Effects of HQK-1001 in Patients With Sickle Cell Disease
NCT ID: NCT01601340
Last Updated: 2015-03-18
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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TERMINATED
PHASE2
77 participants
INTERVENTIONAL
2012-07-31
2013-12-31
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
QUADRUPLE
Study Groups
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HQK-1001
HQK-1001
HQK-1001 tablets, twice daily for 48 weeks
Placebo
Placebo
Placebo tablets, twice daily for 48 weeks
Interventions
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HQK-1001
HQK-1001 tablets, twice daily for 48 weeks
Placebo
Placebo tablets, twice daily for 48 weeks
Eligibility Criteria
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Inclusion Criteria
* Diagnosis of SCD, type Hb SS or Hb S-B0 Thalassemia
* At least 1 episode of SCD pain crisis, acute chest syndrome, other acute SCD complications, or leg ulcers in the 12 months prior to screening
* Not being treated with Hydroxyurea (HU); if HU treatment has been previously administered and then discontinued, at least 3 months must have elapsed since last dose of HU
* If subject has been transfused in the 3 months prior to screening, then Hb A level \< 20% at screening
* Baseline Hb F level obtained within 14 days prior to randomization
* Able to swallow tablets
* Able and willing to give informed consent and/or assent
* If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 14 days of first dose of HQK-1001 and a negative urine pregnancy test prior to dosing on Day 1
* If a subject is a WCBP, she must agree to use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation
* Sexually active male subjects who have not had a vasectomy must agree to use latex condoms with WCBP partners or ensure that their partner(s) use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation.
Exclusion Criteria
* Use of erythropoiesis stimulating agents within 90 days prior to screening
* An SCD pain crisis or SCD-related acute complication within 3 weeks prior to randomization
* More than 5 SCD pain crisis or SCD-related acute complications within 12 months prior to screening
* Pulmonary hypertension requiring therapy
* ALT or AST \> 3x ULN
* Serum creatinine \> 1.5x ULN
* Serum amylase levels \> 1.5x ULN
* Serum lipase level \> 1.5x ULN
* A serious, concurrent illness that would limit ability to complete or comply with the study requirements
* 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 due to SCD-related complication
* Symptomatic peptic ulcer, hiatus hernia, or gastroesophageal reflux disease (GERD)
* History of pancreatitis
* Chronic opiate use, which, in the view of the investigator, could confound evaluation of an investigational drug
* Current abuse of alcohol or drugs
* Use of 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 subjects are currently on anti-viral therapy or will be placed on therapy
12 Years
60 Years
ALL
No
Sponsors
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HemaQuest Pharmaceuticals Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Richard Ghalie, MD, MBA
Role: STUDY_DIRECTOR
HemaQuest Pharmaceuticals Inc.
Locations
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University of South Alabama
Mobile, Alabama, United States
Children's Hospital and Research Center - Oakland
Oakland, California, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Howard University Hospital
Washington D.C., District of Columbia, United States
Georgia Health Sciences University
Augusta, Georgia, United States
University of Illinois at Chicago
Chicago, Illinois, United States
Tufts Medical Center
Boston, Massachusetts, United States
New York Methodist Hospital
Brooklyn, New York, United States
The Children's Hospital at Montefiore Medical Center
The Bronx, New York, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Virginia Commonwealth Univeristy - Center on Health Disparities
Richmond, Virginia, United States
University Health Network Toronto General Hospital
Toronto, Ontario, Canada
Abu El Reesh Pediatric University Hospital
Cairo, , Egypt
Ain Sham University Hospital
Cairo, , Egypt
University of the West Indies
Mona, Kingston 7, , Jamaica
American University of Beirut Medical Center
Beirut, , Lebanon
Chronic Care Center
Beirut, , Lebanon
Rafik Hariri University Hospital
Beirut, , Lebanon
Countries
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References
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Reid ME, El Beshlawy A, Inati A, Kutlar A, Abboud MR, Haynes J Jr, Ward R, Sharon B, Taher AT, Smith W, Manwani D, Ghalie RG. A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2014 Jul;89(7):709-13. doi: 10.1002/ajh.23725. Epub 2014 Apr 15.
Other Identifiers
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HQP 1001-SCD-007
Identifier Type: -
Identifier Source: org_study_id
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