A Study of HQK-1001 in Patients With Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:

A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01322269
• Other study ID #: HQP 1001-SCD-006
• Status: Completed
• Phase: Phase 2
• First received: March 22, 2011
• Last updated: June 11, 2013
• Start date: April 2011

Study information

• Verified date: June 2013
• Source: HemaQuest Pharmaceuticals Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review BoardJamaica: Ministry of HealthEgypt: Ministry of Health and PopulationEgypt: Institutional Review BoardLebanon: Institutional Review BoardCanada: Health CanadaCanada: Ethics Review Committee
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. primary completion date: March 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years to 60 Years

Eligibility

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

Study Design

Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Disease
• Hemoglobin S Disease
• Sickle Cell Anemia
• Sickle Cell Disease
• Sickle Cell Disorders
• Sickling Disorder Due to Hemoglobin S

Intervention

Drug:
• HQK-1001
HQK-1001 tablets, once daily for daily 26 weeks

Locations

Country	Name	City	State
Canada	The Hospital for Sick Children	Toronto	Ontario
Canada	University Health Network Toronto General Hospital	Toronto	Ontario
Egypt	Abu El Reesh Pediatric University Hospital	Cairo
Jamaica	University of the West Indies - Sickle Cell Unit	Mona	Kingston
Lebanon	American University of Beirut Medical Center	Beirut
Lebanon	Rafik Hariri University Hospital	Beirut
Lebanon	Chronic Care Center	Hazmieh
United States	Georgia Health Sciences University - Adult SIckle Cell Center	Augusta	Georgia
United States	Tufts Medical Center	Boston	Massachusetts
United States	University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program	Chapel Hill	North Carolina
United States	University of Illinois at Chicago - Dept of Pediatrics	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology	Dallas	Texas
United States	University of Miami Miller School of Medicine - Dept of Pediatrics	Miami	Florida
United States	Children's Hospital and Research Center - Oakland	Oakland	California
United States	LSU Health Sciences Center - Feist Weiller Cancer Center	Shreveport	Louisiana

Sponsors (1)

Lead Sponsor	Collaborator
HemaQuest Pharmaceuticals Inc.

Countries where clinical trial is conducted

United States,  Canada,  Egypt,  Jamaica,  Lebanon, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety	Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.	Day 1 through Week 30	Yes
Secondary	Fetal hemoglobin levels		Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30	No
Secondary	Incidence of sickle cell crisis events		Day 1 through Week 30	No