Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Mebufotenin, 5-MeO-DMT
GH001 · 6 trials · 6 indications
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.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH001 to determine mebufotenin serum concentrations.
Incidence of adverse events reported in the study and coded by MedDRA.
Adverse events reported in the study and coded by MedDRA.
Local infusion site findings will be assessed as none, mild, moderate and severe for the following signs and symptoms of the applicable site: dryness, redness, swelling, pain, tenderness, and itching and other.
Clinically significant changes in ECG include any significant change in rate or rhythm as determined by the principal investigator
The Modified Observer's Assessment of Alertness and Sedation scale (MOAA/S) will be completed before and after GH002 dosing. Scored from 0 (deep sedation) to 5 (alert)
The CADSS comprises 19 subjective items, ranging from 0 'not at all' to 4 'extremely. Summed together, these subscales form a total dissociative score. Combined score ranges from 0 to 76
Assessment of Discharge Readiness on the administration day by the Principal Investigator, using the Clinical Assessment of Discharge Readiness (CADR).
A detailed questionnaire assessing both suicidal behaviour and suicidal ideation.
A scale to measure psychiatric symptoms. Each symptom is rated 1-7 and a total of 18 symptoms are scored. Combined score ranges from 18 to 126.
For PK analyses, blood samples will be collected before and up to 6 hours after the administration of GH002 to determine 5-MeO-DMT serum concentrations.
For PK analyses, blood samples will be collected before and up to 4 hours after the administration of GH001 to determine 5-MeO-DMT and bufotenine serum concentrations.
Phase 1: The primary endpoint is a binary variable (yes/no) reflecting a combined medical/clinical evaluation of the occurrence of Outcomes 5 to 13. The endpoint will be considered met for any dose level or regimen if the Study Safety Group (SSG) - through a qualitative medical/clinical evaluation - considers that dose level or regimen sufficiently safe and tolerable for potential further clinical development in a subsequent study.
Phase 2: The assessment is done with the Montgomery-Asberg Depression Rating Scale (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.
The safety and tolerability of GH001 is judged by the Study Safety Group based on a combined analysis of reported adverse events, clinical observation, and safety laboratory analyses.
Visual Analogue Scale scored from 0-100
| Arm | Type | Description |
|---|---|---|
| GH001 - Part 1 | EXPERIMENTAL | GH001 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 1 | PLACEBO_COMPARATOR | Placebo 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 2 | OTHER | Patients can receive up to five GH001 IDRs as needed during the OLE based on the patient's clinical response. |
| GH001 | EXPERIMENTAL | A single inhaled dose of GH001 administered via a proprietary aerosol delivery device in 12 subjects |
| Cohort A: Dose A single dose | EXPERIMENTAL | A single dose of GH002 or placebo administered by i.v. bolus injection (randomized as 6 active and 2 placebo subjects) |
| Cohort B: Dose B single dose | EXPERIMENTAL | A single dose of GH002 or placebo administered by i.v. bolus injection (randomized as 6 active and 2 placebo subjects) |
| Cohort C: Dose C single dose | EXPERIMENTAL | A single dose of GH002 or placebo administered by i.v. bolus injection (randomized as 6 active and 2 placebo subjects) |
| Cohort D: Dose D single dose | EXPERIMENTAL | A single dose of GH002 or placebo administered by i.v. bolus injection (randomized as 6 active and 2 placebo subjects) |
| Cohort E: Dose E single dose | EXPERIMENTAL | A single dose of GH002 or placebo administered by i.v. bolus injection (randomized as 6 active and 2 placebo subjects) |
| Cohort F: Dose F single dose | EXPERIMENTAL | A single dose of GH002 or placebo administered by i.v. bolus injection (randomized as 6 active and 2 placebo subjects) |
| Cohort G: Dose G single dose | EXPERIMENTAL | A single dose of GH002 or placebo administered by i.v. bolus injection (randomized as 6 active and 2 placebo subjects) |
| Cohort J: Individualized Dosing Regimen | EXPERIMENTAL | Administration of up to 3 doses of GH002 within a single day (doses to be confirmed following review of data from single-dose part) |
| Group A - 6 mg single-dose | EXPERIMENTAL | A single, inhaled dose of GH001 6 mg or placebo (randomized as 8 active and 2 placebo subjects) |
| Group B - 12 mg single-dose | EXPERIMENTAL | A single, inhaled dose of GH001 12 mg or placebo (randomized as 8 active and 2 placebo subjects) |
| Group C - 18 mg single-dose | EXPERIMENTAL | A single, inhaled dose of GH001 18 mg or placebo (randomized as 8 active and 2 placebo subjects) |
| Group D - Individualized Dosing Regimen, 1-hour interval | EXPERIMENTAL | Administration of up to 3 inhaled doses of GH001 within a single day (6 mg, followed by 12 mg, followed by 18 mg) with a 1-hour dose interval (8 subjects) |
| Group E - Individualized Dosing Regimen, 2-hour interval | EXPERIMENTAL | Administration of up to 3 inhaled doses of GH001 within a single day (6 mg, followed by 12 mg, followed by 18 mg) with a 2-hour dose interval (8 subjects) |
| Phase 1 (Part A): GH001 dose A | EXPERIMENTAL | - |
| Phase 1 (Part A): GH001 dose B | EXPERIMENTAL | - |
| Phase 2 (Part B): GH001 Individualized Dosing Regimen | EXPERIMENTAL | - |
| GH001 dose A | EXPERIMENTAL | - |
| GH001 dose B | EXPERIMENTAL | - |
| GH001 dose C | EXPERIMENTAL | - |
| GH001 dose D | EXPERIMENTAL | - |
| GH001 Individualized Dosing | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| GH001 | DRUG | GH001 administered via inhalation |
| Placebo | DRUG | Placebo administered via inhalation |
| 5 Methoxy N,N Dimethyltryptamine | DRUG | GH001 administered via inhalation |
| GH001 Aerosol Delivery System | DEVICE | GH001 aerosol delivery system |
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 ...
GH001 is an investigational small molecule developed by GH Research PLC (ticker GHRS). It is being studied in healthy adult volunteers and in patients with treatment-resistant depression. The program includes completed Phase 1 pharmacokinetic and safety trials in healthy volunteers and a completed Phase 2 trial in treatment-resistant depression.
GH001 is being developed as a potential treatment for treatment-resistant depression, a condition in which patients do not respond adequately to standard antidepressant therapies. A completed Phase 2 trial, NCT05800860, evaluated GH001 in 81 patients with treatment-resistant depression across Czechia, Germany, Ireland, the Netherlands, Poland, and Spain.
GH001 is developed by GH Research PLC, which trades under the ticker GHRS. The company sponsors the clinical program for GH001, including the Phase 1 pharmacokinetic and safety studies in healthy volunteers and the Phase 2 trial in treatment-resistant depression.
GH001 is in clinical development. Its Phase 1 trials in healthy volunteers have been completed, and a Phase 2 trial in treatment-resistant depression has also been completed. GH001 is investigational and has not been approved by the FDA for any indication.
GH001 has been studied in completed trials including NCT07540494, which assessed pharmacokinetics and safety of GH001 delivered via a GH001 aerosol delivery system in healthy subjects; NCT05163691, a pharmacokinetics trial in healthy volunteers; and NCT05800860, a Phase 2 trial in patients with treatment-resistant depression.
Yes, GH001 is also known as 5 Methoxy N,N Dimethyltryptamine. That alternative name refers to the same investigational small molecule being developed by GH Research PLC under the designation GH001 for study in healthy volunteers and treatment-resistant depression.