Table 10.
Practical checklist to prepare for clinical trials with non-medicinal investigational products
| Raw material | |
| Manufacturer | |
| Source | |
| Production | |
| Quality control (characteristics) | |
| Toxicity studies in vitro, animals, humans | |
| Final product | |
| Manufacturer/Pharmacist | |
| Pre-define the criteria the final product should meet | |
| Reconstitution protocols | |
| Sterilisation protocols | |
| Quality control (characteristics) | |
| Safety control | |
| Dosage/concentration analysis | |
| Contamination with relevant substances, e.g. endotoxion, heavy metals | |
| Shelf life | |
| Pharmacokinetics | |
| Toxicity studies in vitro, animals, humans | |
| Clinical data | |
| Pharmacokinetics | |
| Subject characteristics | |
| Relevant literature | |
| Overall risk-benefit assessment | |
| Administration route (reduce safety risks if possible) | |
| Mechanism of action (tissue specificity) | |
| Analysis of potential effect | |
| Manageability of potential effects | |
| Estimate the risk of side effects | |
| Pre-define how to manage potential effects | |
| Dosage (based on First-In-Man (FIM) guidelines based on the No Observed Adverse Effect Level (NOAEL) in non-clinical safety studies adjusted with allometric factors) | |
| Quantitative regular daily exposure | |
| Study design (e.g. dose-escalation or pilot study) | |
| Subject characteristics (medical history, age etc.) | |
| Appoint an independent data safety monitoring board | |
| Predefine safety endpoints (Table 6) | |
| Perform/report interim analyses on safety criteria during the study. |