A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease

Official Summary

This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 4 participants

Interventions

• BIOLOGICAL: OTQ923 — Single intravenous infusion of OTQ923 cell suspension
• BIOLOGICAL: OTQ923 — Single intravenous infusion of OTQ923, based on review of data from Part A by Health agencies after a formal interim analysis

Primary Outcomes

• Number of participants with adverse events and serious adverse events (up to 24 months)
• Fetal hemoglobin (HbF) expression 6 months after hematopoietic stem cell transplant (HSCT) (at 6 months)
• Time to reach absolute neutrophil count (ANC) ≥500/μL for 3 consecutive days (up to 24 months)

Secondary Outcomes

• Durability of hematologic engraftment (up to 24 months)
• Proportion of subject to achieve 30% of total HbF at 12 months (12 months)
• Time to achieve 30% total HbF (up to 24 months)
• Time to peak total HbF (up to 24 months)
• Percentage of edited WBC and bone marrow cells by time points (up to 24 months)

Trial Locations

• University of Chicago, Chicago, Illinois, United States
• Memorial Sloan Kettering Cancer Ctr, New York, New York, United States
• St Jude Children's Research Hospital, Memphis, Tennessee, United States

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