Sickle Cell Disease
Conditions
Brief summary
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.
Interventions
DRUGPlacebo
Matching placebo capsule. Administered orally once a day on dosing days.
DRUGHQK-1001
HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days.
Sponsors
HemaQuest Pharmaceuticals Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
12 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam. | 126 days |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetics assessed by plasma drug concentration levels. | Days 0, 6, 69 and 97 post first dose |
| Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin. | Every 2 weeks through Day 126 post first dose |
Countries
Jamaica, United States
Outcome results
None listed