Trial record 2 of 356 for:    "Sickle cell anemia"

A Study of HQK-1001 in Patients With Sickle Cell Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT01322269
First received: March 22, 2011
Last updated: June 11, 2013
Last verified: June 2013
  Purpose

The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.


Condition Intervention Phase
Sickle Cell Disease
Sickle Cell Anemia
Sickle Cell Disorders
Hemoglobin S Disease
Sickling Disorder Due to Hemoglobin S
Drug: HQK-1001
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Randomized, Open-Label, Multi-Dose Study 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 [ Time Frame: Day 1 through Week 30 ] [ Designated as safety issue: Yes ]
    Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.


Secondary Outcome Measures:
  • Fetal hemoglobin levels [ Time Frame: Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30 ] [ Designated as safety issue: No ]
  • Incidence of sickle cell crisis events [ Time Frame: Day 1 through Week 30 ] [ Designated as safety issue: No ]

Enrollment: 52
Study Start Date: April 2011
Primary Completion Date: March 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HQK-1001 (30 mg/kg) Drug: HQK-1001
HQK-1001 tablets, once daily for daily 26 weeks
Experimental: HQK-1001 (40 mg/kg) Drug: HQK-1001
HQK-1001 tablets, once daily for daily 26 weeks
Experimental: HQK-1001 (50 mg/kg) Drug: HQK-1001
HQK-1001 tablets, once daily for daily 26 weeks

  Eligibility

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

Inclusion Criteria:

  • Established diagnosis of SCD
  • Males and females between 12 and 60 years of age, inclusive
  • At least 3 episodes of a SCD-related crisis or complication in the 3 years prior to screening OR 1 episode of acute chest syndrome in the 5 years prior to screening
  • If receiving hydroxyurea, must be receiving a stable dose for at least 6 months prior to screening
  • If hydroxyurea treatment has been discontinued, at least 3 months have elapsed since last dose
  • If transfusion in the 4 months prior to screening, then HbA level < 20% at screening
  • Average of the initial two HbF levels ≥ 2.0 % within ≤ 7 days prior to the initial dose of HQK-1001. The two must be obtained ≥ 24 hours apart
  • Ability to swallow tablets
  • Able and willing to give informed consent and assent (if applicable)
  • If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 7 days of first dose of HQK-1001
  • If a subject is a WCBP, she must agree to use an effective form of contraception within 7 days of the initial dose of HQK-1001 and for one month after HQK-1001 discontinuation
  • Sexually active male subjects (with WCBP partners) must agree to use latex condoms or ensure that their partner(s) use an effective form of contraception
  • In the view of the Investigator, subject is able and willing to comply with necessary study procedures

Exclusion Criteria:

  • More than 4 hospitalizations for acute sickle cell related events in the previous 12 months prior to screening
  • Pulmonary hypertension requiring oxygen therapy
  • QTc > 450 msec (male) or 470 msec (female) on screening ECG (QT corrected by Fridericia's formula)
  • Assigned to a regular transfusion program
  • Use of erythropoiesis stimulating agents within 90 days of screening
  • ALT > 3x upper limit of normal (ULN)
  • Serum creatinine > 1.2 mg/dL
  • A serious, concurrent illness that would limit ability to complete or comply with the study requirements
  • An acute vaso-occlusive event within 3 weeks prior to screening
  • Creatine phosphokinase (CK) > 20% above the ULN
  • An acute illness (e.g., febrile, GI, respiratory) within 72 hours prior to screening
  • 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 screening
  • 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: NCT01322269

Locations
United States, California
Children's Hospital and Research Center - Oakland
Oakland, California, United States, 94609
United States, Florida
University of Miami Miller School of Medicine - Dept of Pediatrics
Miami, Florida, United States, 33101
United States, Georgia
Georgia Health Sciences University - Adult SIckle Cell Center
Augusta, Georgia, United States, 30912
United States, Illinois
University of Illinois at Chicago - Dept of Pediatrics
Chicago, Illinois, United States, 60612
United States, Louisiana
LSU Health Sciences Center - Feist Weiller Cancer Center
Shreveport, Louisiana, United States, 71103
United States, Massachusetts
Tufts Medical Center
Boston, Massachusetts, United States, 02111
United States, North Carolina
University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Texas
Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology
Dallas, Texas, United States, 75390-9063
Canada, Ontario
University Health Network Toronto General Hospital
Toronto, Ontario, Canada, MSG 2C4
The Hospital for Sick Children
Toronto, Ontario, Canada, MSG 1X8
Egypt
Abu El Reesh Pediatric University Hospital
Cairo, Egypt
Jamaica
University of the West Indies - Sickle Cell Unit
Mona, Kingston, Jamaica
Lebanon
American University of Beirut Medical Center
Beirut, Lebanon
Rafik Hariri University Hospital
Beirut, Lebanon
Chronic Care Center
Hazmieh, Lebanon
Sponsors and Collaborators
HemaQuest Pharmaceuticals Inc.
Investigators
Study Director: Richard Ghalie, MD, MBA HemaQuest Pharmaceuticals Inc.
  More Information

No publications provided by HemaQuest Pharmaceuticals Inc.

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT01322269     History of Changes
Other Study ID Numbers: HQP 1001-SCD-006
Study First Received: March 22, 2011
Last Updated: June 11, 2013
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board
Jamaica: Ministry of Health
Egypt: Ministry of Health and Population
Egypt: Institutional Review Board
Lebanon: Institutional Review Board
Canada: Health Canada
Canada: Ethics Review Committee

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

ClinicalTrials.gov processed this record on July 29, 2014