Clinical Trials

Multiple Phase 2 and Phase 2/3 clinical trials sponsored by academic institutions and industry partners, such as AMO Pharma Limited and Noscira SA, evaluate the therapeutic potential of Tideglusib. These studies encompass completed evaluations in Alzheimer's disease and autism spectrum disorders, as well as actively recruiting or upcoming trials targeting progressive supranuclear palsy, congenital myotonic dystrophy, amyotrophic lateral sclerosis, and arrhythmogenic cardiomyopathy.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06174220 RECRUITING
Arrhythmogenic Cardiomyopathy; Arrhythmogenic Right Ventricular Cardiomyopathy
Hamilton Health Sciences Corporation
 2025-03-21 PHASE2
NCT05004129 RECRUITING
Congenital Myotonic Dystrophy
AMO Pharma Limited
 2021-08-23 PHASE2; PHASE3
NCT05105958 NOT_YET_RECRUITING
Amyotrophic Lateral Sclerosis
University of Zurich
 2025-12-01 PHASE2
NCT03692312 COMPLETED
Congenital Myotonic Dystrophy
AMO Pharma Limited
 2021-03-03 PHASE2; PHASE3
NCT02586935 COMPLETED
Autism Spectrum Disorders
Holland Bloorview Kids Rehabilitation Hospital
 2016-02-10 PHASE2
NCT02858908 COMPLETED
Myotonic Dystrophy 1
AMO Pharma Limited
 2016-07-20 PHASE2
NCT02586935 Completed
Autism Spectrum Disorders
Evdokia Anagnostou|Holland Bloorview Kids Rehabilitation Hospital|McMaster University|University of Western Ontario Canada|Unity Health Toronto|University of Toronto|Anagnostou Evdokia M.D.
 2016-02-10 Phase 2
NCT01350362 COMPLETED
Alzheimer's Disease
Noscira SA
 2011-04 PHASE2
NCT01049399 COMPLETED
Progressive Supranuclear Palsy
Noscira SA
 2009-12

(data from https://clinicaltrials.gov, updated on 2026-03-12)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tideglusib acts as an irreversible, non-ATP-competitive inhibitor of glycogen synthase kinase-3β (GSK-3β), binding to the catalytic domain to block target protein phosphorylation and restore essential intracellular signaling cascades. By suppressing pathological GSK-3β hyperactivation, the compound promotes cell survival and regulates downstream cellular differentiation pathways, providing the mechanistic foundation for its clinical evaluation in neurodegenerative and muscular disorders such as Alzheimer's disease, amyotrophic lateral sclerosis, and myotonic dystrophy.

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>).

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