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Trial record 15 of 28 for:    sickle cell | "Sickle Cell Trait"

Effects of HQK-1001 in Patients With Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT01601340
Recruitment Status : Terminated
First Posted : May 18, 2012
Last Update Posted : March 18, 2015
Sponsor:
Information provided by (Responsible Party):
HemaQuest Pharmaceuticals Inc.

Tracking Information
First Submitted Date  ICMJE May 12, 2012
First Posted Date  ICMJE May 18, 2012
Last Update Posted Date March 18, 2015
Study Start Date  ICMJE July 2012
Actual Primary Completion Date November 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 17, 2012)
Change from baseline in % fetal hemoglobin [ Time Frame: Day 1 through Week 48 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01601340 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 18, 2012)
  • Incidence and number of SCD pain crises and SCD-related complications [ Time Frame: 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 ]
  • 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 ]
  • 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 ]
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: May 17, 2012)
  • Incidence and number of SCD pain crises and SCD-related complications [ Time Frame: 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 ]
  • 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 ]
  • 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 ]
  • 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 with samples taken pre-dose, and 1, 2, 4, 8, and 10 hours after the morning dose at Week 4.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effects of HQK-1001 in Patients With Sickle Cell Disease
Official Title  ICMJE A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease
Brief Summary The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Sickle Cell Disease
  • Sickle Cell Anemia
  • Sickle Cell Disorders
  • Hemoglobin S Disease
  • Sickling Disorder Due to Hemoglobin S
Intervention  ICMJE
  • Drug: HQK-1001
    HQK-1001 tablets, twice daily for 48 weeks
  • Drug: Placebo
    Placebo tablets, twice daily for 48 weeks
Study Arms  ICMJE
  • Active Comparator: HQK-1001
    Intervention: Drug: HQK-1001
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
Publications * Reid ME, El Beshlawy A, Inati A, Kutlar A, Abboud MR, Haynes J Jr, Ward R, Sharon B, Taher AT, Smith W, Manwani D, Ghalie RG. A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2014 Jul;89(7):709-13. doi: 10.1002/ajh.23725. Epub 2014 Apr 15.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: November 26, 2013)
77
Original Estimated Enrollment  ICMJE
 (submitted: May 17, 2012)
74
Actual Study Completion Date  ICMJE December 2013
Actual Primary Completion Date November 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 60 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Egypt,   Jamaica,   Lebanon,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01601340
Other Study ID Numbers  ICMJE HQP 1001-SCD-007
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party HemaQuest Pharmaceuticals Inc.
Study Sponsor  ICMJE HemaQuest Pharmaceuticals Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Richard Ghalie, MD, MBA HemaQuest Pharmaceuticals Inc.
PRS Account HemaQuest Pharmaceuticals Inc.
Verification Date March 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP