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

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.

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.

Inclusion Criteria:

• Subject must be ≥18 to ≤35 years of age.
• Subject must weigh ≥30 kg.
• Subject must have a diagnosis of Sickle Cell Disease.

Exclusion Criteria:

• Subject must not have had a vaso-occlusive event (VOE) requiring a visit to a healthcare facility within 30 days of screening.
• Subject must not have undergone or attempted and failed previous hematopoietic stem cell (HSC) collection.
• Subject must not have had a prior autologous or allogeneic transplantation, inclusive of gene therapy.
• Male subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.
• Female subject must not be pregnant or breastfeeding. If sexually active, female subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.