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

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Placebo
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:

  • 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

Sites / Locations

  • Trialogic Research
  • Children's Hospital and Research Center at Oakland
  • Century Clinical Research, Inc.
  • Medical College of Georgia
  • University of Illinois at Chicago
  • Johns Hopkins School of Medicine
  • UNC Comprehensive Sickle Cell Program
  • Texas Children's Cancer Center and Hematology Service
  • University of the West Indies, Mona

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Active

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.

Secondary Outcome Measures

Pharmacokinetics assessed by plasma drug concentration levels.
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin.

Full Information

First Posted
February 11, 2009
Last Updated
July 28, 2011
Sponsor
HemaQuest Pharmaceuticals Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT00842088
Brief Title
Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2011
Overall Recruitment Status
Completed
Study Start Date
March 2009 (undefined)
Primary Completion Date
July 2010 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
24 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Active
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Matching placebo capsule. Administered orally once a day on dosing days.
Intervention Type
Drug
Intervention Name(s)
HQK-1001
Intervention Description
HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days.
Primary Outcome Measure Information:
Title
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.
Time Frame
126 days
Secondary Outcome Measure Information:
Title
Pharmacokinetics assessed by plasma drug concentration levels.
Time Frame
Days 0, 6, 69 and 97 post first dose
Title
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin.
Time Frame
Every 2 weeks through Day 126 post first dose

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: 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Susan Perrine, M.D.
Organizational Affiliation
HemaQuest Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Trialogic Research
City
Madison
State/Province
Alabama
ZIP/Postal Code
35758
Country
United States
Facility Name
Children's Hospital and Research Center at Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Century Clinical Research, Inc.
City
Daytona Beach
State/Province
Florida
ZIP/Postal Code
32117
Country
United States
Facility Name
Medical College of Georgia
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
University of Illinois at Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Johns Hopkins School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
UNC Comprehensive Sickle Cell Program
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Texas Children's Cancer Center and Hematology Service
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of the West Indies, Mona
City
Kingston
State/Province
Mona
ZIP/Postal Code
7
Country
Jamaica

12. IPD Sharing Statement

Citations:
PubMed Identifier
22887019
Citation
Kutlar A, Ataga K, Reid M, Vichinsky EP, Neumayr L, Blair-Britt L, Labotka R, Glass J, Keefer JR, Wargin WA, Berenson R, Perrine SP. A phase 1/2 trial of HQK-1001, an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2012 Nov;87(11):1017-21. doi: 10.1002/ajh.23306. Epub 2012 Aug 7.
Results Reference
derived

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

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