The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
24
Trialogic Research
Madison, Alabama, United States
Children's Hospital and Research Center at Oakland
Oakland, California, United States
Century Clinical Research, Inc.
Daytona Beach, Florida, United States
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.
Time frame: 126 days
Pharmacokinetics assessed by plasma drug concentration levels.
Time frame: Days 0, 6, 69 and 97 post first dose
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin.
Time frame: Every 2 weeks through Day 126 post first dose
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Medical College of Georgia
Augusta, Georgia, United States
University of Illinois at Chicago
Chicago, Illinois, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
UNC Comprehensive Sickle Cell Program
Chapel Hill, North Carolina, United States
Texas Children's Cancer Center and Hematology Service
Houston, Texas, United States
University of the West Indies, Mona
Kingston, Mona, Jamaica