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.
Brief Title
A Study of HQK-1001 in Patients With Sickle Cell Disease
Categories
Conditions
Sickle Cell Disease
Sickle Cell Anemia
Sickle Cell Disorders
Hemoglobin S Disease
Sickling Disorder Due to Hemoglobin S
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
* 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
Inclusion 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
* 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
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Estimated
Last Update Submit Date
Maximum Age
60 Years
Minimum Age
12 Years
NCT Id
NCT01322269
Org Class
Industry
Org Full Name
HemaQuest Pharmaceuticals Inc.
Org Study Id
HQP 1001-SCD-006
Overall Status
Completed
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease
Primary Outcomes
Outcome Description
Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.
Outcome Measure
Safety
Outcome Time Frame
Day 1 through Week 30
Secondary Outcomes
Outcome Time Frame
Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30
Outcome Measure
Fetal hemoglobin levels
Outcome Time Frame
Day 1 through Week 30
Outcome Measure
Incidence of sickle cell crisis events
Start Date
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Maximum Age Number (converted to Years and rounded down)
60
Minimum Age Number (converted to Years and rounded down)
12
Investigators
Investigator Type
Principal Investigator
Investigator Name
Deepa Manwani
Investigator Email
dmanwani@montefiore.org
Investigator Phone
718-741-2342