The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate Capsule

Sponsor
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Study ID
NCT06621082
Phase
PHASE2
Status
Not Yet Recruiting

Notify me when recruiting opens

Save your spot on the interest list for this study. We'll keep your details with this study so our team can follow up when recruiting opens.

Not yet recruiting

Add your contact details and location so we can keep your interest tied to this study.

Conditions

  • Neurofibromatosis 1

Eligibility Criteria

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

Interventions

  • Selumetinib — DRUG
    After surgical resection，based on the patient's body surface area (BSA), the patient was given an oral dose of Simetinib bisulfate capsule (20-50mg bid) daily for 30 days for 6 cycles

Study Details

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) 1/2 protein to block the mitogen-activated protein kinase/extracellular 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.

Key Dates

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

Study Design

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

Arms

  • Experimental: Postoperative drug group
    This study started from the recruitment of subjects, confirmed diagnosis by clinical diagnosis and pathological biopsy, preliminary screening according to inclusion and exclusion criteria, and signing of informed consent. Patients who were evaluated as indications for surgical resection and underwent surgical treatment were treated with Smetinib bisulfate capsules. PFS, ORR, and DOR were evaluated after 6 cycles of administration of Simetinib bisulfate capsules (20-50mg bid) daily for 30 days, as individualized by patient body surface area (BSA).

Primary Outcome Measure

2-year progression-free survival (2-year PFS rate) [ Time Frame: Within 2 years from the start of treatment in the trial ]

Central Contacts

Related Studies