Trial rationale
DISEASE
The primary objective of the IB1001-301 clinical trial is to demonstrate the symptomatic benefits of IB1001 treatment given:There are no treatments for the symptoms of NPC, which are highly debilitating and significantly detrimental to functioning, quality of life, and health span.NPC is a life-limiting condition with an extremely high unmet medical need, mandating greater urgency for trials to be conducted as efficiently as possible to maximize the chance they can be made available before the window of therapeutic opportunity is lost [Accordingly, although there is data supporting both the symptomatic and neuroprotective, disease-modifying effects of IB1001 for NPC, the IB1001-301 pivotal trial prioritizes the investigation of the symptomatic benefit to expedite the development and availability of this promising drug candidate. Given the extreme heterogeneity of the disease, a crossover study design is utilized so that each patient serves as their own control and the clinical meaningfulness of the effect can be assessed for each patient.