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


Phase 1/Phase 2
12 Years
60 Years
Not Enrolling
Both
Sickle Cell Disease

Thank you

Trial Information

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


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

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Outcome Measure:

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

Outcome Time Frame:

126 days

Safety Issue:

Yes

Principal Investigator

Susan Perrine, M.D.

Investigator Role:

Study Director

Investigator Affiliation:

HemaQuest Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

HQP 2008-004

NCT ID:

NCT00842088

Start Date:

March 2009

Completion Date:

Related Keywords:

  • Sickle Cell Disease
  • Anemia, Sickle Cell

Name

Location

Johns Hopkins School of Medicine Baltimore, Maryland  21205
University of Illinois at Chicago Chicago, Illinois  60612
Children's Hospital and Research Center at Oakland Oakland, California  94609-1809
Medical College of Georgia Augusta, Georgia  30912
Trialogic Research Madison, Alabama  35758
Century Clinical Research, Inc. Daytona Beach, Florida  32117
UNC Comprehensive Sickle Cell Program Chapel Hill, North Carolina  27599
Texas Children's Cancer Center and Hematology Service Houston, Texas  77030