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

HemaQuest Pharmaceuticals Inc.16 sites in 5 countries52 target enrollmentApril 2011

ConditionsSickle Cell Disease Sickle Cell Anemia Sickle Cell Disorders Hemoglobin S Disease Sickling Disorder Due to Hemoglobin S

InterventionsHQK-1001

DrugsHQK-1001

Overview

Phase: Phase 2
Intervention: HQK-1001
Conditions: Sickle Cell Disease
Sponsor: HemaQuest Pharmaceuticals Inc.
Enrollment: 52
Locations: 16
Primary Endpoint: Safety
Status: Completed
Last Updated: 12 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

April 2011

End Date

March 2012

Last Updated

12 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

HemaQuest Pharmaceuticals Inc.

Responsible Party

Sponsor

Eligibility Criteria

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-
•The two must be obtained ≥ 24 hours apart
•Ability to swallow tablets
•Able and willing to give informed consent and assent (if applicable)

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