Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease

This study has been completed.
Sponsor:
Information provided by:
HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT00842088
First received: February 11, 2009
Last updated: July 28, 2011
Last verified: July 2011
  Purpose

The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.


Condition Intervention Phase
Sickle Cell Disease
Drug: Placebo
Drug: HQK-1001
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)
Primary Purpose: Treatment
Official Title: A Randomized, Blinded, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 in Subjects With Sickle Cell Disease

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, and (4) physical exam. [ Time Frame: 126 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetics assessed by plasma drug concentration levels. [ Time Frame: Days 0, 6, 69 and 97 post first dose ] [ Designated as safety issue: No ]
  • Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin. [ Time Frame: Every 2 weeks through Day 126 post first dose ] [ Designated as safety issue: No ]

Enrollment: 24
Study Start Date: March 2009
Primary Completion Date: July 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Active Drug: HQK-1001
HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days.
Placebo Comparator: Placebo Drug: Placebo
Matching placebo capsule. Administered orally once a day on dosing days.

  Eligibility

Ages Eligible for Study:   12 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of SCD or sickle beta thalassemia (excluding Hemoglobin C)
  • Between 12 and 60 years of age, inclusive
  • At least one episode of a SCD-related crisis or complication (e.g., vaso-occlusive crisis, acute chest syndrome, priapism) per year for an average of 3 years or one episode of acute chest syndrome over the prior 5 years
  • Screening (untransfused) HbF level >/= 2% as analyzed by a central laboratory
  • If receiving hydroxyurea therapy, must be receiving a stable dose for at least 6 months
  • Able and willing to give informed consent
  • If female, must have a negative serum pregnancy test within 7 days of dosing
  • If female, must not be of childbearing potential defined as post-menopausal by at least 2 years or surgically sterile, or must agree to use a medically accepted form of contraception throughout the study
  • If the sexual partner of a male subject is a WCBP, she must agree to use a medically accepted form of birth control for themselves or their partner throughout the study
  • In the view of the Investigator, able to comply with necessary study procedures

Exclusion Criteria:

  • Red blood cell (RBC) transfusion within 3 months prior to beginning study medication
  • Participation in a regular blood transfusion program
  • More than 4 hospitalizations for acute sickle cell-related events in the previous 12 months
  • An acute vaso-occlusive event within 3 weeks prior to receiving first dose of study medication
  • Pulmonary hypertension requiring oxygen
  • QTc > 450 msec on screening
  • Alanine transaminase (ALT) > 3X upper limit of normal (ULN)
  • Creatinine phosphokinase (CPK) > 20% above the ULN
  • Serum creatinine >1.2 mg/dL
  • An acute illness (e.g., febrile, gastrointestinal [GI], respiratory) within 72 hours prior to receiving first dose of study medication
  • History of syncope, clinically significant dysrhythmias or resuscitation from sudden death
  • Chronic opiate use which, in the view of the Investigator, could confound evaluation of an investigational drug
  • Current abuse of alcohol or drugs
  • Received another investigational agent within 4 weeks, or 5 half-lives, whichever is longer, prior to administration of study medication
  • Currently pregnant or breast feeding a child
  • Known infection with HIV-1
  • Infection with hepatitis B or hepatitis C such that patients are currently on therapy or will be placed on therapy during the trial
  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: NCT00842088

Locations
United States, Alabama
Trialogic Research
Madison, Alabama, United States, 35758
United States, California
Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
United States, Florida
Century Clinical Research, Inc.
Daytona Beach, Florida, United States, 32117
United States, Georgia
Medical College of Georgia
Augusta, Georgia, United States, 30912
United States, Illinois
University of Illinois at Chicago
Chicago, Illinois, United States, 60612
United States, Maryland
Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21205
United States, North Carolina
UNC Comprehensive Sickle Cell Program
Chapel Hill, North Carolina, United States, 27599
United States, Texas
Texas Children's Cancer Center and Hematology Service
Houston, Texas, United States, 77030
Jamaica
University of the West Indies, Mona
Kingston, Mona, Jamaica, 7
Sponsors and Collaborators
HemaQuest Pharmaceuticals Inc.
Investigators
Study Director: Susan Perrine, M.D. HemaQuest Pharmaceuticals
  More Information

No publications provided by HemaQuest Pharmaceuticals Inc.

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Susan Perrine, M.D., HemaQuest Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT00842088     History of Changes
Other Study ID Numbers: HQP 2008-004
Study First Received: February 11, 2009
Last Updated: July 28, 2011
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 20, 2014