A Randomized, Blinded, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 in Subjects With Sickle Cell Disease

HemaQuest Pharmaceuticals Inc.9 sites in 2 countries24 target enrollmentMarch 2009

ConditionsSickle Cell Disease

InterventionsHQK-1001 Placebo

DrugsPlacebo HQK-1001

Overview

Phase: Phase 1
Intervention: HQK-1001
Conditions: Sickle Cell Disease
Sponsor: HemaQuest Pharmaceuticals Inc.
Enrollment: 24
Locations: 9
Primary Endpoint: Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.
Status: Completed
Last Updated: 14 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.

Registry

clinicaltrials.gov

Start Date

March 2009

End Date

July 2010

Last Updated

14 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

HemaQuest Pharmaceuticals Inc.

Eligibility Criteria

Inclusion Criteria

•Diagnosis of SCD or sickle beta thalassemia (excluding Hemoglobin C)
•Between 12 and 60 years of age, inclusive
•At least one episode of a SCD-related crisis or complication (e.g., vaso-occlusive crisis, acute chest syndrome, priapism) per year for an average of 3 years or one episode of acute chest syndrome over the prior 5 years
•Screening (untransfused) HbF level \>/= 2% as analyzed by a central laboratory
•If receiving hydroxyurea therapy, must be receiving a stable dose for at least 6 months
•Able and willing to give informed consent
•If female, must have a negative serum pregnancy test within 7 days of dosing
•If female, must not be of childbearing potential defined as post-menopausal by at least 2 years or surgically sterile, or must agree to use a medically accepted form of contraception throughout the study
•If the sexual partner of a male subject is a WCBP, she must agree to use a medically accepted form of birth control for themselves or their partner throughout the study
•In the view of the Investigator, able to comply with necessary study procedures

Exclusion Criteria

•Red blood cell (RBC) transfusion within 3 months prior to beginning study medication
•Participation in a regular blood transfusion program
•More than 4 hospitalizations for acute sickle cell-related events in the previous 12 months
•An acute vaso-occlusive event within 3 weeks prior to receiving first dose of study medication
•Pulmonary hypertension requiring oxygen
•QTc \> 450 msec on screening
•Alanine transaminase (ALT) \> 3X upper limit of normal (ULN)
•Creatinine phosphokinase (CPK) \> 20% above the ULN
•Serum creatinine \>1.2 mg/dL
•An acute illness (e.g., febrile, gastrointestinal \[GI\], respiratory) within 72 hours prior to receiving first dose of study medication

Arms & Interventions

Active

Intervention: HQK-1001

Placebo

Intervention: Placebo

Outcomes

Primary Outcomes

Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.

Time Frame: 126 days

Secondary Outcomes

Pharmacokinetics assessed by plasma drug concentration levels.(Days 0, 6, 69 and 97 post first dose)
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin.(Every 2 weeks through Day 126 post first dose)