Clinical trial NCT05445128: Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Sickle Cell Disease
Keywords: SCD
MGTA-145
Plerixafor
Mobilization
Apheresis
Lead sponsor: Ensoma
Sponsor class: INDUSTRY
Collaborators: name: bluebird bio
First posted: 2022-07-06
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Maximum age (years): 35
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Apheresis Collection Yield
timeFrame: Up to 2 days
measure: 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.
timeFrame: Up to 30 days
measure: 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.
timeFrame: Up to 11 days
measure: Vital Signs - Number of participants with clinically significant changes from baseline in vital signs
timeFrame: Up to 11 days
measure: Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters.
timeFrame: Up to 11 days
Secondary outcomes:
measure: Mobilization Effects of single-day and two-day dosing with MGTA-145 and plerixafor in peripheral blood in patients with SCD
timeFrame: Up to 2 days
measure: Investigate plasma concentrations of MGTA-145 per timepoint of collection (Pharmacokinetics)
timeFrame: Up to 2 days
measure: Assess presence of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA])
timeFrame: Up to 11 days
measure: Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA])
timeFrame: Up to 11 days
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: Magenta Therapeutics
name: Ji Hyun Lee, MD, MPH
role: STUDY_DIRECTOR
References: