Clinical Trials
Multiple clinical trials sponsored by Summit Therapeutics have investigated ezutromid for the treatment of Duchenne muscular dystrophy. Although a Phase 1 clinical trial successfully completed its evaluation of the drug's safety and pharmacokinetic profile, a subsequent Phase 2 clinical trial assessing utrophin modulation in ambulatory pediatric male patients was ultimately terminated. Together, these investigations characterize the clinical landscape for ezutromid in addressing dystrophin-deficient neuromuscular conditions.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT02858362 | Terminated | Duchenne Muscular Dystrophy |
Summit Therapeutics |
2016-06 | Phase 2 |
| NCT02383511 | Completed | Muscular Dystrophy Duchenne |
Summit Therapeutics |
2015-02 | Phase 1 |
(data from https://clinicaltrials.gov, updated on 2024-05-22)
Compliance for Clinical Use
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>).