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

Phase 1

Completed

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

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo
HQK-1001	HQK-1001	HQK-1001

Primary Outcome Measures

Name	Time	Method
Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam	168 days

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetics assessed by plasma drug concentration levels	140 days
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin	168 days