Phase II Study of HL-085 to Treat Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

What this Trial Is About

Researchers are evaluating HL-085 in adults with Neurofibromatosis Type 1 NF1 who have inoperable and symptomatic plexiform neurofibromas PN. This multi-center, open-label, single-arm Phase II study aims to assess the safety, efficacy, and pharmacokinetics of HL-085. The study includes two parts phase IIa to determine a recommended dose and evaluate preliminary safety and efficacy, and phase IIb to further assess safety and efficacy in a larger group of patients. During phase IIa, approximately 15 patients will receive HL-085 orally at a dose of 9 mg twice daily in continuous 21-day cycles. Dosage adjustments to 12 mg or 6 mg twice daily may be made if needed. Phase IIb will enroll around 35 patients to receive HL-085 at the recommended dose determined from phase IIa. Treatment continues in cycles of 21 days. Participants will undergo regular assessments including MRI scans to measure tumor response at the end of cycles 4, 8, 12, 16, 20, 24, 28, and 32, then every 8 cycles thereafter. Researchers will monitor objective response rate, disease control rate, duration of response, progression-free survival up to 3 years, and pharmacokinetics during treatment. Patients must be able to complete follow-up exams and provide informed consent, with overall participation lasting through multiple treatment cycles and evaluations.

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