Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis

Completed · Phase 2 · Has a placebo group

Conditions studied: Sickle Cell Disease, Vaso-occlusive Crisis, Pain Crisis

In brief

GMI-1070 is a new drug that may reduce the stickiness of cells in the blood. The purpose of this study is to evaluate whether GMI-1070 can reduce the time it takes for pain to go away in patients with vaso-occlusive crisis (also known as a sickle cell pain crisis). The study will also collect information on the safety of GMI-1070, how much of the drug is in the blood and urine, and if there are any other effects when used in patients who are in the hospital for a sickle cell pain crisis.

Key facts

Study ID: NCT01119833
Run by: GlycoMimetics Incorporated
People needed: 81
Starts: 2010-05-01
Expected to finish: 2013-12-01
Last updated by the study team: 2020-05-13

Who can join

Age: 12 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

12 to 60 years of age
Confirmed diagnosis of sickle cell disease (HbSS or HbS-β0thalassemia)
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 initial medical evaluation in the Emergency Department/clinic for VOC;
o Subjects treated as an outpatient within the past 48 hours for the same VOC episode may be enrolled if dosing is also expected within 24 hours of their second (admitting) presentation.
Documented and observed written informed consent (and assent, where applicable)

You may not qualify if…

Infection, diagnosed or strongly suspected, as evidenced by one or more of the following:
Fever >39°C (102.2°F)
In the presence of fever ≥38.5°C (101.3°F), 1 of the following:
Positive findings (suspicious for infection) on diagnostic tests, such as cerebral spinal fluid [CSF] evaluation, radiographs, or bacterial culture of normally sterile sites
Exam findings leading to diagnosed or strongly suspected bone or joint infection
Determination by physician that bacterial or serious systemic viral infection is likely (eg, influenza, mononucleosis)
Subjects may be included with uncomplicated urinary tract infections (provided they do not have fever ≥38.5° C [101.3° F] or costo-vertebral angle [CVA] tenderness), and/or suspected minor viral syndromes (upper respiratory infection symptoms but no symptoms suggestive of bacterial infection other than uncomplicated otitis media or uncomplicated streptococcal pharyngitis)
Acute chest syndrome, diagnosed or strongly suspected, as evidenced by a new infiltrate on chest radiograph, and 1 or more of the following:
Fever >39° C (102.2° F)
Hypoxia (confirmed by arterial blood gases [ABG] with paO2 <70 mmHg)
Chest pain
Suspicious findings on exam (tachypnea, intercostal retractions, wheezing, and/or rales)
Sickle cell disease (SCD) pain atypical of VOC, including hepatic or splenic sequestration, cholecystitis, or pneumonia.
Acute stroke, acute priapism, severe avascular necrosis of the hip/shoulder when the presenting pain is only in the affected hip/shoulder
Serum creatinine:
>1.2 mg/dL for subjects 16 to 60 years of age
>1.0 mg/dL for subjects 12 to 15 years of age
Alanine transaminase (ALT/SGPT) >2x upper limit of normal (ULN) (based on clinic laboratory normal range)
Hemoglobin <5 g/dL
Platelets <100,000/mm3
Recent (within the past 30 days) major surgery, hospitalization for other than VOC, documented serious bacterial infection requiring antibiotic treatment, or significant bleeding
Hospitalization for uncomplicated VOC, or treated with parenteral pain medications in other medical settings such as the emergency department or day hospital for uncomplicated VOC, within past 14 days.
o Subjects may be included if treated as an outpatient within the past 48 hours for the same VOC episode.
Recent (within the past 90 days) cerebrovascular accident, transient ischemic attack, or seizure
pRBC transfusions in the past 14 days

Where it is running

University of Alabama Hospital — Birmingham, Alabama, United States
Alta Bates Summit Medical Center — Berkeley, California, United States
University of California, Davis Medical Center — Sacramento, California, United States
University of Colorado — Aurora, Colorado, United States
University of Connecticut Health Center — Farmington, Connecticut, United States
Children's National Medical Center — Washington D.C., District of Columbia, United States
University of Miami Miller School of Medicine — Miami, Florida, United States
Georgia Health Sciences University — Augusta, Georgia, United States
University of Illinois, Chicago — Chicago, Illinois, United States
The Johns Hopkins School of Medicine — Baltimore, Maryland, United States
Boston Medical Center — Boston, Massachusetts, United States
Karmanos Cancer Institute — Detroit, Michigan, United States
University of Mississippi Medical Center — Jackson, Mississippi, United States
New York Methodist Hospital — Brooklyn, New York, United States
Children's Hospital at Montefiore — The Bronx, New York, United States
Duke University Medical Center — Durham, North Carolina, United States
Cincinnati Childrens' Hospital — Cincinnati, Ohio, United States
The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
UT Southwestern Medical Center at Dallas — Dallas, Texas, United States
Virginia Commonwealth University — Richmond, Virginia, United States
The Hospital for Sick Children — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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