Effects of HQK-1001 in Patients With Sickle Cell Disease
Status: | Recruiting |
---|---|
Conditions: | Anemia, Hematology |
Therapuetic Areas: | Hematology |
Healthy: | No |
Age Range: | 12 - 60 |
Updated: | 5/27/2013 |
Start Date: | July 2012 |
Contact: | Richard Ghalie, MD, MBA |
Email: | rghalie@hemaquest.com |
Phone: | 858.356.5583 |
A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease
The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with
sickle cell disease.
Inclusion Criteria:
- Males and females between 12 and 60 years of age
- 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:
- Assigned to a regular transfusion program
- 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
We found this trial at
13
sites
747 52nd St
Oakland, California 94609
Oakland, California 94609
(510) 428-3000
![Children's Hospital and Research Center Oakland](/wp-content/uploads/logos/children-s-hospital-and-research-center-oakland.jpg)
Children's Hospital and Research Center Oakland For nearly 100 years, Children's Hospital & Research Center...
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Tufts Medical Center Tufts Medical Center is an internationally-respected academic medical center – a teaching...
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University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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Georgia Health Sciences University Georgia Regents University, home of the Medical College of Georgia, is...
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New York Methodist Hospital A voluntary, acute-care teaching hospital, New York Methodist Hospital's mission is...
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University of Illinois at Chicago A major research university in the heart of one of...
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University of South Alabama "University of South Alabama is a public institution that was founded...
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