Clinical Trials

Multiple clinical trials evaluate interventions for pediatric conditions, including liver diseases and Duchenne muscular dystrophy. A Phase II trial sponsored by Summit Therapeutics evaluated pediatric subjects with Duchenne muscular dystrophy but has been terminated. Additionally, an unphased trial sponsored by the Institute of Liver and Biliary Sciences India is actively recruiting pediatric participants for liver disease interventions, reflecting research supported by both academic and industry sponsors.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05770284 Recruiting
Liver Diseases
Institute of Liver and Biliary Sciences India
 2023-03-15 Not Applicable
NCT02858362 Terminated
Duchenne Muscular Dystrophy
Summit Therapeutics
 2016-06 Phase 2

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

Check the (S)-Methylisothiourea sulfate product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

(S)-Methylisothiourea sulfate selectively inhibits inducible nitric oxide synthase, thereby suppressing the overproduction of nitric oxide and downstream reactive nitrogen species in damaged tissues. By reducing nitric oxide-mediated inflammatory signaling and oxidative cellular injury, this inhibition provides a rational approach to counter tissue degeneration in inflammatory liver diseases and neuromuscular disorders like Duchenne muscular 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.