Clinical trial NCT01601340 (phase 2, status: terminated)
Conditions: sickle cell disease, sickle cell anemia, sickle cell disorders, hemoglobin s disease, sickling disorder due to hemoglobin s
ICD codes: D57.1, D57.20, D57.212, D57.219, D57.211, D57.213, D57.218
Drugs: hqk-1001, placebo
SMILES: CN1C(=O)C=C(N2CCC[C@@H](N)C2)N(CC2=C(C=CC=C2)C#N)C1=O
Eligibility criteria:

        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