Treatment of Neurofibromatosis Type 1 with HL-085

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

This study is testing a new treatment called HL-085 to see if it can safely help adults with Neurofibromatosis Type 1 and inoperable tumors.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 70 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Shanghai Kechow Pharma, Inc. (industry)
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT05331105 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of HL-085 in adults with Neurofibromatosis Type 1 (NF1) and inoperable plexiform neurofibromas. It consists of two phases: Phase IIa focuses on determining the recommended dose and assessing preliminary safety and efficacy, while Phase IIb aims to further evaluate these aspects in a larger group of patients. Participants will receive HL-085 at varying doses on a continuous schedule, with safety and efficacy monitored throughout the study.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with inoperable and symptomatic plexiform neurofibromas and a confirmed diagnosis of Neurofibromatosis Type 1.

Not a fit: Patients with operable plexiform neurofibromas or those who do not meet the diagnostic criteria for NF1 may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from inoperable plexiform neurofibromas associated with NF1.

How similar studies have performed: While there have been studies on treatments for NF1, the specific approach using HL-085 is novel and has not been extensively tested in this context.

Eligibility 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.

Where this trial is running

Shanghai, Shanghai Municipality

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

Study contacts

• Study coordinator: Zhimei Zhu, Master

Conditions: Neurofibromatosis 1, Plexiform Neurofibromas