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

Official 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.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 15 participants

Interventions

• GENETIC: nula-cel Drug Product — nula-cel is administered via IV infusion following a myeloablative conditioning regimen

Primary Outcomes

• Proportion of patients who reach neutrophil engraftment (42 days post-infusion)
• Incidence rate of treatment-related mortality (100 days post-infusion)
• Incidence rate of treatment-related mortality (12 months post-infusion)
• Overall survival (24 months post-infusion)
• Frequency and severity of AEs/SAEs (24 months post-infusion)

Secondary Outcomes

• Time to neutrophil engraftment (through study completion, up to 24 months post-infusion)
• Time to platelet engraftment (through study completion, up to 24 months post-infusion)
• Evaluation of gene correction levels in peripheral myeloid cells (through study completion, up to 24 months post-infusion)
• Evaluation of adult Hgb as a percentage of total Hgb (through study completion, up to 24 months post-infusion)
• Evaluation of HbS as a percentage of total Hgb (through study completion, up to 24 months post-infusion)

Trial Locations

• Children's Hospital Los Angeles, Los Angeles, California, United States
• Lucile Packard Children's Hospital, Palo Alto, California, United States
• Washington University, St Louis, Missouri, United States
• Nationwide Children's Hospital, Columbus, Ohio, United States

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