HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
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
Shanghai Kechow Pharma, Inc.
70 participants
Oct 18, 2021
INTERVENTIONAL
Conditions
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
Inclusion Criteria12
- 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 Criteria12
- 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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Interventions
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.
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT05331105