Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule

Sponsor
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Study ID
NCT06620354
Phase
PHASE2
Status
Not Yet Recruiting

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Conditions

  • Neurofibromatosis 1

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Selumetinib — DRUG
    Patients without indications for surgical excision were evaluated with 6 cycles of daily oral smetinib capsules (20-50mg bid) for 30 days, individually calculated based on patient body surface area (BSA)

Study Details

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) 1/2 protein, blocking the mitogen-activated protein kinase/extracellular 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.

Key Dates

Start date
Sep 30, 2024
Status verified
Sep 2024
Primary completion
Nov 30, 2026
Completion
Dec 31, 2026

Study Design

Enrollment
33 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Drug group
    Patients assessed as having no indication for surgical resection were treated with smetinib bisulfate capsules. Based on individual patient body surface area (BSA), Smetinib bisulfate capsules (20-50mg bid) were taken orally daily for 30 days for 6 cycles to evaluate tumor reduction efficacy, ORR, and resection range.

Primary Outcome Measure

Objective response rate (ORR) [ Time Frame: Within two years of medication ]

Central Contacts

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