A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
Who is this study for? Patients with severe sickle-cell disease
What treatments are being studied? GPH101
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel 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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 40
Healthy Volunteers: f
View:
• ≥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
Locations
United States
California
Lucile Packard Children's Hospital
RECRUITING
Palo Alto
Missouri
Washington University
RECRUITING
St Louis
Ohio
Nationwide Children's Hospital
NOT_YET_RECRUITING
Columbus
Contact Information
Primary
Restore Clinical Study Support
RestoreStudySupport@kamautx.com
650-416-6562
Time Frame
Start Date: 2021-11-15
Estimated Completion Date: 2028-12-31
Participants
Target number of participants: 15
Treatments
Experimental: nula-cel Drug Product
nula-cel Drug Product is a human autologous CRISPR-Cas9 edited and sickle mutation-corrected HSPC product.
Related Therapeutic Areas
Sponsors
Leads: Kamau Therapeutics