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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05445128
• Other study ID #: 145-SCD-204
• Status: Terminated
• Phase: Phase 2
• Start date: June 24, 2022
• Est. completion date: February 2, 2023

Study information

• Verified date: January 2024
• Source: Ensoma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 1
• Est. completion date: February 2, 2023
• Est. primary completion date: December 8, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 35 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Biological:
• MGTA-145
MGTA-145 will be administered as an IV infusion

Drug:
• Plerixafor
240 µg/kg administered subcutaneously

Locations

Country	Name	City	State
United States	National Institutes of Health	Bethesda	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	St. Jude Children's Research Hospital	Memphis	Tennessee

Sponsors (2)

Lead Sponsor	Collaborator
Ensoma	bluebird bio

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Apheresis Collection Yield	Determination of the yield of CD34+ cells after either one or two consecutive days of MGTA-145 and plerixafor mobilization followed by apheresis.	Up to 2 days
Primary	Assess incidence of treatment emergent adverse events leading to study drug discontinuation based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.		Up to 30 days
Primary	Assess the incidence of treatment emergent >/= Grade 3 clinical laboratory abnormalities based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.		Up to 11 days
Primary	Vital Signs - Number of participants with clinically significant changes from baseline in vital signs		Up to 11 days
Primary	Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters.		Up to 11 days
Secondary	Mobilization Effects of single-day and two-day dosing with MGTA-145 and plerixafor in peripheral blood in patients with SCD	Determination of peak peripheral blood CD34+ counts	Up to 2 days
Secondary	Investigate plasma concentrations of MGTA-145 per timepoint of collection (Pharmacokinetics)		Up to 2 days
Secondary	Assess presence of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA])		Up to 11 days
Secondary	Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA])		Up to 11 days