Clinical trial NCT00790127: Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Beta Thalassemia
Lead sponsor: HemaQuest Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2008-11-13
Enrollment: 21
Sex: ALL
Minimum age (years): 12
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 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

Primary outcomes:
measure: Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam
timeFrame: 168 days
Secondary outcomes:
measure: Pharmacokinetics assessed by plasma drug concentration levels
timeFrame: 140 days
measure: Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin
timeFrame: 168 days
Other outcomes:
Locations:
city: Beirut
country: Lebanon
facility: Chronic Care Center
city: Bangkok
country: Thailand
facility: Siriraj Hospital
state: Bangkoknoin District
Study officials:
affiliation: Siriraj Hospital
name: Noppadol Siritanaratkul, MD
role: PRINCIPAL_INVESTIGATOR
References: