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

Phase 2

Completed

• Conditions: Sickle Cell Disease; Sickle Cell Anemia; Sickle Cell Disorders; Hemoglobin S Disease; Sickling Disorder Due to Hemoglobin S
• Interventions: Drug: HQK-1001

• Registration Number: NCT01322269
• Lead Sponsor: HemaQuest Pharmaceuticals Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-03-22
• Study First Submitted QC: 2011-03-23
• Study First Posted: 2011-03-24
• Study Start: 2011-04
• Primary Completion: 2012-03
• Status Verified: 2013-06
• Disposition First Submitted: 2013-06-11
• Disposition First Submitted QC: 2013-06-11
• Last Update Submitted: 2013-06-11
• Disposition First Posted: 2013-06-19
• Last Update Posted: 2013-06-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 52

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

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
HQK-1001 (30 mg/kg)	HQK-1001	-
HQK-1001 (40 mg/kg)	HQK-1001	-
HQK-1001 (50 mg/kg)	HQK-1001	-

Primary Outcome Measures

Name	Time	Method
Safety	Day 1 through Week 30	Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.

Secondary Outcome Measures

Name	Time	Method
Fetal hemoglobin levels	Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30
Incidence of sickle cell crisis events	Day 1 through Week 30

Trial Locations

Locations (16)

LSU Health Sciences Center - Feist Weiller Cancer Center; 🇺🇸 Shreveport, Louisiana, United States

Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology; 🇺🇸 Dallas, Texas, United States

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

University of Illinois at Chicago - Dept of Pediatrics; 🇺🇸 Chicago, Illinois, United States

University Health Network Toronto General Hospital; 🇨🇦 Toronto, Ontario, Canada

Rafik Hariri University Hospital; 🇱🇧 Beirut, Lebanon

Chronic Care Center; 🇱🇧 Hazmieh, Lebanon

Children's Hospital and Research Center - Oakland; 🇺🇸 Oakland, California, United States

Georgia Health Sciences University - Adult SIckle Cell Center; 🇺🇸 Augusta, Georgia, United States

Abu El Reesh Pediatric University Hospital; 🇪🇬 Cairo, Egypt

University of the West Indies - Sickle Cell Unit; 🇯🇲 Mona, Kingston, Jamaica

University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program; 🇺🇸 Chapel Hill, North Carolina, United States

Tufts Medical Center; 🇺🇸 Boston, Massachusetts, United States

University of Miami Miller School of Medicine - Dept of Pediatrics; 🇺🇸 Miami, Florida, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

American University of Beirut Medical Center; 🇱🇧 Beirut, Lebanon