Clinical Trials

Multiple Phase I and Phase II clinical trials sponsored by Hoffmann-La Roche have evaluated the selective compound RO5186582 (Basmisanil) in healthy volunteers and patients with central nervous system disorders. Completed Phase I studies established the drug's safety, pharmacokinetics, and receptor occupancy in healthy participants. Subsequent Phase II trials investigated therapeutic efficacy, yielding completed studies in Dup15q syndrome and schizophrenia alongside terminated trials in stroke and Down syndrome.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05307679 Completed
Dup15q Syndrome
Hoffmann-La Roche
 2022-12-16 Phase 2
NCT02928393 Terminated
Stroke
Hoffmann-La Roche
 2017-02-20 Phase 2
NCT02953639 Completed
Schizophrenia
Hoffmann-La Roche
 2016-11-30 Phase 2
NCT02484703 Terminated
Down Syndrome
Hoffmann-La Roche
 2015-10-28 Phase 2
NCT02534207 Completed
Healthy Volunteer
Hoffmann-La Roche
 2015-08 Phase 1
NCT02470832 Completed
Healthy Patient Study
Hoffmann-La Roche
 2015-05 Phase 1

(data from https://clinicaltrials.gov, updated on 2024-05-22)

Check the RO5186582 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

RO5186582 acts as a highly selective negative allosteric modulator of GABAA receptors containing the α5 subunit, binding to allosteric sites to diminish GABA-induced inhibitory chloride influx and enhance hippocampal neuronal excitability. By restoring balanced neurotransmission and promoting synaptic plasticity, this selective inhibition aims to alleviate cognitive and functional deficits associated with clinical trial indications such as Down syndrome, schizophrenia, Dup15q syndrome, and stroke.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

Jump to

Note: Technical data last updated: Sep 1, 2026.