NCT04819841

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Kamau Therapeutics · Industry
• Sex: All
• Age: 12 Years – 40 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Participants diagnosed with severe SCD will receive nula-cel via IV infusion following myeloablative conditioning in an autologous HSCT setting.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
GENETIC	nula-cel Drug Product	nula-cel is administered via IV infusion following a myeloablative conditioning regimen

Timeline

• Start date: 2021-11-15
• Primary completion: 2026-12-31
• Completion: 2028-12-31
• First posted: 2021-03-29
• Last updated: 2026-03-11

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study