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

Primary Purpose

Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
HQK-1001
Sponsored by
HemaQuest Pharmaceuticals Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease

Eligibility Criteria

12 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Hospital and Research Center - Oakland
  • University of Miami Miller School of Medicine - Dept of Pediatrics
  • Georgia Health Sciences University - Adult SIckle Cell Center
  • University of Illinois at Chicago - Dept of Pediatrics
  • LSU Health Sciences Center - Feist Weiller Cancer Center
  • Tufts Medical Center
  • University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program
  • Cincinnati Children's Hospital Medical Center
  • Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology
  • The Hospital for Sick Children
  • University Health Network Toronto General Hospital
  • Abu El Reesh Pediatric University Hospital
  • University of the West Indies - Sickle Cell Unit
  • American University of Beirut Medical Center
  • Rafik Hariri University Hospital
  • Chronic Care Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

HQK-1001 (30 mg/kg)

HQK-1001 (40 mg/kg)

HQK-1001 (50 mg/kg)

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Fetal hemoglobin levels
Incidence of sickle cell crisis events

Full Information

First Posted
March 22, 2011
Last Updated
June 11, 2013
Sponsor
HemaQuest Pharmaceuticals Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01322269
Brief Title
A Study of HQK-1001 in Patients With Sickle Cell Disease
Official Title
A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease
Study Type
Interventional

2. Study Status

Record Verification Date
June 2013
Overall Recruitment Status
Completed
Study Start Date
April 2011 (undefined)
Primary Completion Date
March 2012 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
HemaQuest Pharmaceuticals Inc.

4. Oversight

Data Monitoring Committee
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
52 (Actual)

8. Arms, Groups, and Interventions

Arm Title
HQK-1001 (30 mg/kg)
Arm Type
Experimental
Arm Title
HQK-1001 (40 mg/kg)
Arm Type
Experimental
Arm Title
HQK-1001 (50 mg/kg)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
HQK-1001
Intervention Description
HQK-1001 tablets, once daily for daily 26 weeks
Primary Outcome Measure Information:
Title
Safety
Description
Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.
Time Frame
Day 1 through Week 30
Secondary Outcome Measure Information:
Title
Fetal hemoglobin levels
Time Frame
Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30
Title
Incidence of sickle cell crisis events
Time Frame
Day 1 through Week 30

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Richard Ghalie, MD, MBA
Organizational Affiliation
HemaQuest Pharmaceuticals Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital and Research Center - Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
University of Miami Miller School of Medicine - Dept of Pediatrics
City
Miami
State/Province
Florida
ZIP/Postal Code
33101
Country
United States
Facility Name
Georgia Health Sciences University - Adult SIckle Cell Center
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
University of Illinois at Chicago - Dept of Pediatrics
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
LSU Health Sciences Center - Feist Weiller Cancer Center
City
Shreveport
State/Province
Louisiana
ZIP/Postal Code
71103
Country
United States
Facility Name
Tufts Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
Facility Name
University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390-9063
Country
United States
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
MSG 1X8
Country
Canada
Facility Name
University Health Network Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
MSG 2C4
Country
Canada
Facility Name
Abu El Reesh Pediatric University Hospital
City
Cairo
Country
Egypt
Facility Name
University of the West Indies - Sickle Cell Unit
City
Mona
State/Province
Kingston
Country
Jamaica
Facility Name
American University of Beirut Medical Center
City
Beirut
Country
Lebanon
Facility Name
Rafik Hariri University Hospital
City
Beirut
Country
Lebanon
Facility Name
Chronic Care Center
City
Hazmieh
Country
Lebanon

12. IPD Sharing Statement

Citations:
PubMed Identifier
23828223
Citation
Kutlar A, Reid ME, Inati A, Taher AT, Abboud MR, El-Beshlawy A, Buchanan GR, Smith H, Ataga KI, Perrine SP, Ghalie RG. A dose-escalation phase IIa study of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2013 Nov;88(11):E255-60. doi: 10.1002/ajh.23533. Epub 2013 Oct 3.
Results Reference
derived

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

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