Beta Thalassemia
Conditions
Brief summary
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia
Interventions
DRUGHQK-1001
HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days
DRUGPlacebo
Matching placebo capsules administered once a day, orally, for 56 days
Sponsors
HemaQuest Pharmaceuticals Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* Clinical diagnosis of beta thalassemia intermedia or hemoglobin E beta thalassemia * Average of the initial two hemoglobin levels of less than 10 g/dL within ≥ 30 days prior to randomization. * Age ≥ 12 and ≤ 60 years * Able and willing to give informed consent or assent (if less than 18 years of age) and comply with all study procedures * If female and of childbearing potential, must have a documented negative pregnancy test at Day -1 and must agree to use one or more locally medically accepted methods of contraception in the month prior to randomization, through the entire study, and for 4 weeks after the last dosing of study medication
Exclusion criteria
* Spleen palpable ≥ 2 cm below the left costal margin * Pulmonary hypertension requiring oxygen therapy * QTc \> 450 msec on screening ECG * Infection with hepatitis C, hepatitis B requiring therapy * Known infection with HIV * Red blood cell (RBC) transfusions within 2 months prior to administration of study medication with stable hemoglobin levels * Fever greater than 38.5°C in the week prior to administration of study medication * ALT \> 4x upper limit of normal (ULN) * Baseline elevation of CPK value prior to randomization * Treatment with hydroxyurea within 2 months prior to administration of study medication * Cancer diagnosis within 5 years of randomization (except for non-melanoma cutaneous malignancies) * Serum creatinine \> 1.5 mg/dl * Received investigational systemic therapy within 30 days prior to randomization * Currently pregnant or breast feeding a child * Subject history of clinically significant arrhythmias or syncope * Known current drug or alcohol abuse
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam | 168 days |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetics assessed by plasma drug concentration levels | 140 days |
| Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin | 168 days |
Countries
Lebanon, Thailand
Outcome results
None listed