Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
This study has been completed.
Sponsor:
HemaQuest Pharmaceuticals Inc.
Information provided by:
HemaQuest Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT00842088
First received: February 11, 2009
Last updated: July 28, 2011
Last verified: July 2011
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Sickle Cell Disease |
Drug: Placebo Drug: HQK-1001 |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
sickle cell disease
MedlinePlus related topics:
Sickle Cell Anemia
U.S. FDA Resources
Further study details as provided by HemaQuest Pharmaceuticals Inc.:
Primary Outcome Measures:
- Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam. [ Time Frame: 126 days ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Pharmacokinetics assessed by plasma drug concentration levels. [ Time Frame: Days 0, 6, 69 and 97 post first dose ] [ Designated as safety issue: No ]
- Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin. [ Time Frame: Every 2 weeks through Day 126 post first dose ] [ Designated as safety issue: No ]
| Enrollment: | 24 |
| Study Start Date: | March 2009 |
| Primary Completion Date: | July 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| 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.
|
Eligibility| Ages Eligible for Study: | 12 Years to 60 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
- 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00842088
Locations
| United States, Alabama | |
| Trialogic Research | |
| Madison, Alabama, United States, 35758 | |
| United States, California | |
| Children's Hospital and Research Center at Oakland | |
| Oakland, California, United States, 94609 | |
| United States, Florida | |
| Century Clinical Research, Inc. | |
| Daytona Beach, Florida, United States, 32117 | |
| United States, Georgia | |
| Medical College of Georgia | |
| Augusta, Georgia, United States, 30912 | |
| United States, Illinois | |
| University of Illinois at Chicago | |
| Chicago, Illinois, United States, 60612 | |
| United States, Maryland | |
| Johns Hopkins School of Medicine | |
| Baltimore, Maryland, United States, 21205 | |
| United States, North Carolina | |
| UNC Comprehensive Sickle Cell Program | |
| Chapel Hill, North Carolina, United States, 27599 | |
| United States, Texas | |
| Texas Children's Cancer Center and Hematology Service | |
| Houston, Texas, United States, 77030 | |
| Jamaica | |
| 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 |
More Information
No publications provided by HemaQuest Pharmaceuticals Inc.
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Susan Perrine, M.D., HemaQuest Pharmaceuticals, Inc. |
| ClinicalTrials.gov Identifier: | NCT00842088 History of Changes |
| Other Study ID Numbers: | HQP 2008-004 |
| Study First Received: | February 11, 2009 |
| Last Updated: | July 28, 2011 |
| Health Authority: | United States: Food and Drug Administration |
Additional relevant MeSH terms:
|
Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia |
Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on May 16, 2013