A Long-term Extension Study of ORX750 in Participants With Narcolepsy and Idiopathic Hypersomnia

Not Applicable

Recruiting

• Conditions: Narcolepsy Type 1; Narcolepsy Type 2; Idiopathic Hypersomnia
• Interventions: Drug: ORX750

• Registration Number: NCT07096674
• Lead Sponsor: Centessa Pharmaceuticals (UK) Limited

Brief Summary

This study is a long-term extension (LTE) of the parent Study ORX750 0201, and will provide long-term open-label safety, tolerability, and efficacy of ORX750 in participants with narcolepsy type 1 (NT1), narcolepsy type 2 (NT2), and idiopathic hypersomnia (IH).

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-07
• Study First Submitted: 2025-07-02
• Study First Submitted QC: 2025-07-24
• Study First Posted: 2025-07-31
• Study Start: 2025-08-12
• Last Update Submitted: 2025-08-13
• Last Update Posted: 2025-08-15
• Primary Completion: 2026-04-30
• Study Completion: 2026-04-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

• Diagnosis of narcolepsy (NT1 or NT2) or IH who was eligible for and completed the full treatment period in the eligible parent study ORX750-0201 (CRYSTAL-1)
• Is willing and able to adhere to additional protocol requirements

Exclusion Criteria

• Development of any new disease/disorder that in the opinion of the investigator would make the participant unable to continue the study
• Unable to refrain from excluded medications, including those used for treatment of narcolepsy or idiopathic hypersomnia

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Narcolepsy Type 1	ORX750	Narcolepsy Type 1 (Open Label)
Narcolepsy Type 2	ORX750	Narcolepsy Type 2 (Open Label)
Idiopathic Hypersomnia	ORX750	Idiopathic Hypersomnia (Open Label)

Primary Outcome Measures

Name	Time	Method
Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs	Up to day 70	Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
Abnormal Laboratory Tests	Up to day 70	Number of Participants With Abnormal Changes From Baseline in Laboratory Tests
Abnormal Vital Signs	Up to day 70	Number of Participants With Abnormal Change From Baseline in Vital Signs
Abnormal ECG	Up to day 70	Number of Participants With Abnormal Change From Baseline in Electrocardiograms (ECG)
Suicidal Ideation or Behavior	Up to day 70	Number of Participants With Suicidal Ideation or Suicidal Behavior as Measured Using Columbia-Suicide Severity Rating Scale (C-SSRS)

Secondary Outcome Measures

Name	Time	Method
Cmax	Up to day 63	Cmax: Maximum Observed Plasma Concentration for ORX750
Tmax	Up to day 63	Tmax: Time of Maximum Concentration for ORX750
AUClast	Up to day 63	AUClast: Area Under the Plasma Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration for ORX750
Cmax,ss	Up to day 63	Cmax,ss: Maximum Observed Concentration at Steady State for ORX750
AUCτ	Up to day 63	AUCτ: Area Under the Concentration-time Curve During a Dosing Interval at Steady State for ORX750
Maintenance of Wakefulness Test	Up to day 63	Change From Baseline in Mean sleep latency (average of the first 4 trials) in the Maintenance of Wakefulness Test (MWT)
Epworth Sleepiness Scale	Up to day 63	Change From Baseline in Epworth Sleepiness Scale (ESS)

Trial Locations

Locations (1)

Site Number 1; 🇺🇸 Huntersville, North Carolina, United States