Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
NCT ID: NCT00842088
Last Updated: 2011-08-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1/PHASE2
24 participants
INTERVENTIONAL
2009-03-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Active
HQK-1001
HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days.
Placebo
Placebo
Matching placebo capsule. Administered orally once a day on dosing days.
Interventions
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Placebo
Matching placebo capsule. Administered orally once a day on dosing days.
HQK-1001
HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
12 Years
60 Years
ALL
No
Sponsors
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HemaQuest Pharmaceuticals Inc.
INDUSTRY
Responsible Party
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HemaQuest Pharmaceuticals, Inc.
Principal Investigators
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Susan Perrine, M.D.
Role: STUDY_DIRECTOR
HemaQuest Pharmaceuticals
Locations
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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
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
Countries
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References
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Kutlar A, Ataga K, Reid M, Vichinsky EP, Neumayr L, Blair-Britt L, Labotka R, Glass J, Keefer JR, Wargin WA, Berenson R, Perrine SP. A phase 1/2 trial of HQK-1001, an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2012 Nov;87(11):1017-21. doi: 10.1002/ajh.23306. Epub 2012 Aug 7.
Other Identifiers
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HQP 2008-004
Identifier Type: -
Identifier Source: org_study_id
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