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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

ClinicalTrials.gov processed this record on October 30, 2014