3,4-Diaminopyridine Clinical Trials

Size	Price	Qty
25mg	USD 97
1g	USD 870

Clinical Trials

A Phase 2 clinical trial sponsored by Catalyst Pharmaceuticals Inc. evaluated 3,4-diaminopyridine to assess its long-term safety in ambulatory patients with Spinal Muscular Atrophy Type 3. Designed to investigate the drug's potential for managing neuromuscular symptoms in this population, the recruitment status for this investigation was ultimately documented as terminated. Overall, these clinical data summarize the investigation of 3,4-diaminopyridine as a potential therapeutic agent for patients with Spinal Muscular Atrophy Type 3.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT03819660	Terminated	Spinal Muscular Atrophy Type 3	Catalyst Pharmaceuticals Inc.	2019-03-07	Phase 2

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

Check the 3,4-Diaminopyridine product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

3,4-Diaminopyridine selectively blocks presynaptic voltage-gated potassium channels, which prolongs the action potential duration and increases presynaptic calcium ion influx. This enhanced calcium entry facilitates the exocytosis of acetylcholine at the neuromuscular junction, providing a mechanistic rationale for restoring neuromuscular transmission in conditions such as Spinal Muscular Atrophy Type 3.

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>).