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

Completed · Phase 2

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 safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.

Key facts

Study ID

NCT01322269

Run by

HemaQuest Pharmaceuticals Inc.

People needed

52

Starts

2011-04-01

Last updated by the study team

2013-06-19

Who can join

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

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Children's Hospital and Research Center - Oakland — Oakland, California, United States
• University of Miami Miller School of Medicine - Dept of Pediatrics — Miami, Florida, United States
• Georgia Health Sciences University - Adult SIckle Cell Center — Augusta, Georgia, United States
• University of Illinois at Chicago - Dept of Pediatrics — Chicago, Illinois, United States
• LSU Health Sciences Center - Feist Weiller Cancer Center — Shreveport, Louisiana, United States
• Tufts Medical Center — Boston, Massachusetts, United States
• University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program — Chapel Hill, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology — Dallas, Texas, United States
• The Hospital for Sick Children — Toronto, Ontario, Canada
• University Health Network Toronto General Hospital — Toronto, Ontario, Canada
• Abu El Reesh Pediatric University Hospital — Cairo, Egypt
• University of the West Indies - Sickle Cell Unit — Mona, Kingston, Jamaica
• American University of Beirut Medical Center — Beirut, Lebanon
• Rafik Hariri University Hospital — Beirut, Lebanon
• Chronic Care Center — Hazmiyeh, Lebanon

Full record on ClinicalTrials.gov

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