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

Recruiting now · Phase 2

Conditions studied: Neurofibromatosis 1, Plexiform Neurofibromas

In brief

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)

Key facts

Study ID

NCT05331105

Run by

Shanghai Kechow Pharma, Inc.

People needed

70

Starts

2021-10-18

Expected to finish

2028-10-31

Last updated by the study team

2023-05-31

Who can join

Age: 18 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine — Shanghai, Shanghai Municipality, China (enrolling)

Full record on ClinicalTrials.gov

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