Neurofibromatosis type 1 (NF1)
Conditions
Interventions
Sponsors
Jinan Children's Hospital
Eligibility
Sex/Gender
All
Age
2 Years to 16 Years
Inclusion criteria
Inclusion criteria: 1.Pediatric and adolescent patients aged 2–16 years; 2.Patients with neurofibromatosis type 1 (NF1) diagnosed in accordance with the revised diagnostic criteria of the 2021 International Consensus Group for Neurofibromatosis Type 1 Diagnostic Criteria; 3.Patients with inoperable plexiform neurofibromas (PN) complicated by intracranial unidentified bright objects (UBOs); 4.The patient is able to provide written informed consent. Data collection may only be initiated after written informed consent has been obtained. The patient, or the patient's parents/legal guardians, must voluntarily and be capable of providing written informed consent, and parental or legal guardian consent with appropriate documentation must be obtained during the informed consent process.
Exclusion criteria
Exclusion criteria: 1.Presence of malignant peripheral nerve sheath tumor (MPNST) or other malignant tumors requiring chemotherapy or radiotherapy; 2.Presence of other life-threatening diseases, medical conditions, or organ dysfunction; 3.Currently participating in any other clinical trial at the time of enrollment or initiation of luvometinib treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| DQ (2–6 yrs) / IQ (6–16 yrs); | — |
Secondary
| Measure | Time frame |
|---|---|
| Pain improvement;Change rate of UBOs;change rate of PN volume;Pediatric behavioral problems;Quality of Life (QoL); | — |
Countries
China
Contacts
Public ContactGuangyu Wang
Jinan Children's Hospital
Outcome results
None listed