A completed Phase 1 clinical trial evaluated 2-furoic acid to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with sickle cell disease. Conducted through a multi-institutional collaboration spanning academic, government, and industry entities—including the National Heart, Lung, and Blood Institute, TRND, Baxalta, and Takeda—this study marks an important milestone in determining the compound's therapeutic potential for managing red blood cell disorders.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT01597401 | Completed | Sickle Cell Disease |
Baxalta now part of Shire|SAIC-Frederick Inc.|Therapeutics for Rare and Neglected Diseases (TRND)|QS Pharma|National Chung Cheng University|Infrared Imaging and Thermometry Unit Biomedical Engineering and Physical Science Shared Resource (NIBIB)|ClinPharm Consulting LLC|Ricerca Biosciences LLC|National Heart Lung and Blood Institute (NHLBI)|Cato Research|Takeda |
2012-05-12 | Phase 1 |
(data from https://clinicaltrials.gov, updated on 2024-05-22)
Check the
2-Furoic acid
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
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>).
Note: Technical data last updated: Sep 1, 2026.