NCT03653247

A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

A Phase 1/2, Open-Label, Multicenter, Single-Arm Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Sangamo Therapeutics · Industry
• Sex: All
• Age: 18 Years – 40 Years
• Healthy volunteers: Not accepted

Summary

This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using BIVV003.

Detailed description

Subject participation in this study will be approximately 136 weeks. Enrolled subjects will be asked to participate in a separate long-term follow-up study to monitor the safety and efficacy of BIVV003 treatment for a total of 15 years post-transplant.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
BIOLOGICAL	Plerixafor	Plerixafor subcutaneous injection will be administered prior to apheresis.
DRUG	Busulfan	Busulfan IV infusion will be administered as myeloablative conditioning therapy.
GENETIC	BIVV003	BIVV003 will be administered as an IV infusion following myeloablative conditioning with busulfan.

Timeline

• Start date: 2019-03-06
• Primary completion: 2025-07-17
• Completion: 2025-07-17
• First posted: 2018-08-31
• Last updated: 2025-09-12

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study