N-Acetylcysteine amide (NACA) Clinical Trials

Size	Price	Qty
5mg	USD 147
25mg	USD 370

Clinical Trials

Multiple clinical trials sponsored by Nacuity Pharmaceuticals, Inc. are evaluating N-acetylcysteine amide across Phase 1 through Phase 3 development for Retinitis Pigmentosa, Usher Syndrome, and Cystinosis. A Phase 3 trial, currently not yet recruiting, focuses on photoreceptor preservation in Retinitis Pigmentosa and Usher Syndrome. Additionally, an active, non-recruiting Phase 1/2 trial assesses the pharmacokinetic and pharmacodynamic properties of an oral formulation in patients with Cystinosis.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT07290530	NOT_YET_RECRUITING	Retinitis Pigmentosa (RP); Usher Syndrome	Nacuity Pharmaceuticals, Inc.	2026-12	PHASE3
NCT05994534	ACTIVE_NOT_RECRUITING	Cystinosis	Nacuity Pharmaceuticals, Inc.	2023-10-29	PHASE1; PHASE2

(data from https://clinicaltrials.gov, updated on 2026-07-01)

Check the N-Acetylcysteine amide (NACA) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

As a membrane-penetrating antioxidant, N-acetylcysteine amide attenuates reactive oxygen species accumulation and inhibits downstream activation of the NF-κB and HIF-1α signaling pathways. This suppression of oxidative stress and pro-inflammatory signaling prevents cellular apoptosis and tissue damage, offering potential therapeutic efficacy for photoreceptor preservation in Retinitis Pigmentosa and Usher Syndrome as well as metabolic protection in Cystinosis.

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>).