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Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00842088
Recruitment Status : Completed
First Posted : February 12, 2009
Last Update Posted : August 1, 2011
Information provided by:
HemaQuest Pharmaceuticals Inc.

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Placebo Drug: HQK-1001 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Blinded, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 in Subjects With Sickle Cell Disease
Study Start Date : March 2009
Actual Primary Completion Date : July 2010

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Active Drug: HQK-1001
HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days.

Placebo Comparator: Placebo Drug: Placebo
Matching placebo capsule. Administered orally once a day on dosing days.

Primary Outcome Measures :
  1. Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam. [ Time Frame: 126 days ]

Secondary Outcome Measures :
  1. Pharmacokinetics assessed by plasma drug concentration levels. [ Time Frame: Days 0, 6, 69 and 97 post first dose ]
  2. Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin. [ Time Frame: Every 2 weeks through Day 126 post first dose ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   12 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of SCD or sickle beta thalassemia (excluding Hemoglobin C)
  • Between 12 and 60 years of age, inclusive
  • At least one episode of a SCD-related crisis or complication (e.g., vaso-occlusive crisis, acute chest syndrome, priapism) per year for an average of 3 years or one episode of acute chest syndrome over the prior 5 years
  • Screening (untransfused) HbF level >/= 2% as analyzed by a central laboratory
  • If receiving hydroxyurea therapy, must be receiving a stable dose for at least 6 months
  • Able and willing to give informed consent
  • If female, must have a negative serum pregnancy test within 7 days of dosing
  • If female, must not be of childbearing potential defined as post-menopausal by at least 2 years or surgically sterile, or must agree to use a medically accepted form of contraception throughout the study
  • If the sexual partner of a male subject is a WCBP, she must agree to use a medically accepted form of birth control for themselves or their partner throughout the study
  • In the view of the Investigator, able to comply with necessary study procedures

Exclusion Criteria:

  • Red blood cell (RBC) transfusion within 3 months prior to beginning study medication
  • Participation in a regular blood transfusion program
  • More than 4 hospitalizations for acute sickle cell-related events in the previous 12 months
  • An acute vaso-occlusive event within 3 weeks prior to receiving first dose of study medication
  • Pulmonary hypertension requiring oxygen
  • QTc > 450 msec on screening
  • Alanine transaminase (ALT) > 3X upper limit of normal (ULN)
  • Creatinine phosphokinase (CPK) > 20% above the ULN
  • Serum creatinine >1.2 mg/dL
  • An acute illness (e.g., febrile, gastrointestinal [GI], respiratory) within 72 hours prior to receiving first dose of study medication
  • History of syncope, clinically significant dysrhythmias or resuscitation from sudden death
  • Chronic opiate use which, in the view of the Investigator, could confound evaluation of an investigational drug
  • Current abuse of alcohol or drugs
  • Received another investigational agent within 4 weeks, or 5 half-lives, whichever is longer, prior to administration of study medication
  • Currently pregnant or breast feeding a child
  • Known infection with HIV-1
  • Infection with hepatitis B or hepatitis C such that patients are currently on therapy or will be placed on therapy during the trial

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00842088

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United States, Alabama
Trialogic Research
Madison, Alabama, United States, 35758
United States, California
Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
United States, Florida
Century Clinical Research, Inc.
Daytona Beach, Florida, United States, 32117
United States, Georgia
Medical College of Georgia
Augusta, Georgia, United States, 30912
United States, Illinois
University of Illinois at Chicago
Chicago, Illinois, United States, 60612
United States, Maryland
Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21205
United States, North Carolina
UNC Comprehensive Sickle Cell Program
Chapel Hill, North Carolina, United States, 27599
United States, Texas
Texas Children's Cancer Center and Hematology Service
Houston, Texas, United States, 77030
University of the West Indies, Mona
Kingston, Mona, Jamaica, 7
Sponsors and Collaborators
HemaQuest Pharmaceuticals Inc.
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Study Director: Susan Perrine, M.D. HemaQuest Pharmaceuticals
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Susan Perrine, M.D., HemaQuest Pharmaceuticals, Inc. Identifier: NCT00842088    
Other Study ID Numbers: HQP 2008-004
First Posted: February 12, 2009    Key Record Dates
Last Update Posted: August 1, 2011
Last Verified: July 2011
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn