Clincosm LogoSign in Get a demo

Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease

Sponsor
HemaQuest Pharmaceuticals Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT00842088
Collaborator
(none)
24
Enrollment
9
Locations
2
Arms
2.7
Patients Per Site

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 1/Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
24 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Triple (Participant, Care Provider, Investigator)
Primary Purpose:
Treatment
Official Title:
A Randomized, Blinded, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 in Subjects With Sickle Cell Disease
Study Start Date :
Mar 1, 2009
Actual Primary Completion Date :
Jul 1, 2010

Arms and Interventions

Arm Intervention/Treatment
Experimental: Active

Drug: HQK-1001
HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days.
Placebo Comparator: Placebo

Drug: Placebo
Matching placebo capsule. Administered orally once a day on dosing days.

Outcome Measures

Primary Outcome Measures

  1. Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam. [126 days]

Secondary Outcome Measures

  1. Pharmacokinetics assessed by plasma drug concentration levels. [Days 0, 6, 69 and 97 post first dose]

  2. Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin. [Every 2 weeks through Day 126 post first dose]

Eligibility Criteria

Criteria

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

  • 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 administration of study medication

  • 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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Trialogic Research Madison Alabama United States 35758
2 Children's Hospital and Research Center at Oakland Oakland California United States 94609
3 Century Clinical Research, Inc. Daytona Beach Florida United States 32117
4 Medical College of Georgia Augusta Georgia United States 30912
5 University of Illinois at Chicago Chicago Illinois United States 60612
6 Johns Hopkins School of Medicine Baltimore Maryland United States 21205
7 UNC Comprehensive Sickle Cell Program Chapel Hill North Carolina United States 27599
8 Texas Children's Cancer Center and Hematology Service Houston Texas United States 77030
9 University of the West Indies, Mona Kingston Mona Jamaica 7

Sponsors and Collaborators

  • HemaQuest Pharmaceuticals Inc.

Investigators

  • Study Director: Susan Perrine, M.D., HemaQuest Pharmaceuticals

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
, ,
ClinicalTrials.gov Identifier:
NCT00842088
Other Study ID Numbers:
  • HQP 2008-004
First Posted:
Feb 12, 2009
Last Update Posted:
Aug 1, 2011
Last Verified:
Jul 1, 2011
Additional relevant MeSH terms:
Anemia, Sickle Cell

Study Results

No Results Posted as of Aug 1, 2011