| NCT02186418 |
SCD (2) BT (9, of which 6 |
IV Melphalan Treosulfan |
Plerixafor G-CSF and |
1 and 6.9 19.5 (I.O. |
RVT1801 |
0.3 |
Aruvant/ CCHMC San Raffaele Telethon |
| NCT02453477 |
pediatric) |
and Thiotepa |
Plerixafor |
infusion) |
GLOBE |
0.93 |
Institute for Gene Therapy (SR-TIGET) Italy |
|
NCT01745120 (HGB-204) |
BT (18 as of 4/2018) |
Busulfan Busulfan (adjusted |
G-CSF and Plerixafor G-CSF and Plerixafor |
8.1 |
BB305 |
0.7 |
bluebird bio (multicenter, 6 sites) bluebird bio (Necker |
|
NCT02151526 (HGB-205) |
BT and SCD (4 as of 4/2018) |
based on daily PK monitoring) |
(after 3 months of enhanced transfusion) |
10.5 |
BB305 |
1.3 |
Children’s Hospital in Paris) |
|
NCT02140554 (HGB-206) 3 groups: A, B, C |
SCD (7, A); (2, B); and (9, C) |
Busulfan |
BM harvest (A, B)/ Plerixafor (C) |
2.1 (A) 2.7 (B) 6.5 (C) |
BB305 |
0.6 (A) 3.1 (B) 3.8 (C) |
bluebird bio (USA) |
|
NCT02906202 (HGB 207) |
Non beta0/0 BT including pediatric (16) |
Busulfan |
G-CSF and Plerixafor |
NA |
BB305 |
3.1 |
bluebird bio (international, multicenter) |
|
NCT03207009 (HGB 212) |
beta0/0 BT (3) |
Busulfan |
G-CSF and Plerixafor |
NA |
BB305 |
NA |
bluebird bio (international, multicenter) |
