Deflazacort Clinical Trials

Size	Price	Qty
50mg	USD 97

Clinical Trials

Several Phase 1 and Phase 3 clinical trials sponsored by entities including PTC Therapeutics and Parc de Salut Mar have evaluated deflazacort in healthy volunteers, patients with renal impairment, and individuals with Duchenne or limb-girdle muscular dystrophy. Completed early-phase studies successfully assessed the safety, excretion, and pharmacokinetic parameters of deflazacort and its metabolites. Conversely, certain advanced evaluations were ultimately terminated or withdrawn, reflecting a varied landscape of clinical research outcomes for the compound.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT04791345	Unknown status	Healthy Volunteers	Parc de Salut Mar	2021-02-26	Phase 1
NCT03783923	Terminated	Limb-Girdle Muscular Dystrophy	PTC Therapeutics	2019-10-31	Phase 3
NCT03642145	Withdrawn	Duchenne Muscular Dystrophy	PTC Therapeutics	2018-10-31	Phase 3
NCT02251600	Completed	Duchenne Muscular Dystrophy	PTC Therapeutics	2014-12	Phase 1
NCT02286622	Completed	Renal Impairment	PTC Therapeutics	2014-12	Phase 1

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

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Deflazacort selectively binds to cytoplasmic glucocorticoid receptors, inducing receptor translocation to the nucleus where it modulates transcription factors to suppress pro-inflammatory cytokine expression and immune activation. This downstream cellular inhibition limits tissue inflammation and muscle fiber damage, providing therapeutic benefit in inflammatory disorders and neuromuscular conditions such as 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>).