Phase 2 Open-Label Study Evaluating Safety and Effects of Oral HLX-1502 in Patients with Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas

What this Trial Is About

This trial investigates the safety and effects of HLX-1502 in people aged 12 years and older with Neurofibromatosis Type 1 NF1 who have progressive or symptomatic plexiform neurofibromas PN. The study is open-label and phase 2, aiming to assess how well participants tolerate the treatment and how effective it is, especially in those aged 16 and above, with a smaller group of 12 to 15 year olds also included. Participants will take HLX-1502 orally three times daily for 12 cycles, each cycle lasting 28 days. Those who show partial response, stable disease after previous progression, or clinical improvement may continue treatment for an additional 12 cycles, making a total of 24 cycles. The treatment is given continuously during these cycles. Throughout the study, participants will be monitored for tumor response using volumetric MRI, with assessments of safety and tolerability conducted regularly. Researchers will measure primary outcomes at 12 months and some at 24 months, including tumor response rate, duration and time to response, and pharmacokinetics of the drug. The total participation can last up to 24 cycles, with ongoing evaluations to understand the effects and safety of HLX-1502.

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1