Several clinical trials evaluate therapeutic interventions across indications such as systemic lupus erythematosus, non-alcoholic fatty liver disease, idiopathic pulmonary fibrosis, Parkinson disease, and uterine corpus carcinoma associated with Lynch syndrome. Spanning Phase 1, Phase 1/2, Phase 2, and unassigned stages, these studies aim to determine safety, pharmacokinetics, and therapeutic efficacy. Sponsored by industry, academic, and government entities—including GlaxoSmithKline, the National Cancer Institute, the Gynecologic Oncology Group, and the State University of New York at Buffalo—recruitment statuses range from not yet recruiting to actively recruiting.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT01199250 | Not yet recruiting | Lynch Syndrome|Recurrent Uterine Corpus Carcinoma|Stage I Uterine Corpus Cancer|Stage II Uterine Corpus Cancer|Stage III Uterine Corpus Cancer|Stage IV Uterine Corpus Cancer |
Gynecologic Oncology Group|National Cancer Institute (NCI)|GOG Foundation |
2100-01 | -- |
| NCT06339034 | Not yet recruiting | Parkinson Disease |
State University of New York at Buffalo|The Cure Parkinson''s Trust |
2024-06 | Phase 1|Phase 2 |
| NCT06317285 | Not yet recruiting | Idiopathic Pulmonary Fibrosis |
GlaxoSmithKline |
2024-04-01 | Phase 2 |
| NCT06104319 | Recruiting | Non-alcoholic Fatty Liver Disease |
GlaxoSmithKline |
2024-01-22 | Phase 2 |
| NCT06188507 | Not yet recruiting | Systemic Lupus Erythematosus |
GlaxoSmithKline |
2024-01-11 | Phase 1 |
(data from https://clinicaltrials.gov, updated on 2024-05-22)
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>).