Table 3.
Summary of recent clinical trials targeting CGD. SAE, serious adverse event.
| Trial centre | Vector | Conditioning | Patients | Outcome | SAE | Insertion site(s) | References |
|---|---|---|---|---|---|---|---|
| US | γ-Retroviral (amphotropic) | None | 5 | No clinical benefit | None | – |
Malech et al. (1997) Malech (2000) Goebel and Dinauer (2003) Kang et al. (2010) |
| None | 5 | No clinical benefit | None | – | |||
| Busulfan (10 mg/kg) | 3 | Transient clinical benefit | None | – | |||
| Germany | γ-Retroviral (SFFV LTR) | Busulfan (8.8 mg/kg) | 2 | Long term clinical benefit | Both developed MDS with monosomy 7; 1 died from sepsis | MDS EVI1 |
Ott et al. (2006) Stein et al. (2010) Bianchi et al. (2009) Bianchi et al. (2011) |
| Switzerland | 2 | Transient clinical benefit | 1 patient developed MDS | None | |||
| UK | γ-Retroviral (MLV LTR) | Melphalan (140 mg/m2) | 1 | Transient clinical benefit | None | – |
Personal communication Thrasher AJ |
| γ-Retroviral (SFFV LTR) | 3 | ||||||
| Korea | γ-Retroviral | Busulfan (6.4 mg/kg) + Fludarabine (120 mg/m2) | 2 | Transient clinical benefit | None | – | Kang et al. (2011) |
| Switzerland, Germany, France, UK | SIN lentivector, myeloid promoter | Busulfan (12–16 mg/kg) | 1 | Trial open | – | – | – |