Brief Summary
The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease.
Brief Title
Effects of HQK-1001 in Patients With Sickle Cell Disease
Categories
Completion Date
Completion Date Type
Actual
Conditions
Sickle Cell Disease
Sickle Cell Anemia
Sickle Cell Disorders
Hemoglobin S Disease
Sickling Disorder Due to Hemoglobin S
Eligibility Criteria
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
* 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
Inclusion Criteria
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.
* 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.
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
NCT01601340
Org Class
Industry
Org Full Name
HemaQuest Pharmaceuticals Inc.
Org Study Id
HQP 1001-SCD-007
Overall Status
Terminated
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease
Primary Outcomes
Outcome Measure
Change from baseline in % fetal hemoglobin
Outcome Time Frame
Day 1 through Week 48
Secondary Outcomes
Outcome Time Frame
Day 1 through Week 52
Outcome Measure
Incidence and number of SCD pain crises and SCD-related complications
Outcome 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
Outcome Measure
Subject reported daily pain scale scores and analgesic use
Outcome Time Frame
Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48
Outcome Measure
Change in FACIT Fatigue Scale results
Outcome Time Frame
Day 1 through Week 52
Outcome Measure
Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments
Outcome Description
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.
Outcome Time Frame
1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48
Outcome Measure
HQK-1001 pharmacokinetic parameters
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