Status:
COMPLETED
Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
Lead Sponsor:
HemaQuest Pharmaceuticals Inc.
Conditions:
Beta Thalassemia
Eligibility:
All Genders
12-60 years
Phase:
PHASE1
PHASE2
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
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
March 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2010
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT00790127
Start Date
March 1 2009
End Date
May 1 2010
Last Update
May 20 2011
Active Locations (2)
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1
Chronic Care Center
Beirut, Lebanon
2
Siriraj Hospital
Bangkok, Bangkoknoin District, Thailand, 10700