| Allison Intondi, PhD | |
| 650-484-0886 | |
| [email protected] |
1 United States site
12 to 40 Years
Phase 1/Phase 2
Interventional
All
Genetic
Unknown
This study is a first-in-human, single-arm, open-label Phase I/II study of GPH101 in
approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary
objective is to evaluate safety of the treatment in this patient population, as well as
preliminary efficacy and pharmacodynamic data.
– ≥12 to ≤ 40 years
– Severe disease, as defined by having experienced at least one of the following
SCD-related events despite appropriate supportive care measures:
– recurrent severe VOC (≥ 4 episodes in the preceding 2 years)
– ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year)
– Lansky/Karnofsky performance status of ≥ 80
– Available 10/10 HLA-matched donor
– Prior HSCT or gene therapy
– Prior or current malignancy or myeloproliferative or a significant coagulation or
immunodeficiency disorder
– Clinically significant and active bacterial, viral, fungal or parasitic infection
– Pregnancy or breastfeeding in a postpartum female