Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease

Completed · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.

Key facts

Study ID

NCT00842088

Run by

HemaQuest Pharmaceuticals Inc.

People needed

24

Starts

2009-03-01

Last updated by the study team

2011-08-01

Who can join

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

You may qualify if…

• Diagnosis of SCD or sickle beta thalassemia (excluding Hemoglobin C)
• Between 12 and 60 years of age, inclusive
• At least one episode of a SCD-related crisis or complication (e.g., vaso-occlusive crisis, acute chest syndrome, priapism) per year for an average of 3 years or one episode of acute chest syndrome over the prior 5 years
• Screening (untransfused) HbF level >/= 2% as analyzed by a central laboratory
• If receiving hydroxyurea therapy, must be receiving a stable dose for at least 6 months
• Able and willing to give informed consent
• If female, must have a negative serum pregnancy test within 7 days of dosing
• If female, must not be of childbearing potential defined as post-menopausal by at least 2 years or surgically sterile, or must agree to use a medically accepted form of contraception throughout the study
• If the sexual partner of a male subject is a WCBP, she must agree to use a medically accepted form of birth control for themselves or their partner throughout the study
• In the view of the Investigator, able to comply with necessary study procedures

You may not qualify if…

• Red blood cell (RBC) transfusion within 3 months prior to beginning study medication
• Participation in a regular blood transfusion program
• More than 4 hospitalizations for acute sickle cell-related events in the previous 12 months
• An acute vaso-occlusive event within 3 weeks prior to receiving first dose of study medication
• Pulmonary hypertension requiring oxygen
• QTc > 450 msec on screening
• Alanine transaminase (ALT) > 3X upper limit of normal (ULN)
• Creatinine phosphokinase (CPK) > 20% above the ULN
• Serum creatinine >1.2 mg/dL
• An acute illness (e.g., febrile, gastrointestinal [GI], respiratory) within 72 hours prior to receiving first dose of study medication
• 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 administration of study medication
• 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

• Trialogic Research — Madison, Alabama, United States
• Children's Hospital and Research Center at Oakland — Oakland, California, United States
• Century Clinical Research, Inc. — Daytona Beach, Florida, United States
• Medical College of Georgia — Augusta, Georgia, United States
• University of Illinois at Chicago — Chicago, Illinois, United States
• Johns Hopkins School of Medicine — Baltimore, Maryland, United States
• UNC Comprehensive Sickle Cell Program — Chapel Hill, North Carolina, United States
• Texas Children's Cancer Center and Hematology Service — Houston, Texas, United States
• University of the West Indies, Mona — Kingston, Mona, Jamaica

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.