@article{ref1,
title="A phase 1/2 trial to assess safety and efficacy of a vaporized 5-methoxy-N,N-dimethyltryptamine formulation (GH001) in patients with treatment-resistant depression",
journal="Frontiers in psychiatry",
year="2023",
author="Reckweg, J.T. and van Leeuwen, C.J. and Henquet, C. and van Amelsvoort, T. and Theunissen, E.L. and Mason, N.L. and Paci, R. and Terwey, T.H. and Ramaekers, J.G.",
volume="14",
number="",
pages="-",
abstract="BACKGROUND: Treatment-resistant depression (TRD) is a substantial public health burden, but current treatments have limited effectiveness. The aim was to investigate the safety and potential antidepressant effects of the serotonergic psychedelic drug 5-MeO-DMT in a vaporized formulation (GH001) in adult patients with TRD. <br><br>METHODS: The Phase 1 part (n = 8) of the trial investigated two single dose levels of GH001 (12 mg, 18 mg) with a primary endpoint of safety, and the Phase 2 part (n = 8) investigated an individualized dosing regimen (IDR) with up to three increasing doses of GH001 (6 mg, 12 mg, and 18 mg) within a single day, with a primary endpoint of efficacy, as assessed by the proportion of patients in remission (MADRS ≤ 10) on day 7. <br><br>RESULTS: Administration of GH001 via inhalation was well tolerated. The proportion of patients in remission (MADRS ≤ 10) at day 7 was 2/4 (50%) and 1/4 (25%) in the 12 mg and 18 mg groups of Phase 1, respectively, and 7/8 (87.5%) in the IDR group of Phase 2, meeting its primary endpoint (p < 0.0001). All remissions were observed from day 1, with 6/10 remissions observed from 2 h. The mean MADRS change from baseline to day 7 was −21.0 (−65%) and − 12.5 (−40%) for the 12 and 18 mg groups, respectively, and − 24.4 (−76%) for the IDR. <br><br>CONCLUSION: Administration of GH001 to a cohort of 16 patients with TRD was well tolerated and provided potent and ultra-rapid antidepressant effects. Individualized dosing with up to three doses of GH001 on a single day was superior to single dose administration. Clinical Trial registration: Clinicaltrials.gov Identifier NCT04698603. Copyright © 2023 Reckweg, van Leeuwen, Henquet, van Amelsvoort, Theunissen, Mason, Paci, Terwey and Ramaekers.<p /> <p>Language: en</p>",
language="en",
issn="1664-0640",
doi="10.3389/fpsyt.2023.1133414",
url="http://dx.doi.org/10.3389/fpsyt.2023.1133414"
}