A Phase 2, Open-Label, Single Arm, Non-Controlled, Single-Stage Study to Evaluate the Safety and Effects of HLX-1502 in Patients with Plexiform Neurofibroma and Neurofibromatosis Type 1

GOALS

 To evaluate the effect of HLX-1502 on PN response rate by 12 cycles, assessed by volumetric MRI imaging

DRUGS

HLX-1502

ELIGIBILITY

Participants 16 years of age or older with a diagnosis of NF1 and a PN that is progressive or causes significant morbidity.

PROTOCOL

In this single-stage clinical trial, the primary endpoint is PN response by 12 cycles. The null hypothesis being tested is that response rate is ≤ 5% vs. the alternative hypothesis that the re-sponse rate is ≥ 25%. With one sided type I error of 0.05 a sample size of 16 evaluable partici-pants will provide 80% power to test this hypothesis. If the number of responders is 3 or more, the null hypothesis is rejected suggesting efficacy of the treatment. If the number of responders is 2 or fewer, the null hypothesis will not be rejected. Anticipating a maximum non-evaluable percentage of 20%, 20 participants will be enrolled into the study to achieve at least 16 evalua-ble participants.

PROTOCOL CHAIRS

• Miriam Bornhorst, MD, Chair
• Michael Fisher, MD, Co-Chair
• Kathryn Nevel, MD, Co-Chair

CURRENT STATUS

Currently Enrolling