Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease
- Registration Number
- NCT05445128
- Lead Sponsor
- Ensoma
- 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...
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 1
- Subject must be ≥18 to ≤35 years of age.
- Subject must weigh ≥30 kg.
- Subject must have a diagnosis of Sickle Cell Disease.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A: Single Day Dosing/Apheresis MGTA-145 Single dose of MGTA-145 in combination with plerixafor followed by apheresis Part A: Single Day Dosing/Apheresis Plerixafor Single dose of MGTA-145 in combination with plerixafor followed by apheresis Part B: 2-Day Dosing/Apheresis MGTA-145 MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days Part B: 2-Day Dosing/Apheresis Plerixafor MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
- Primary Outcome Measures
Name Time Method 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 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 Apheresis Collection Yield Up to 2 days Determination of the yield of CD34+ cells after either one or two consecutive days of MGTA-145 and plerixafor mobilization followed by apheresis.
Vital Signs - Number of participants with clinically significant changes from baseline in vital signs Up to 11 days Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters. Up to 11 days
- Secondary Outcome Measures
Name Time Method Mobilization Effects of single-day and two-day dosing with MGTA-145 and plerixafor in peripheral blood in patients with SCD Up to 2 days Determination of peak peripheral blood CD34+ counts
Assess presence of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA]) Up to 11 days Investigate plasma concentrations of MGTA-145 per timepoint of collection (Pharmacokinetics) Up to 2 days Assess titers of MGTA-145 Anti-Drug Antibodies (ADA) in plasma samples (using electrochemiluminescent immunoassay [ECLIA]) Up to 11 days
Trial Locations
- Locations (3)
National Institutes of Health
🇺🇸Bethesda, Maryland, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
St. Jude Children's Research Hospital
🇺🇸Memphis, Tennessee, United States