Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
A Randomized, Blinded, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 in Subjects With Sickle Cell Disease
1 other identifier
interventional
24
2 countries
9
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 11, 2009
CompletedFirst Posted
Study publicly available on registry
February 12, 2009
CompletedStudy Start
First participant enrolled
March 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2010
CompletedAugust 1, 2011
July 1, 2011
1.3 years
February 11, 2009
July 28, 2011
Conditions
Outcome Measures
Primary Outcomes (1)
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.
126 days
Secondary Outcomes (2)
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
Study Arms (2)
Active
EXPERIMENTALPlacebo
PLACEBO COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
- +2 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (9)
Trialogic Research
Madison, Alabama, 35758, United States
Children's Hospital and Research Center at Oakland
Oakland, California, 94609, United States
Century Clinical Research, Inc.
Daytona Beach, Florida, 32117, United States
Medical College of Georgia
Augusta, Georgia, 30912, United States
University of Illinois at Chicago
Chicago, Illinois, 60612, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, 21205, United States
UNC Comprehensive Sickle Cell Program
Chapel Hill, North Carolina, 27599, United States
Texas Children's Cancer Center and Hematology Service
Houston, Texas, 77030, United States
University of the West Indies, Mona
Kingston, Mona, 7, Jamaica
Related Publications (1)
Kutlar A, Ataga K, Reid M, Vichinsky EP, Neumayr L, Blair-Britt L, Labotka R, Glass J, Keefer JR, Wargin WA, Berenson R, Perrine SP. A phase 1/2 trial of HQK-1001, an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2012 Nov;87(11):1017-21. doi: 10.1002/ajh.23306. Epub 2012 Aug 7.
PMID: 22887019DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Susan Perrine, M.D.
HemaQuest Pharmaceuticals
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
February 11, 2009
First Posted
February 12, 2009
Study Start
March 1, 2009
Primary Completion
July 1, 2010
Last Updated
August 1, 2011
Record last verified: 2011-07