From: A theoretical framework for early human studies: uncertainty, intervention ensembles, and boundaries
 | Examples | Cetuximab | Belimumab | Gene transfer for AID-SCID |
---|---|---|---|---|
Treatment dimension | Â | |||
Dose | mg/kg; cells; vector particles… | 250 mg/m2 | 10 mg/kg | ≥8 × 10^6 cells |
Schedule | Daily; 4× weekly w/2 wk holidays… | Weekly until disease progression | 3× @ 2-wk intervals; 4-wk intervals after |  |
Administration/target | Infusion; oral; intratumoral… | Intravenous infusion | Intravenous infusion | Intravenous infusion |
Co-intervention | Radiotherapy; immunosuppresion… |  | Standard of care | Non-myeloablative conditioning |
Risk mitigation | Cardiac monitoring; liver enzymes… | Serum electrolytes monitoring | Monitor patients for hypersensitivity |  |
Timing delivery | Symptom onset; 12 h after event… | After irinotecan or oxaliplatin failure |  | 3 weeks after stopping PEG-ADA |
Population dimensions | ||||
Diagnostic criteria | Marker positivity; diagnostic score… | KRAS mutation in codon 12 or 13 | α-nuclear Ab titer ≥ 1:80 and/or α-dsDNA |  |
Contraindications | Concurrent infection; clothing disorder… |  | Prior anaphylaxis to belimumab | Neutralizing Abs to vector (AAV8) |
Indications | Disease; injury; at-risk population… | Metastatic colorectal cancer | Active systemic lupus erythematosus (SLE) | ADA-SCID |
Age | Â | Adults | Non-geriatric adults | Children |
Outcome dimensions | ||||
Endpoint | Change on scale; time to event… | Survival | SLE responder index | Immune system reconstitution |
Duration | Short term; ‘long term’ remission… |  | 1 year | Permanent |