Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia

A Multi-National, Blinded, Placebo-Controlled, Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HQK-1001 in Subjects With Beta Thalassemia Intermedia, Including Hemoglobin E Beta Thalassemia

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

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