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

Phase 2
Terminated
Conditions
Interventions
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
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.
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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.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Part A: Single Day Dosing/ApheresisMGTA-145Single dose of MGTA-145 in combination with plerixafor followed by apheresis
Part A: Single Day Dosing/ApheresisPlerixaforSingle dose of MGTA-145 in combination with plerixafor followed by apheresis
Part B: 2-Day Dosing/ApheresisMGTA-145MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
Part B: 2-Day Dosing/ApheresisPlerixaforMGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
Primary Outcome Measures
NameTimeMethod
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 YieldUp 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 signsUp 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
NameTimeMethod
Mobilization Effects of single-day and two-day dosing with MGTA-145 and plerixafor in peripheral blood in patients with SCDUp 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

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