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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT00790127 |
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Date of registration:
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11/11/2008 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
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Scientific title:
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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 |
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Date of first enrolment:
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March 2009 |
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Target sample size:
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21 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00790127 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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Lebanon
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Thailand
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United States
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Contacts
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Name:
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Noppadol Siritanaratkul, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Siriraj Hospital |
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Key inclusion & exclusion criteria
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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
Age minimum:
12 Years
Age maximum:
60 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Beta Thalassemia
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Intervention(s)
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Drug: HQK-1001
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Drug: Placebo
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Primary Outcome(s)
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Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam
[Time Frame: 168 days]
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Secondary Outcome(s)
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Pharmacokinetics assessed by plasma drug concentration levels
[Time Frame: 140 days]
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Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin
[Time Frame: 168 days]
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Secondary ID(s)
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HQP-2008-003b
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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