ORX750 for Narcolepsy and Idiopathic Hypersomnia

(CRYSTAL-1 Trial)

This trial tests a new treatment called ORX750, designed to reduce daytime sleepiness in people with narcolepsy and idiopathic hypersomnia (IH). The trial evaluates the safety of ORX750 and its effects on these conditions, which cause excessive daytime sleepiness (EDS) and, in some cases, sudden muscle weakness (cataplexy). Participants will receive either ORX750 or a placebo (a pill with no active medication) for comparison. Individuals diagnosed with Narcolepsy Type 1, Narcolepsy Type 2, or IH who are willing to stop their current medications might be suitable for this study. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to significant advancements in treatment.

Yes, you will need to stop taking all medications used for treating narcolepsy or idiopathic hypersomnia to participate in this trial.

Research shows that ORX750 is a new treatment being tested for people with narcolepsy and idiopathic hypersomnia. Early studies have examined the safety of ORX750 in humans. Initial results suggest that most participants did not experience serious side effects, indicating the treatment is generally well-tolerated.

ORX750 is designed to mimic orexin, a brain protein that helps maintain wakefulness during the day. Although more research is needed, the current phase of testing suggests that ORX750 is promising in terms of safety. This stage of research typically checks for serious side effects and helps determine how the treatment affects the body.

Most treatments for narcolepsy and idiopathic hypersomnia typically focus on stimulating the central nervous system to reduce excessive daytime sleepiness. However, ORX750 works differently by targeting the orexin system, which plays a crucial role in regulating wakefulness and sleep. This new mechanism of action is what excites researchers, as it has the potential to address the root cause of these sleep disorders more directly than current stimulant-based therapies. Additionally, ORX750 may offer a more targeted approach with possibly fewer side effects compared to traditional treatments like modafinil or amphetamines.

Research has shown that ORX750, a new treatment under study in this trial, appears promising for individuals with narcolepsy and idiopathic hypersomnia. In earlier studies, ORX750 helped participants stay awake longer during the day. Specifically, it increased the time it took for participants to fall asleep by more than 10 minutes compared to a placebo. Additionally, ORX750 was well tolerated, with most participants experiencing no major side effects. This trial will evaluate ORX750 in separate arms for narcolepsy type 1, narcolepsy type 2, and idiopathic hypersomnia. These early results suggest that ORX750 could help reduce excessive daytime sleepiness in these conditions.¹³⁴⁶⁷