Effects of HQK-1001 in Patients With Sickle Cell Disease
Effects of HQK-1001 in Patients With 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