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

RecruitingPhase 2Ages 18 Years - 80 YearsAllShanghai Kechow Pharma, Inc.
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In plain words

What the trial is, and whether it could suit you

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)

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๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Age: patients must be โ‰ฅ18 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: โ‘  โ‰ฅ6 cafe-au-lait macules ; โ‘ก Axillary freckling or freckling in inguinal regions; โ‘ข โ‰ฅ2 Lisch nodules (iris hamartomas); โ‘ฃ A distinctive bony lesion such as dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex); โ‘ค An optic pathway glioma; โ‘ฅ 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.
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What it tests

  • HL-085
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Where

1 site
Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine
Shanghai, China
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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