HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

• Status: Recruiting
• Trial end date: 2028-10-31
• Target enrollment: 0
• Participant gender: All

Summary

This is a Multi-center, Open-label, Single-arm Phase II Study to Evaluate the Efficacy and Safety of HL-085 in the treatment of Adult Participants with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas(PN)

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Shanghai Kechow Pharma, Inc.

Criteria

Inclusion Criteria:

• Age: patients must be ≥18 years of age at the time of study entry.

• Diagnosis: Patients must have inoperable and symptomatic plexiform neurofibromas(PN), and patients must have NF1 mutation or meet at least 1 of the following NF1 diagnostic criteria:

① ≥6 cafe-au-lait macules ;

② Axillary freckling or freckling in inguinal regions;

③ ≥2 Lisch nodules (iris hamartomas);

④ A distinctive bony lesion such as dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex);

⑤ An optic pathway glioma;

⑥ First-degree relative with NF1.

• Patients must have a measurable lesion, defined as at least 3 cm in length, amenable to MRI for efficacy assessment.

• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.

• Patients are able to understand and voluntarily sign a written informed consent form.

• Patients must be willing and able to complete study procedures and follow-up examinations.

Exclusion Criteria:

• Patients who are unable to undergo MRI scans (prosthesis, prosthesis, braces, etc.) or patients with lesions that cannot be evaluated by MRI.

• Patients do not have adequate organ function.

• Patients who are unable to take drugs orally, have difficulty swallowing or anything that may lead to inadequate drug absorption.

• Prior treatment with MEK 1/2 inhibitors.

• Patients known to be allergic to the ingredients or analogues of the study drug.

• Patients with previous or current retinal diseases such as retinal vein occlusion (RVO), retinal pigment epithelium detachment (RPED), central serous retinopathy (CSR), etc. (except retinopathy caused by research diseases).

• With infections or other uncontrolled disease.

• Strong CYP2C9 inhibitors or inducers within 7 days before treatment of the study drug.

• Patients who received surgery within 4 weeks or radiotherapy within 6 weeks before enrollment.

• Patients who participated in any other clinical study treatment within 4 weeks before enrollment.

• Patients treated with anti-NF1 treatment with unresolved chronic toxicity.

• Clinical judgment by the investigator that the patient should not participate in the study.