor shrinkage after medication by monitoring the effect and duration of solid tumor shrinkage of patients And the postoperative recurrence time so as to verify the effectiveness of smeitinib bisulfate capsule in reducing the recurrence rate of patients after NF1
neurofibromatosis type 1 NF1 is an autosomal dominant disorder in which 50 of patients have familial inherited mutations and 50 have sporadic mutations NF1 gene encodes neurofibrin down-regulates the activity of Ras-Raf pathway and inhibits cell proliferation Neurofibrin defects can lead to overactivation of the RAS pathway resulting in uncontrolled cell proliferation in patients with NF1 At present surgery is the most commonly used and most important treatment for neurofibromatosis and neurofibroma has the characteristic of growing along the nerve root so it is difficult to solve all the lesions through surgery The lesions consist of a wide range of nerve and vascular tissues mixed with normal tissues and the surgical resection is difficult and bleeding is frequent and the recurrence after incomplete resection is as high as 50 For NF1 in the head and neck some patients require a second operation one year after surgery and the proportion of patients with partial resection undergoing a second operation is higher than that of patients with subtotal resection Five years after surgery more than 50 of patients needed a second operation
As a MEK inhibitor Smetinib bisulfate capsule can induce tumor shrinkage by selectively binding mitogen-activated protein kinase MEK 12 protein to block the mitogen-activated protein kinaseextracellular signal regulatory kinase signaling pathway that regulates key cellular responses To create conditions for disease control radical surgical resection reducing postoperative recurrence and reducing complications Based on the targeted therapy of smeitinib bisulfate capsule this study evaluated the treatment of NF1 patients after surgical treatment and evaluated the effect of tumor shrinkage after medication by monitoring the effect and duration of solid tumor shrinkage of patients And the postoperative recurrence time so as to verify the effectiveness of smeitinib bisulfate capsule in reducing the recurrence rate of patients after NF1
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None