Treatment of Neurofibromatosis Type 1 with HL-085

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine
  Shanghai, Shanghai Municipality, China Recruiting

Study contacts

• Study coordinator: Zhimei Zhu, Master
• Email: zhuzm@kechowpharma.com
• Phone: 86 215201345822

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.