Trial record 16 of 95 for:    "Beta-thalassemia"

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

This study has been completed.
Sponsor:
Information provided by:
HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT00790127
First received: November 11, 2008
Last updated: May 18, 2011
Last verified: May 2011
  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 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by NLM:


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 ]

Enrollment: 21
Study Start Date: March 2009
Study Completion Date: May 2010
Primary Completion Date: May 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Placebo
Drug: Placebo
Matching placebo capsules administered once a day, orally, for 56 days
Experimental: HQK-1001
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 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00790127

Locations
Lebanon
Chronic Care Center
Beirut, Lebanon
Thailand
Siriraj Hospital
Bangkok, Bangkoknoin District, Thailand, 10700
Sponsors and Collaborators
HemaQuest Pharmaceuticals Inc.
Investigators
Principal Investigator: Noppadol Siritanaratkul, MD Siriraj Hospital
  More Information

No publications provided

Responsible Party: Susan Perrine, M.D. Chief Scientific Officer, VP Clinical Affairs, HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT00790127     History of Changes
Other Study ID Numbers: HQP-2008-003b
Study First Received: November 11, 2008
Last Updated: May 18, 2011
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

Additional relevant MeSH terms:
Beta-Thalassemia
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on April 22, 2014