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GH001

Phase 2

Treatment-resistant Depression | Small molecule | Psychiatry |GH Research PLC|Last Updated: May 31, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment81

FDA Designations

No designations recorded

Clinical trial landscape

GH001 · 1 trial · 1 indication

Phase 2 1
NCT05800860A Trial of GH001 in Patients With Treatment-resistant DepressionTreatment-resistant Depression
COMPLETED81 Analytics
PHASE2COMPLETED
A Trial of GH001 in Patients With Treatment-resistant Depression
Treatment-resistant DepressionUnlock trial analytics

Study Endpoints

Primary Endpoints

Mean change in MADRS from Baseline to Day 7
Baseline to Day 7

The assessment is done with the MADRS, a diagnostic questionnaire with ten items for measuring the severity of depressive episodes in patients with mood disorders. A higher MADRS score indicates more severe depression, and each item is scored from 0 to 6. The overall score ranges from 0 to 60.

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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
GH001 - Part 1EXPERIMENTALGH001 is administered via inhalation, as an IDR consisting of up to 3 increasing doses of GH001 (6 mg, 12 mg, and 18 mg), on a single day. The second and third doses are only administered if the patient did not achieve intense psychoactive effects (a peak experience \[PE\]) at the previously administered dose.
Placebo - Part 1PLACEBO_COMPARATORPlacebo is administered via inhalation, as an IDR consisting of up to 3 doses of Placebo, on a single day. The second and third doses are only administered if the patient did not achieve intense psychoactive effects (a PE) at the previously administered dose.
Open-Label Extension (OLE) - Part 2OTHERPatients can receive up to five GH001 IDRs as needed during the OLE based on the patient's clinical response.

Interventions

NameTypeDescription
GH001DRUGGH001 administered via inhalation
PlaceboDRUGPlacebo administered via inhalation
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Eligibility Criteria

Age Range18 Years to 64 Years
SexALL
Healthy VolunteersNo
Study Sites11

Main Inclusion Criteria: 1. Is in the age range between 18 and 64 years (inclusive) at the time of informed consent; 2. Meets the trial criteria for TRD as assessed by a study psychiatrist: 1. Meets the Diagnostic and Statistical Manual of Mental Disorders 5 (DSM-5) criteria for single-episode ...

Countries:CzechiaGermanyIrelandNetherlandsPolandSpain
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Frequently asked questions about GH001

What is GH001 used for?

GH001 is an investigational small molecule being developed for the treatment of treatment-resistant depression. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.

Who makes GH001?

GH001 is being developed by GH Research PLC, a biopharmaceutical company listed on the stock exchange under the ticker GHRS.

What phase is GH001 in?

GH001 is in Phase 2 clinical development for treatment-resistant depression. It has completed one Phase 2 trial and is not yet approved for commercial use.

What clinical trials is GH001 in?

GH001 has been studied in a completed Phase 2 clinical trial with the identifier NCT05800860. This trial enrolled 81 participants with treatment-resistant depression and was conducted in Czechia, Germany, Ireland, Netherlands, Poland, and Spain.

Is GH001 FDA approved?

GH001 is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for treatment-resistant depression, meaning it has not yet received regulatory approval for any use.