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Effects of HQK-1001 in Patients With Sickle Cell Disease

Sponsor
HemaQuest Pharmaceuticals Inc. (Industry)
Overall Status
Terminated
CT.gov ID
NCT01601340
Collaborator
(none)
77
Enrollment
18
Locations
2
Arms
17
Duration (Months)
4.3
Patients Per Site
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
77 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease
Study Start Date :
Jul 1, 2012
Actual Primary Completion Date :
Nov 1, 2013
Actual Study Completion Date :
Dec 1, 2013

Arms and Interventions

Arm Intervention/Treatment
Active Comparator: HQK-1001

Drug: HQK-1001
HQK-1001 tablets, twice daily for 48 weeks
Placebo Comparator: Placebo

Drug: Placebo
Placebo tablets, twice daily for 48 weeks

Outcome Measures

Primary Outcome Measures

  1. Change from baseline in % fetal hemoglobin [Day 1 through Week 48]

Secondary Outcome Measures

  1. Incidence and number of SCD pain crises and SCD-related complications [Day 1 through Week 52]

  2. Subject reported daily pain scale scores and analgesic use [7 consecutive days following clinic visits at Day 1, and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48]

  3. Change in FACIT Fatigue Scale results [Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48]

  4. Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments [Day 1 through Week 52]

  5. HQK-1001 pharmacokinetic parameters [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.

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years to 60 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 University of South Alabama Mobile Alabama United States 36617-2238
2 Children's Hospital and Research Center - Oakland Oakland California United States 94609
3 Children's National Hospital Washington District of Columbia United States 20010
4 Howard University Hospital Washington District of Columbia United States 20060
5 Georgia Health Sciences University Augusta Georgia United States 30912
6 University of Illinois at Chicago Chicago Illinois United States 60612
7 Tufts Medical Center Boston Massachusetts United States 02111
8 The Children's Hospital at Montefiore Medical Center Bronx New York United States 10467
9 New York Methodist Hospital Brooklyn New York United States 11215
10 University of North Carolina at Chapel Hill Chapel Hill North Carolina United States 27599
11 Virginia Commonwealth Univeristy - Center on Health Disparities Richmond Virginia United States 23298
12 University Health Network Toronto General Hospital Toronto Ontario Canada M5G2C4
13 Abu El Reesh Pediatric University Hospital Cairo Egypt
14 Ain Sham University Hospital Cairo Egypt
15 University of the West Indies Mona, Kingston 7 Jamaica
16 American University of Beirut Medical Center Beirut Lebanon
17 Chronic Care Center Beirut Lebanon
18 Rafik Hariri University Hospital Beirut Lebanon

Sponsors and Collaborators

  • HemaQuest Pharmaceuticals Inc.

Investigators

  • Study Director: Richard Ghalie, MD, MBA, HemaQuest Pharmaceuticals Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT01601340
Other Study ID Numbers:
  • HQP 1001-SCD-007
First Posted:
May 18, 2012
Last Update Posted:
Mar 18, 2015
Last Verified:
Mar 1, 2015
Additional relevant MeSH terms:
Anemia, Sickle Cell
Sickle Cell Trait
Disease

Study Results

No Results Posted as of Mar 18, 2015