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

Sponsor
HemaQuest Pharmaceuticals Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT01322269
Collaborator
(none)
52
Enrollment
16
Locations
3
Arms
3.3
Patients Per Site

Study Details

Study Description

Brief Summary

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 or Disease Intervention/Treatment Phase
Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
52 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease
Study Start Date :
Apr 1, 2011
Actual Primary Completion Date :
Mar 1, 2012

Arms and Interventions

Arm Intervention/Treatment
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

Outcome Measures

Primary Outcome Measures

  1. Safety [Day 1 through Week 30]

    Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.

Secondary Outcome Measures

  1. Fetal hemoglobin levels [Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30]

  2. Incidence of sickle cell crisis events [Day 1 through Week 30]

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years to 60 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Locations

Site City State Country Postal Code
1 Children's Hospital and Research Center - Oakland Oakland California United States 94609
2 University of Miami Miller School of Medicine - Dept of Pediatrics Miami Florida United States 33101
3 Georgia Health Sciences University - Adult SIckle Cell Center Augusta Georgia United States 30912
4 University of Illinois at Chicago - Dept of Pediatrics Chicago Illinois United States 60612
5 LSU Health Sciences Center - Feist Weiller Cancer Center Shreveport Louisiana United States 71103
6 Tufts Medical Center Boston Massachusetts United States 02111
7 University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program Chapel Hill North Carolina United States 27599
8 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
9 Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology Dallas Texas United States 75390-9063
10 The Hospital for Sick Children Toronto Ontario Canada MSG 1X8
11 University Health Network Toronto General Hospital Toronto Ontario Canada MSG 2C4
12 Abu El Reesh Pediatric University Hospital Cairo Egypt
13 University of the West Indies - Sickle Cell Unit Mona Kingston Jamaica
14 American University of Beirut Medical Center Beirut Lebanon
15 Rafik Hariri University Hospital Beirut Lebanon
16 Chronic Care Center Hazmieh Lebanon

Sponsors and Collaborators

  • HemaQuest Pharmaceuticals Inc.

Investigators

  • Study Director: Richard Ghalie, MD, MBA, HemaQuest Pharmaceuticals Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT01322269
Other Study ID Numbers:
  • HQP 1001-SCD-006
First Posted:
Mar 24, 2011
Last Update Posted:
Jun 19, 2013
Last Verified:
Jun 1, 2013
Additional relevant MeSH terms:
Anemia, Sickle Cell
Sickle Cell Trait
Disease

Study Results

No Results Posted as of Jun 19, 2013