NCT05445128

Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease

A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of MGTA-145 in Combination With Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients With Sickle Cell Disease

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Ensoma · Industry
• Sex: All
• Age: 18 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor.

Detailed description

This Phase 2, multicenter, open-label study will be conducted in 2 parts (Parts A and B). Part A is intended to characterize the efficacy, safety, PK and PD of a single dose of MGTA-145 and plerixafor for HSC mobilization and apheresis collection in patients with SCD. Part B is designed to characterize the efficacy, safety, PK and PD of 2 consecutive days of dosing with MGTA-145 and plerixafor for HSC mobilization and apheresis collection in patients with SCD.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
BIOLOGICAL	MGTA-145	MGTA-145 will be administered as an IV infusion
DRUG	Plerixafor	240 µg/kg administered subcutaneously

Timeline

• Start date: 2022-06-24
• Primary completion: 2022-12-08
• Completion: 2023-02-02
• First posted: 2022-07-06
• Last updated: 2025-07-25
• Results posted: 2025-07-25

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study