Effects of HQK-1001 in Patients With Sickle Cell Disease

This study has been terminated.
Sponsor:
Information provided by (Responsible Party):
HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT01601340
First received: May 12, 2012
Last updated: November 26, 2013
Last verified: November 2013
  Purpose

The purpose of this study is to evaluate the effects of HQK-1001 on Hb F 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
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease

Resource links provided by NLM:


Further study details as provided by HemaQuest Pharmaceuticals Inc.:

Primary Outcome Measures:
  • Change from baseline in % fetal hemoglobin [ Time Frame: Day 1 through Week 48 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Incidence and number of SCD pain crises and SCD-related complications [ Time Frame: Day 1 through Week 52 ] [ Designated as safety issue: No ]
  • Subject reported daily pain scale scores and analgesic use [ Time Frame: 7 consecutive days following clinic visits at Day 1, and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48 ] [ Designated as safety issue: No ]
  • Change in FACIT Fatigue Scale results [ Time Frame: Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48 ] [ Designated as safety issue: No ]
  • Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments [ Time Frame: Day 1 through Week 52 ] [ Designated as safety issue: Yes ]
  • HQK-1001 pharmacokinetic parameters [ Time Frame: 1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48 ] [ Designated as safety issue: No ]
    A subset of subjects (7) will undergo sampling for detailed analysis of pharmacokinetic parameters (AUC, Cmax) with samples taken pre-dose, and 1, 2, 4, 8, and 10 hours after the morning dose at Week 4.


Enrollment: 77
Study Start Date: July 2012
Estimated Study Completion Date: December 2013
Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: HQK-1001 Drug: HQK-1001
HQK-1001 tablets, twice daily for 48 weeks
Placebo Comparator: Placebo Drug: Placebo
Placebo tablets, twice daily for 48 weeks

  Eligibility

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

Inclusion Criteria:

  • Males and females between 12 and 60 years of age
  • Diagnosis of SCD, type Hb SS or Hb S-B0 Thalassemia
  • At least 1 episode of SCD pain crisis, acute chest syndrome, other acute SCD complications, or leg ulcers in the 12 months prior to screening
  • Not being treated with Hydroxyurea (HU); if HU treatment has been previously administered and then discontinued, at least 3 months must have elapsed since last dose of HU
  • If subject has been transfused in the 3 months prior to screening, then Hb A level < 20% at screening
  • Baseline Hb F level obtained within 14 days prior to randomization
  • Able to swallow tablets
  • Able and willing to give informed consent and/or assent
  • If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 14 days of first dose of HQK-1001 and a negative urine pregnancy test prior to dosing on Day 1
  • If a subject is a WCBP, she must agree to use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation
  • Sexually active male subjects who have not had a vasectomy must agree to use latex condoms with WCBP partners or ensure that their partner(s) use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation.

Exclusion Criteria:

  • Assigned to a regular transfusion program
  • Use of erythropoiesis stimulating agents within 90 days prior to screening
  • An SCD pain crisis or SCD-related acute complication within 3 weeks prior to randomization
  • More than 5 SCD pain crisis or SCD-related acute complications within 12 months prior to screening
  • Pulmonary hypertension requiring therapy
  • ALT or AST > 3x ULN
  • Serum creatinine > 1.5x ULN
  • Serum amylase levels > 1.5x ULN
  • Serum lipase level > 1.5x ULN
  • A serious, concurrent illness that would limit ability to complete or comply with the study requirements
  • 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 due to SCD-related complication
  • Symptomatic peptic ulcer, hiatus hernia, or gastroesophageal reflux disease (GERD)
  • History of pancreatitis
  • Chronic opiate use, which, in the view of the investigator, could confound evaluation of an investigational drug
  • Current abuse of alcohol or drugs
  • Use of 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 subjects are currently on anti-viral therapy or will be placed on therapy
  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: NCT01601340

Locations
United States, Alabama
University of South Alabama
Mobile, Alabama, United States, 36617-2238
United States, California
Children's Hospital and Research Center - Oakland
Oakland, California, United States, 94609
United States, District of Columbia
Children's National Hospital
Washington, District of Columbia, United States, 20010
Howard University Hospital
Washington, District of Columbia, United States, 20060
United States, Georgia
Georgia Health Sciences University
Augusta, Georgia, United States, 30912
United States, Illinois
University of Illinois at Chicago
Chicago, Illinois, United States, 60612
United States, Massachusetts
Tufts Medical Center
Boston, Massachusetts, United States, 02111
United States, New York
The Children's Hospital at Montefiore Medical Center
Bronx, New York, United States, 10467
New York Methodist Hospital
Brooklyn, New York, United States, 11215
United States, North Carolina
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
United States, Virginia
Virginia Commonwealth Univeristy - Center on Health Disparities
Richmond, Virginia, United States, 23298
Canada, Ontario
University Health Network Toronto General Hospital
Toronto, Ontario, Canada, M5G2C4
Egypt
Ain Sham University Hospital
Cairo, Egypt
Abu El Reesh Pediatric University Hospital
Cairo, Egypt
Jamaica
University of the West Indies
Mona, Kingston 7, Jamaica
Lebanon
Chronic Care Center
Beirut, Lebanon
American University of Beirut Medical Center
Beirut, Lebanon
Rafik Hariri University Hospital
Beirut, 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: NCT01601340     History of Changes
Other Study ID Numbers: HQP 1001-SCD-007
Study First Received: May 12, 2012
Last Updated: November 26, 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, Sickle Cell
Disease
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Pathologic Processes

ClinicalTrials.gov processed this record on October 22, 2014