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 12-15 years of age or older with a diagnosis of NF1 and a PN that is progressive or causes significant morbidity.
PROTOCOL
16+ years of age
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 response rate is ≥ 25%. With one sided type I error of 0.05 a sample size of 16 evaluable participants 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 evaluable participants.
12-15 yrs of age
10 participants will be enrolled on this exploratory study to investigate the safety and efficacy of HLX-1502 in a small cohort of 12 to 15- year-olds. Of the 10 participants, a minimum of 5 will be required to have progressive disease, as defined in the eligibility criteria, at study entry. Enrollment of patients with non-progressive disease will stop once 5 of them have been enrolled, to ensure that a minimum of 5 patients with progressive disease are enrolled. This study will primarily include descriptive statistics rather than hypothesis testing. Descriptive analyses will be performed for the pediatric cohort overall, and then separately for patients with progressive disease and those without.
PROTOCOL CHAIRS
- Miriam Bornhorst, MD, Chair
- Michael Fisher, MD, Co-Chair
- Kathryn Nevel, MD, Co-Chair
CURRENT STATUS
The adult cohort (≥16 years of age or older) is closed to enrollment. The pediatric cohort is still open to enrollment and is for those 12-15 years of age.