Ignite Creation Date:
2024-10-26 @ 3:41 PM
Last Modification Date:
2024-10-26 @ 3:41 PM
Study NCT ID:
NCT06620354
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-20
Brief Title:
Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Prospective One-arm Phase II Clinical Study of Smeitinib Hydrosulfate Capsules for the Treatment of Patients With Type I Neurofibromatosis
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 who currently lack effective drug therapy and have a high recurrence rate after surgical resection As a MEK inhibitor Smetinib bisulfate capsule can induce tumor shrinkage by selectively binding mitogen-activated protein kinase MEK 12 protein blocking the mitogen-activated protein kinaseextracellular signal regulatory kinase signaling pathway that regulates key cell responses To create conditions for disease control radical surgical resection reducing postoperative recurrence and reducing complications The purpose of this study was to provide treatment with Smetinib bisulfate for patients with type I neurofibromatosis observe the therapeutic effect in stages convert patients without surgical indications into patients with surgical indications increase the proportion of surgical resection and reduce the recurrence rate Objective tumor response rate ORR after drug treatment was used as the main outcome index in this study The resectable scope duration of remission DOR progression-free survival PFS were used as secondary outcome indicators to investigate the improvement of resectable rate reduction of resectable scope and postoperative complications tumor shrinkage effect and the stability of curative effect of the use of smetinib bisulfate capsule on type I neurofibromatosis
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 deficiency can lead to overactivation of RAS pathway resulting in uncontrolled cell proliferation in patients with NF1 5 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
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 Smetinib bisulfate capsule this study administered medication to enrolled patients By monitoring the tumor shrinkage effect of patients with solid tumors and evaluating postoperative surgical indications for patients without surgical indications before medication the effectiveness of smetinib bisulfate capsule in the treatment of NF1 was verified
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 deficiency can lead to overactivation of RAS pathway resulting in uncontrolled cell proliferation in patients with NF1 5 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
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 Smetinib bisulfate capsule this study administered medication to enrolled patients By monitoring the tumor shrinkage effect of patients with solid tumors and evaluating postoperative surgical indications for patients without surgical indications before medication the effectiveness of smetinib bisulfate capsule in the treatment of NF1 was verified
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