Dose of the investigational drug and adverse events of bacterial and fungal infections by study phases
| Phase | Parameter | Thalidomide (n = 3) | Placebo (n = 5) | ||||||
|---|---|---|---|---|---|---|---|---|---|
| T1 | T2 | T3 | P1 | P2 | P3 | P4 | P5 | ||
| Blinded phase | Infection | Aspergillosis CRP increase | None | Otitis media | None | None | None | None | Thymic abscess |
| Pustular eczema | |||||||||
| Lymphadenitis | |||||||||
| Investigational drug dose at initiation, mg | 30 | 130 | 20 | 20 | 90 | 100 | 50 | 20 | |
| (min, max) | (30, 45) | (95, 130) | (20, 30) | (20, 30) | (90, 130) | (100, 150) | (50, 75) | (20, 30) | |
| Dose modification | Increased | Decreased | Increased | Increased | Increased | Increased | Increased | Increased | |
| Reason for modification | Insufficient efficacy | Drowsiness | Insufficient efficacy | Insufficient efficacy | Insufficient efficacy | Insufficient efficacy | Insufficient efficacy | Insufficient efficacy | |
| Extension phase | Infection | None | Perirectal abscess | None | Balanoposthitis | Lymphadenitis | Infectious enteritis | – | Thymic abscess |
| Esophageal candidiasis | |||||||||
| Gastritis | Perirectal abscess | ||||||||
| Thalidomide dose at initiation, mg | 45 | 130 | 30 | 20 | 90 | 100 | 50 | 20 | |
| (min, max) | – | – | – | (20, 30) | – | (100, 150) | – | (20, 30) | |
| Dose modification | – | – | – | Increased | – | Increased | – | Increased | |
| Reason for modification | – | – | – | Insufficient efficacy | – | Insufficient efficacy | – | Insufficient efficacy | |
| Phase | Parameter | Thalidomide ( | Placebo ( | ||||||
|---|---|---|---|---|---|---|---|---|---|
| T1 | T2 | T3 | P1 | P2 | P3 | P4 | P5 | ||
| Blinded phase | Infection | Aspergillosis | None | Otitis media | None | None | None | None | Thymic abscess |
| Pustular eczema | |||||||||
| Lymphadenitis | |||||||||
| Investigational drug dose at initiation, mg | 30 | 130 | 20 | 20 | 90 | 100 | 50 | 20 | |
| (min, max) | (30, 45) | (95, 130) | (20, 30) | (20, 30) | (90, 130) | (100, 150) | (50, 75) | (20, 30) | |
| Dose modification | Increased | Decreased | Increased | Increased | Increased | Increased | Increased | Increased | |
| Reason for modification | Insufficient efficacy | Drowsiness | Insufficient efficacy | Insufficient efficacy | Insufficient efficacy | Insufficient efficacy | Insufficient efficacy | Insufficient efficacy | |
| Extension phase | Infection | None | Perirectal abscess | None | Balanoposthitis | Lymphadenitis | Infectious enteritis | – | Thymic abscess |
| Esophageal candidiasis | |||||||||
| Gastritis | Perirectal abscess | ||||||||
| Thalidomide dose at initiation, mg | 45 | 130 | 30 | 20 | 90 | 100 | 50 | 20 | |
| (min, max) | – | – | – | (20, 30) | – | (100, 150) | – | (20, 30) | |
| Dose modification | – | – | – | Increased | – | Increased | – | Increased | |
| Reason for modification | – | – | – | Insufficient efficacy | – | Insufficient efficacy | – | Insufficient efficacy | |
The CGD-related infections included bacterial and fungal infections, but not viral infections. The table shows the CGD-related infections that developed or worsened during each phase. CRP, C-reactive protein.
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