HL-085: IIa: HL-085 capsule 9mg administered orally twice daily in a continuous 21-day treatment cycle. If required, dosing schedule can be adjusted to 12mg BID, 6mg BID, or other dosage regimens.
IIb: HL-085 at the recommended dose or dosage regimen.
Study 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)
Eligibility
Sex
ALL
Min age
18 Years
Max age
80 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Objective Response Rate (ORR) — At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days) To assess the efficacy of HL-085 on the tumor volume (plexiform neurofibromas) using volumetric MRI per REiNS criteria. ORR is defined as the percentage of patients who have achieved a confirmed Partial Responses (PR) or Complete Responses (CR).
Trial sites (1)
Facility
City
Region
Status
Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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