Neurofibromatosis type 1
Conditions
Interventions
Luvometinib treatment group:NA
Sponsors
Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine
Eligibility
Sex/Gender
All
Age
2 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1. Pediatric and adolescent patients aged 2–18 years (>= 2 years old and <= 18 years old); 2. Patients diagnosed with neurofibromatosis type 1 (NF1) in accordance with the diagnostic criteria of the 2021 International Neurofibromatosis Diagnostic Criteria Consensus Group; 3. Patients with symptomatic and inoperable plexiform neurofibromas (PN).
Exclusion criteria
Exclusion criteria: 1. Complicated with malignant peripheral nerve sheath tumors (MPNST) or other malignant tumors requiring chemotherapy or radiotherapy; 2. Complicated with other life-threatening diseases, medical conditions, or organ dysfunction; 3. Participating in other clinical trials at the time of enrollment or initiation of Luwotemetinib treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Objective response rate;Disease control rate;Clinical benefit rate;The median tumor volume change of the best response;Time to response;Progression free survival;Time to progression;Duration of response;Change of tumor volume; | — |
Secondary
| Measure | Time frame |
|---|---|
| The types and frequencies of adverse events;Severe adverse enents and toxic reaction caused discontinuation;Visual improvement(Via standard visual assessment);Improvement of quality of life(Via quality of life questionnaire);Long term efficacy and safety (By long term follow-up and record of adverse events); | — |
Countries
China
Contacts
Public ContactHu Xiaojie
Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed