Why are we doing this research?
The research purposes of this study are to:
- Test the effect of GMI-1070 on vaso-occlusive crisis.
- Test how safe a number of doses of intravenous (IV-through the vein) GMI-1070 are in people with Sickle Cell Disease (SCD) who have vaso-occlusive crisis.
- Find out how much GMI-1070 is in the blood and urine of subjects after taking the study drug.
- Measure the effect of GMI-1070 on various blood tests that show evidence of inflammation (a response of body tissue to injury or irritation) and adhesion (stickiness) of cells in your body.
Who can participate?
To be eligible for inclusion, you must fulfill each of the following criteria at screening, and must continue to fulfill these criteria prior to dosing:
- 12 to 60 years of age
- Confirmed diagnosis of sickle cell disease (HbSS or HbS-β0 thalassemia)
- Diagnosis of VOC at the time of enrollment
- Hospitalized or in process of admission at the time of enrollment
- Able to receive the first dose of study drug within 24 hours of first medical evaluation in the Emergency Department/clinic for VOC
- Documented and observed written informed consent (and assent, where applicable)
To be eligible for the study, you may not have any of the following:
- Infection that is diagnosed or strongly suspected by your doctors.
- Acute chest syndrome that is diagnosed or strongly suspected by your doctors. Acute chest syndrome is a pneumonia-like illness in someone with sickle cell disease that can be seen on an X-ray of the chest.
- SCD pain that is not usually associated with vaso-occlusive crisis, such as pain or swelling in your liver, spleen, or lungs.
- Acute priapism (prolonged erection of the penis)
- Severe pain in the hip or shoulder due to damage to tissues or bone
- Recent (within the past 30 days) major surgery, hospitalization for a complication other than VOC (pain crisis), documented serious infection requiring medicine, or significant bleeding
- Hospitalization for uncomplicated VOC (pain crisis) or treatment with IV (by vein) pain medications (IV meds) in other medical settings such as the emergency department or day hospital for uncomplicated VOC within the past 14 days (subjects may be included if treated as an outpatient with the past 48 hours for the same VOC episode)
- Recent (within the past 90 days) stroke, transient ischemic attack (TIA—similar to a stroke, but lasting less than one day and causing no permanent damage), or seizure
- Blood transfusions in the past 14 days
- Steroid medicines taken by mouth or vein within 48 hours prior to enrollment(Inhaled or topical steroids are allowed).
- If you are on certain long-term, daily, or long-acting opioids (OxyContin, MS Contin), and have needed a change in dose in the past 14 days OR pain requiring medical attention in the past 14 days. A change in opioid medication for acute pain in the past 48 hours and directly related to your current hospitalization for pain crisis (VOC) is allowed.
- Greater than 5 episodes of hospitalization for VOC in the past 6 months (180 days)
- Medical or mental condition that, in the opinion of the investigator, may pose a risk to you for participation or interfere with the conduct or results of the study
- Currently receiving, or have received within the previous 4 weeks, any other investigational agent
- Previous administration of GMI-1070
- Pregnant or breastfeeding
- Active use of illicit drugs and/or alcohol dependence
Will you/your child be paid to be in this research study?
You will be given a small payment to reimburse you for your time. You will be given $50 for completing Visit 2, $75 for completing Visit 3, and $75 for completing Visit 4.