A Study of HQK-1001 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: NCT01322269
Recruitment Status : Completed
First Posted : March 24, 2011
Last Update Posted : June 19, 2013
Information provided by (Responsible Party):
HemaQuest Pharmaceuticals Inc.

Brief Summary:
The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Sickle Cell Anemia Sickle Cell Disorders Hemoglobin S Disease Sickling Disorder Due to Hemoglobin S Drug: HQK-1001 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 52 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease
Study Start Date : April 2011
Primary Completion Date : March 2012

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: HQK-1001 (30 mg/kg) Drug: HQK-1001
HQK-1001 tablets, once daily for daily 26 weeks
Experimental: HQK-1001 (40 mg/kg) Drug: HQK-1001
HQK-1001 tablets, once daily for daily 26 weeks
Experimental: HQK-1001 (50 mg/kg) Drug: HQK-1001
HQK-1001 tablets, once daily for daily 26 weeks

Primary Outcome Measures :
  1. Safety [ Time Frame: Day 1 through Week 30 ]
    Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.

Secondary Outcome Measures :
  1. Fetal hemoglobin levels [ Time Frame: Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30 ]
  2. Incidence of sickle cell crisis events [ Time Frame: Day 1 through Week 30 ]

Information from the National Library of Medicine

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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:

  • Established diagnosis of SCD
  • Males and females between 12 and 60 years of age, inclusive
  • At least 3 episodes of a SCD-related crisis or complication in the 3 years prior to screening OR 1 episode of acute chest syndrome in the 5 years prior to screening
  • If receiving hydroxyurea, must be receiving a stable dose for at least 6 months prior to screening
  • If hydroxyurea treatment has been discontinued, at least 3 months have elapsed since last dose
  • If transfusion in the 4 months prior to screening, then HbA level < 20% at screening
  • Average of the initial two HbF levels ≥ 2.0 % within ≤ 7 days prior to the initial dose of HQK-1001. The two must be obtained ≥ 24 hours apart
  • Ability to swallow tablets
  • Able and willing to give informed consent and assent (if applicable)
  • If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 7 days of first dose of HQK-1001
  • If a subject is a WCBP, she must agree to use an effective form of contraception within 7 days of the initial dose of HQK-1001 and for one month after HQK-1001 discontinuation
  • Sexually active male subjects (with WCBP partners) must agree to use latex condoms or ensure that their partner(s) use an effective form of contraception
  • In the view of the Investigator, subject is able and willing to comply with necessary study procedures

Exclusion Criteria:

  • More than 4 hospitalizations for acute sickle cell related events in the previous 12 months prior to screening
  • Pulmonary hypertension requiring oxygen therapy
  • QTc > 450 msec (male) or 470 msec (female) on screening ECG (QT corrected by Fridericia's formula)
  • Assigned to a regular transfusion program
  • Use of erythropoiesis stimulating agents within 90 days of screening
  • ALT > 3x upper limit of normal (ULN)
  • Serum creatinine > 1.2 mg/dL
  • A serious, concurrent illness that would limit ability to complete or comply with the study requirements
  • An acute vaso-occlusive event within 3 weeks prior to screening
  • Creatine phosphokinase (CK) > 20% above the ULN
  • An acute illness (e.g., febrile, GI, respiratory) within 72 hours prior to screening
  • 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 screening
  • 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): NCT01322269

United States, California
Children's Hospital and Research Center - Oakland
Oakland, California, United States, 94609
United States, Florida
University of Miami Miller School of Medicine - Dept of Pediatrics
Miami, Florida, United States, 33101
United States, Georgia
Georgia Health Sciences University - Adult SIckle Cell Center
Augusta, Georgia, United States, 30912
United States, Illinois
University of Illinois at Chicago - Dept of Pediatrics
Chicago, Illinois, United States, 60612
United States, Louisiana
LSU Health Sciences Center - Feist Weiller Cancer Center
Shreveport, Louisiana, United States, 71103
United States, Massachusetts
Tufts Medical Center
Boston, Massachusetts, United States, 02111
United States, North Carolina
University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Texas
Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology
Dallas, Texas, United States, 75390-9063
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, MSG 1X8
University Health Network Toronto General Hospital
Toronto, Ontario, Canada, MSG 2C4
Abu El Reesh Pediatric University Hospital
Cairo, Egypt
University of the West Indies - Sickle Cell Unit
Mona, Kingston, Jamaica
American University of Beirut Medical Center
Beirut, Lebanon
Rafik Hariri University Hospital
Beirut, Lebanon
Chronic Care Center
Hazmieh, Lebanon
Sponsors and Collaborators
HemaQuest Pharmaceuticals Inc.
Study Director: Richard Ghalie, MD, MBA HemaQuest Pharmaceuticals Inc.

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: HemaQuest Pharmaceuticals Inc. Identifier: NCT01322269     History of Changes
Other Study ID Numbers: HQP 1001-SCD-006
First Posted: March 24, 2011    Key Record Dates
Last Update Posted: June 19, 2013
Last Verified: June 2013

Additional relevant MeSH terms:
Anemia, Sickle Cell
Sickle Cell Trait
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn