Effects of HQK-1001 in Patients With Sickle Cell Disease

March 17, 2015 updated by: HemaQuest Pharmaceuticals Inc.

A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease

The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
77

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G2C4
        • University Health Network Toronto General Hospital
  • Egypt
      • Cairo, Egypt
        • Abu El Reesh pediatric university hospital
      • Cairo, Egypt
        • Ain Sham University Hospital
  • Jamaica
      • Mona, Kingston 7, Jamaica
        • University of the West Indies
  • Lebanon
      • Beirut, Lebanon
        • American University of Beirut Medical Center
      • Beirut, Lebanon
        • Rafik Hariri University Hospital
      • Beirut, Lebanon
        • Chronic Care Center
  • United States
    • Alabama
      • Mobile, Alabama, United States, 36617-2238
        • University Of South Alabama
    • California
      • Oakland, California, United States, 94609
        • Children's Hospital and Research Center - Oakland
    • District of Columbia
      • Washington, District of Columbia, United States, 20060
        • Howard University Hospital
      • Washington, District of Columbia, United States, 20010
        • Children's National Hospital
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Georgia Health Sciences University
    • Illinois
      • Chicago, Illinois, United States, 60612
        • University of Illinois at Chicago
    • Massachusetts
      • Boston, Massachusetts, United States, 02111
        • Tufts Medical Center
    • New York
      • Bronx, New York, United States, 10467
        • The Children's Hospital at Montefiore Medical Center
      • Brooklyn, New York, United States, 11215
        • New York Methodist Hospital
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • University of North Carolina at Chapel Hill
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth Univeristy - Center on Health Disparities

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years to 60 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: QUADRUPLE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
PLACEBO_COMPARATOR: Placebo
Drug: Placebo
Placebo tablets, twice daily for 48 weeks
ACTIVE_COMPARATOR: HQK-1001
Drug: HQK-1001
HQK-1001 tablets, twice daily for 48 weeks

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change from baseline in % fetal hemoglobin
Time Frame: Day 1 through Week 48
Day 1 through Week 48

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Incidence and number of SCD pain crises and SCD-related complications
Time Frame: Day 1 through Week 52
Day 1 through Week 52
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
7 consecutive days following clinic visits at Day 1, and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48
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
Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48
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
Day 1 through Week 52
HQK-1001 pharmacokinetic parameters
Time Frame: 1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48
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.
1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
HemaQuest Pharmaceuticals Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2012

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 1, 2013

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2013

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 12, 2012

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 17, 2012

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 18, 2012

Study Record Updates

Last Update Posted (ESTIMATE)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 18, 2015

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 17, 2015

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2015

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • HQP 1001-SCD-007

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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