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

• Status: Completed
• Trial end date: 2010-05-01
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: HemaQuest Pharmaceuticals Inc.

Criteria

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