This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD. Participants will be treated for 64 weeks: for 12 weeks in the dose escalation period, where the dose will be escalated to each participant's individual MTD; and for 52 weeks at the MTD in the maintenance period.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
63
Participants will receive anumigilimab, escalated to a maximum dose guided by absolute neutrophil count (ANC) and safety concerns.
Volume matched saline will be administered SC.
UAMS Medical Center
Little Rock, Arkansas, United States
RECRUITINGUniversity of California Irvine
Number of participants with Treatment Emergent Adverse Events (TEAEs), Overall and by severity, seriousness and relationship to Investigational Product (IP)
Time frame: From baseline up to Week 75
Percentage of participants with TEAEs Overall and by severity, seriousness and relationship to IP
Time frame: From baseline up to Week 75
Number of participants with Adverse Events of Special Interest (AESI)
The following AEs are to be reported as AESI: neutropenia (Common Terminology Criteria for Adverse Events \[CTCAE\] Grade 3 or 4), severe infection and neutropenic fever.
Time frame: From baseline up to Week 75
Percentage of participants with AESI
The following AEs are to be reported as AESI: neutropenia (CTCAE Grade 3 or 4), severe infection and neutropenic fever.
Time frame: From baseline up to Week 75
Number of participants with clinically relevant changes from baseline in laboratory assessments and vital signs
Time frame: From baseline up to Week 75
Percentage of participants with clinically relevant changes from baseline in laboratory assessments and vital signs
Time frame: From baseline up to Week 75
Serum concentration of Anumigilimab
Time frame: From the first dose of anumigilimab to Week 65
Annualized rate of Vaso-occlusive crisis (VOC) (medical facility)
VOC (medical facility) is defined as VOC that results in a visit to a medical facility and treatment with parenteral opioids or a parenteral nonsteroidal anti-inflammatory drug.
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Orange, California, United States
The Foundation for Sickle Cell Disease
Hollywood, Florida, United States
RECRUITINGArthur M. Blank Hospital- Children's Healthcare of Atlanta
Atlanta, Georgia, United States
RECRUITINGCenter for Cancer and Blood Disorders
Bethesda, Maryland, United States
RECRUITINGSouthern Specialty Research
Flowood, Mississippi, United States
RECRUITINGJacobi Medical Center
The Bronx, New York, United States
RECRUITINGOhio State University
Columbus, Ohio, United States
RECRUITINGVirginia Commonwealth University
Richmond, Virginia, United States
RECRUITINGCHU Saint-Pierre
Brussels, Belgium
RECRUITING...and 6 more locations
Time frame: During the maintenance period from Week 13 to Week 65