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

HemaQuest Pharmaceuticals

Status and phase

Completed

Phase 2

Phase 1

Conditions

Beta Thalassemia

Treatments

Drug: Placebo

Drug: HQK-1001

Study type

Interventional

Funder types

Industry

Identifiers

NCT00790127

HQP-2008-003b

Details and patient eligibility

About

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

Enrollment

21 patients

Sex

All

Ages

12 to 60 years old

Volunteers

No Healthy Volunteers

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