A Phase I/II Study of the Correction of a Single Nucleotide Mutation (Adenosine –> Thymine [A–>T]) in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease

Study point of contact

Allison Intondi, PhD
650-484-0886
[email protected]

Locations

1 United States site

Age

12 to 40 Years

Phase

Phase 1/Phase 2

Study type

Interventional

Gender

All

Interventions

Genetic

Compensation

Unknown

About the study

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.

participation requirements

– ≥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

participation restrictions

– 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

Locations

  • Palo Alto, California, United States, Lucile Packard Children's Hospital, 94304