A new clinical trial investigating a potential new treatment called MGTA-145 to mobilize stem cells and apheresis collection in patients with sickle cell disease (SCD) has just opened. 

During the phase 2, multicenter, open-label trial that consists of 2 parts, MGTA-145 will be given together with plerixafor, an immunostimulant used to mobilize hematopoietic stem cells into the bloodstream in patients with cancer. 

The first part of the trial aims to characterize the safety, efficacy, pharmacokinetics, and pharmacodynamics of a single dose of MGTA-145 plus plerixafor while the second part will characterize the safety, efficacy, pharmacokinetics, and pharmacodynamics of MGTA-145 and plerixafor gave on 2 consecutive days.

The trial aims to enroll 14 patients with SCD, aged 18 to 35 years. The primary outcome measures of the trial include the yield of apheresis collection, the incidence of treatment-emergent adverse events, and the number of participants with clinically significant changes from baseline in vital signs and hematology and clinical chemistry laboratory parameters.

Secondary outcome measures include the mobilization effects of single-day and 2-day dosing with MGTA-145 and plerixafor, the plasma concentrations of MGTA-145, and the presence and titer of antidrug antibodies in the plasma.

Read more about the treatment of SCD

The trial is currently recruiting participants at Boston Children’s Hospital in Massachusetts and is expected to be completed in December 2023.

MGTA-145 is a chemokine receptor type 2 agonist protein that is being developed by Magenta Therapeutics. In a phase 1 clinical trial in healthy volunteers, MGTA-145 has been shown to rapidly and reliably mobilize high numbers of functional stem cells in just 1 day. (Current mobilization standard of care requires at least 5 days).

There is currently no cure for SCD. However, there are several gene transfer and gene editing strategies that are being investigated to allow the development of a one-time cure for the disease. The collection of a sufficient number of hematopoietic stem cells of high purity is essential for such a treatment.

Reference

Study of MGTA-145 and plerixafor in patients with sickle cell disease. US National Library of Medicine. Updated July 6, 2022. Accessed July 7, 2022.