Neurofibromatosis type 1 (NF1)
Conditions
Interventions
Luvometinib:None
Sponsors
Shandong Provincial Hospital
Eligibility
Sex/Gender
All
Age
2 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1.Pediatric patients aged =2 years and <18 years, with a confirmed diagnosis of Neurofibromatosis Type 1 (NF1), and symptomatic, inoperable plexiform neurofibromas (PN); 2.Presence of at least one inoperable target PN measurable by volumetric MRI analysis; 3.Adequate organ and bone marrow function; 4.Signed written informed consent;
Exclusion criteria
Exclusion criteria: 1.Confirmed or suspected malignant glioma or malignant peripheral nerve sheath tumor (MPNST) (low-grade gliomas, including optic pathway gliomas not requiring systemic therapy or radiotherapy, are excluded); 2.Prior treatment with selumetinib or other MEK1/2 inhibitors; 3.Presence of other active malignant neoplasms; 4.Severe hepatic or renal insufficiency; 5.Inability to comply with study requirements;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Objective Response Rate; | — |
Secondary
| Measure | Time frame |
|---|---|
| Duration of Response;2-year Progression-Free Survival Rate;Disease Control Rate;1-year Progression-Free Survival Rate;Clinical Benefit Rate;Progression-Free Survival;Correlation between core protein biomarkers and clinical efficacy;Time to Response (TTR);Time to Progression (TTP); | — |
Countries
China
Contacts
Public ContactZhang Zhen
Shandong Provincial Hospital
Outcome results
None listed