90 Participants Needed

ORX750 for Narcolepsy and Idiopathic Hypersomnia

OC
Overseen ByOrexin Centessa Program Lead
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Centessa Pharmaceuticals (UK) Limited
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called ORX750 for individuals with sleep disorders such as narcolepsy type 1, narcolepsy type 2, and idiopathic hypersomnia. The goal is to assess the safety and effectiveness of this medication over an extended period. It is open to those who have participated in a previous related study and have a confirmed diagnosis of one of these sleep disorders. Participants must be willing to follow the study's rules and refrain from starting new medications for their condition. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

Yes, you will need to stop taking certain medications, especially those used to treat narcolepsy or idiopathic hypersomnia, as they are excluded from the trial.

Is there any evidence suggesting that ORX750 is likely to be safe for humans?

Research has shown that ORX750 has been tested for safety in people with sleep disorders like narcolepsy and idiopathic hypersomnia. In earlier studies, participants received multiple doses of ORX750 to observe their body's response. ORX750 was generally well-tolerated, with most people not experiencing serious side effects. Some might have had mild reactions, but serious problems were rare. As a Phase 2 trial, the treatment has already demonstrated some safety in earlier studies. ORX750 targets a specific part of the brain to help manage sleep disorders, and previous research supports its safety for this purpose. However, each person's experience can differ.12345

Why do researchers think this study treatment might be promising for narcolepsy and idiopathic hypersomnia?

Researchers are excited about ORX750 because it represents a novel approach for treating narcolepsy and idiopathic hypersomnia. Unlike standard treatments like modafinil or sodium oxybate, which mainly focus on symptom management by promoting wakefulness or enhancing sleep quality, ORX750 targets the underlying mechanisms of these sleep disorders. This drug acts on the orexin system, which is crucial for regulating sleep-wake cycles. By directly influencing this system, ORX750 has the potential to address the root cause of excessive daytime sleepiness, offering a more targeted and effective treatment.

What evidence suggests that ORX750 might be an effective treatment for narcolepsy and idiopathic hypersomnia?

Research shows that ORX750, the investigational treatment in this trial, has promising results for treating narcolepsy type 1, narcolepsy type 2, and idiopathic hypersomnia. Studies found that ORX750 was well tolerated and improved wakefulness. Specifically, for narcolepsy type 2, a 2.5 mg dose proved especially effective, allowing individuals to stay awake for about 32 minutes without falling asleep. In those with idiopathic hypersomnia, there was a noticeable increase in wakefulness duration. Overall, the evidence suggests that ORX750 could be a strong option for managing these sleep disorders.13467

Are You a Good Fit for This Trial?

This trial is for people with narcolepsy type 1 (NT1), narcolepsy type 2 (NT2), or idiopathic hypersomnia (IH) who have already been part of the parent Study ORX750 0201. It's designed to continue observing the long-term effects of the drug ORX750.

Inclusion Criteria

Is willing and able to adhere to additional protocol requirements
I have narcolepsy or IH and completed the CRYSTAL-1 study.

Exclusion Criteria

I cannot stop taking certain medications, including those for narcolepsy or hypersomnia.
I have no new health issues that would stop me from continuing the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ORX750 in an open-label format to assess long-term safety, tolerability, and efficacy

10 weeks
Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive ORX750 to assess long-term outcomes

Long-term

What Are the Treatments Tested in This Trial?

Interventions

  • ORX750
Trial Overview The study tests the safety, tolerability, and effectiveness of a medication called ORX750 over an extended period in patients with different types of narcolepsy and idiopathic hypersomnia. This is an open-label extension meaning everyone gets the active drug.
How Is the Trial Designed?
3Treatment groups
Experimental Treatment
Group I: Narcolepsy Type 2Experimental Treatment1 Intervention
Group II: Narcolepsy Type 1Experimental Treatment1 Intervention
Group III: Idiopathic HypersomniaExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Centessa Pharmaceuticals (UK) Limited

Lead Sponsor

Trials
2
Recruited
100+

Citations

Phase 1 Clinical Data with Orexin Receptor 2 (OX2R) ...Conclusions: Results support that ORX750 was well tolerated across the evaluated dose range, and clinically meaningful and statistically significant ...
Release DetailsIn the 4.0 mg cohort (n=10), ORX750 achieved a >10 minute change from baseline in mean sleep latency compared with placebo on the MWT at Week 2 ...
0861 A Phase 2a, Double-blind, Placebo-controlled Study of ...This study will evaluate the safety, tolerability, efficacy, and PK of multiple doses of ORX750 for the first time in patients with NT1, NT2, ...
CRYSTAL-1 Study DesignNarcolepsy types 1 (NT1) and 2 (NT2) and idiopathic hypersomnia (IH) are rare, debilitating central disorders of hypersomnolence ...
NCT06752668 | A Study of ORX750 in Participants With ...Meets the diagnostic criteria of Narcolepsy Type 1 (NT1), Type 2 (NT2) or Idiopathic Hypersomnia (IH) according to ICSD-3-TR criteria; Is willing and able to ...
0861 A Phase 2a, Double-blind, Placebo-controlled Study of ...This study will evaluate the safety, tolerability, efficacy, and PK of multiple doses of ORX750 for the first time in patients with NT1, NT2, and IH.
Study on the Safety and Effects of ORX750 for Patients ...This study investigates the safety and effects of the drug ORX750 on patients suffering from sleep disorders, specifically narcolepsy and ...
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