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golexanolone

Phase 1

Primary Biliary Cholangitis (PBC) | Small molecule | Other |Cognition Therapeutics, Inc.|Last Updated: Mar 9, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment84

FDA Designations

No designations recorded

Clinical trial landscape

golexanolone · 1 trial · 1 indication

Phase 1 1
NCT07304843A Two-part Study to Investigate the Effects in Adults of Two Doses of Golexanolone in Patients With Primary Biliary Cholangitis (PBC) With Fatigue and Cognitive DysfunctionPrimary Biliary Cholangitis (PBC)
RECRUITING84 Analytics
PHASE1RECRUITING
A Two-part Study to Investigate the Effects in Adults of Two Doses of Golexanolone in Patients With Primary Biliary Cholangitis (PBC) With Fatigue and Cognitive Dysfunction
Primary Biliary Cholangitis (PBC)Unlock trial analytics

Study Endpoints

Primary Endpoints

1. Frequency, intensity, and seriousness of adverse events (AEs) from baseline to Day 28 (part B)
From enrollment to the end of treatment at 28 days

Frequency, intensity, and seriousness of adverse events (AEs) from baseline to Day 28

Frequency, intensity, and seriousness of adverse events (AEs) from baseline to Day 5 (part A)
From Baseline to Day 5

Frequency, intensity, and seriousness of adverse events (AEs)

Secondary Endpoints

1. Change from baseline to Day 28 in PBC-40 scores for each of the domains (cognition, itch, fatigue, social, emotional, and general symptoms) (part B)
From baseline to Day 28
2. Change from baseline to Day 28 in EQ-5D-3L tool (part B)
From baseline to Day 28
3. Change from baseline to Day 28 in daytime sleepiness related symptoms using the Epworth Sleepiness Scale (ESS) (part B)
From baseline to Day 28
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part B: Treatment arm 1 (golexanolone 40 mg)EXPERIMENTAL40 mg golexanolone BID
Part B: Treatment arm 2 (golexanolone 80 mg)EXPERIMENTAL80 mg golexanolone BID
PlaceboPLACEBO_COMPARATORPart B: Placebo BID
Part A: GolexanoloneEXPERIMENTAL40 mg golexanolone BID
Part A: PlaceboPLACEBO_COMPARATORPlacebo BID

Interventions

NameTypeDescription
golexanoloneDRUGsoft gelatin capsules, oral dosage twice per day for up to 28 days
PlaceboDRUGsoft gelatin capsules, oral dosage twice a day for up to 28 days
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites36

Inclusion Criteria: * Male and female subjects age ≥ 18 years * Diagnosis of PBC based on the presence of ≥2 of 3 key disease characteristics * Clinically significant fatigue defined for the purposes of this study as a PBC-40 fatigue domain score of ≥29 at screening * Clinically significant cogniti...

Countries:GermanyGreeceHungaryItalySerbiaSpainTurkey (Türkiye)United Kingdom
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Frequently asked questions about golexanolone

What is golexanolone used for?

Golexanolone is an investigational small molecule being developed for Primary Biliary Cholangitis (PBC), specifically for patients with fatigue and cognitive dysfunction. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who is developing golexanolone?

Golexanolone is being developed by Cognition Therapeutics, Inc., a biopharmaceutical company traded on the stock exchange under the ticker CGTX. The company is conducting clinical trials to evaluate the drug's effects in patients with Primary Biliary Cholangitis.

What phase is golexanolone in?

Golexanolone is in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved for commercial use. The ongoing Phase 1 trial is recruiting participants to study the drug's safety and effects in adults with Primary Biliary Cholangitis.

What clinical trials is golexanolone in?

Golexanolone is being studied in a Phase 1 clinical trial registered as NCT07304843. This two-part study investigates the effects of two doses of golexanolone in adults with Primary Biliary Cholangitis who experience fatigue and cognitive dysfunction. The trial is currently recruiting participants across multiple countries.

Is golexanolone being tested in a randomized controlled trial?

Yes, the Phase 1 clinical trial of golexanolone is randomized, double-blind, and placebo-controlled. The study plans to enroll 84 participants and includes biomarker selection. It is currently recruiting patients in Germany, Greece, Hungary, Italy, Serbia, Spain, Turkey, and the United Kingdom.