Effects of HQK-1001 in Patients With Sickle Cell Disease

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S

In brief

The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease.

Key facts

Study ID

NCT01601340

Run by

HemaQuest Pharmaceuticals Inc.

People needed

77

Starts

2012-07-01

Expected to finish

2013-12-01

Last updated by the study team

2015-03-18

Who can join

Age: 12 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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

Where it is running

• 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

Full record on ClinicalTrials.gov

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