Tiratricol Clinical Trials

Size	Price	Qty
50mg	USD 147
1g	USD 670

Clinical Trials

A Phase 3 clinical trial is actively recruiting male patients affected by Monocarboxylate Transporter 8 deficiency, also known as Allan-Herndon-Dudley Syndrome, to evaluate tiratricol in rare genetic metabolic disorders. Sponsored by Rare Thyroid Therapeutics International AB in collaboration with Premier Research Group plc and Egetis Therapeutics, this study assesses the physiological outcomes of tiratricol withdrawal compared to placebo. Ultimately, the trial aims to establish the compound's safety and efficacy in managing thyroid hormone signaling impairment resulting from this transporter deficiency.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT05579327	Recruiting	Monocarboxylate Transporter 8 Deficiency\|Allan-Herndon-Dudley Syndrome	Rare Thyroid Therapeutics International AB\|Premier Research Group plc\|Egetis Therapeutics	2023-07-21	Phase 3

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

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Tiratricol functions as a thyroid hormone analogue that directly binds to thyroid hormone receptors, driving downstream gene transcription independently of Monocarboxylate Transporter 8 cell entry. This restores tissue-level thyroid hormone action and normalizes cellular metabolic activity, offering a targeted therapeutic mechanism for treating Monocarboxylate Transporter 8 deficiency and Allan-Herndon-Dudley Syndrome.

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>).