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Phase 2 Study of HQK-1001 in Patients With Beta Thalassemia
This study is not yet open for participant recruitment.
Verified by HemaQuest Pharmaceuticals Inc., November 2008
Sponsored by: HemaQuest Pharmaceuticals Inc.
Information provided by: HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT00790127
  Purpose

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


Condition Intervention Phase
Beta Thalassemia
Drug: HQK-1001
Drug: Placebo
Phase II

Genetics Home Reference related topics: beta thalassemia
MedlinePlus related topics: Thalassemia
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title: A Multi-National, Double Blind, 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

Further study details as provided by HemaQuest Pharmaceuticals Inc.:

Primary Outcome Measures:
  • Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam [ Time Frame: 168 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetics assessed by plasma drug concentration levels [ Time Frame: 140 days ] [ Designated as safety issue: No ]
  • Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin [ Time Frame: 168 days ] [ Designated as safety issue: No ]

Estimated Enrollment: 56
Study Start Date: December 2008
Estimated Study Completion Date: November 2009
Estimated Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo: Placebo Comparator
Placebo
Drug: Placebo
Matching placebo capsules administered once a day, orally, for 56 days
HQK-1001: Experimental
HQK-1001
Drug: HQK-1001
HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days

  Eligibility

Ages Eligible for Study:   12 Years to 45 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinical diagnosis of beta thalassemia intermedia or hemoglobin E beta thalassemia
  • Steady-state hemoglobin level less than 10 g/dL
  • Age ≥ 12 and ≤ 45 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
  • If male and partner is of childbearing potential, must agree to use one or more locally medically accepted methods of birth control for themselves and their partner 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
  • Diagnosis of hepatitis C, hepatitis B or HIV and receiving treatment within 1 month prior to study drug administration
  • Red blood cell (RBC) transfusions within 3 months prior to administration of study medication
  • Fever greater than 38.5°C in the week prior to administration of study medication
  • ALT > 3x 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 6 weeks prior to randomization
  • Currently pregnant or breast feeding a child
  • Sexually active without contraception
  • Subject history of wide complex tachyarrhythmias or resuscitation from sudden death
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00790127

Contacts
Contact: Sally Case 617-750-5943 scase@hemaquest.com

Locations
United States, California
Children's Hospital & Research Center
Oakland, California, United States, 94609
Lebanon
Chronic Care Center
Beirut, Lebanon
Thailand, Bangkoknoin District
Siriraj Hospital
Bangkok, Bangkoknoin District, Thailand, 10700
Sponsors and Collaborators
HemaQuest Pharmaceuticals Inc.
  More Information

Responsible Party: HemaQuest Pharmaceuticals Inc. ( Susan Perrine, M.D. Chief Scientific Officer, VP Clinical Affairs )
Study ID Numbers: HQP-2008-003b
Study First Received: November 11, 2008
Last Updated: November 12, 2008
ClinicalTrials.gov Identifier: NCT00790127  
Health Authority: United States: Food and Drug Administration;   Thailand: Food and Drug Administration;   Thailand: Ethical Committee;   Lebanon: Ministry of Public Health;   Lebanon: Institutional Review Board

Study placed in the following topic categories:
Anemia, Hemolytic, Congenital
Thalassemia minor
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Beta-Thalassemia
Beta-thalassemia
Anemia
Anemia, Hemolytic
Hemoglobinopathy
Thalassemia

ClinicalTrials.gov processed this record on January 16, 2009