A Study of HQK-1001 in Patients With Sickle Cell Disease
A Study of HQK-1001 in Patients With Sickle Cell Disease
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.
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