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

Sponsor
GlycoMimetics Incorporated (Industry)
Overall Status
Completed
CT.gov ID
NCT01119833
Collaborator
(none)
81
Enrollment
22
Locations
2
Arms
43
Duration (Months)
3.7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase
Phase 2

Detailed Description

Patients being admitted to the hospital for pain crisis may be eligible for this study. In addition, patients should be 12-60 years old and have sickle cell types SS or S-beta-thalassemia. People who take part in the study will be evaluated and then randomly assigned to receive either GMI-1070 or a placebo by IV, in addition to all other usual treatments for their pain crisis.

During the hospital stay for pain crisis, GMI-1070 or placebo will be given twice a day, and patients will be asked about their pain severity (pain score) at the beginning of the study and every few hours during their hospital stay. Their general health, vital signs, lab tests, and pain medications will also be checked on a regular basis through the hospital stay. When a patient is feeling well enough to go home, the study drug (GMI-1070 or placebo) will be stopped, and the patient may go home. Participants will be asked to come back to clinic for a check-up a few days after leaving the hospital, and one month after leaving the hospital.

Study Design

Study Type:
Interventional
Actual Enrollment :
81 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Pharmacokinetics of GMI-1070, A Pan-Selectin Inhibitor, In Subjects Hospitalized For Sickle Cell Vaso-Occlusive Crisis
Study Start Date :
May 1, 2010
Actual Primary Completion Date :
Dec 1, 2012
Actual Study Completion Date :
Dec 1, 2013

Arms and Interventions

Arm Intervention/Treatment
Experimental: GMI-1070

Drug: GMI-1070
Intravenous GMI-1070 given twice a day during hospital stay for sickle cell pain crisis
Placebo Comparator: Placebo

Drug: Placebo
Intravenous Placebo given twice a day during hospital stay for sickle cell pain crisis
Other Names:
  • saline placebo

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Reduction in time to resolution of vaso-occlusive crisis [Up to 7 days or resolution]

      Including pain score, feeling ready to leave the hospital, and actual time of leaving the hospital

    Secondary Outcome Measures

    1. Safety during the study [Up to 28 days post last dose]

      Including changes in physical exam, lab tests, and vital signs

    2. Pharmacokinetics [Baseline thru 36 hrs post last dose]

      Pharmacokinetics including half-life and concentration of GMI-1070 in the blood and urine

    3. Markers of inflammation and cell stickiness in the blood [Up thru 28 days post last dose]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years to 60 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. 12 to 60 years of age

    2. Confirmed diagnosis of sickle cell disease (HbSS or HbS-β0thalassemia)

    3. Diagnosis of VOC at the time of enrollment

    4. Hospitalized or in process of admission at the time of enrollment

    5. 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.

    1. Documented and observed written informed consent (and assent, where applicable)
    Exclusion Criteria:
    1. 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)

    1. 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)

    1. Sickle cell disease (SCD) pain atypical of VOC, including hepatic or splenic sequestration, cholecystitis, or pneumonia.

    2. Acute stroke, acute priapism, severe avascular necrosis of the hip/shoulder when the presenting pain is only in the affected hip/shoulder

    3. 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

    1. Alanine transaminase (ALT/SGPT) >2x upper limit of normal (ULN) (based on clinic laboratory normal range)

    2. Hemoglobin <5 g/dL

    3. Platelets <100,000/mm3

    4. Recent (within the past 30 days) major surgery, hospitalization for other than VOC, documented serious bacterial infection requiring antibiotic treatment, or significant bleeding

    5. 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.

    1. Recent (within the past 90 days) cerebrovascular accident, transient ischemic attack, or seizure

    2. pRBC transfusions in the past 14 days

    3. Systemic steroid therapy within 48 hours prior to enrollment or expectation that therapy may be used during the study (inhaled or topical steroids are allowed)

    4. For those on chronic or long-acting opioids, a change in dose in the past 14 days OR pain requiring medical attention in the past 14 days (change in opioid medication for acute pain in the past 48 hours and directly related to this VOC admission is allowed)

    5. Greater than 5 episodes of hospitalization for VOC in the past 6 months (180 days)

    6. Medical or psychiatric condition that, in the opinion of the investigator, may pose a risk to the subject for participation or interfere with the conduct or results of the study

    7. Currently receiving, or has received within the previous 4 weeks, any other investigational agent

    8. Previous administration of GMI-1070

    9. Expectation that the subject will not be able to be followed for the duration of the study

    10. Pregnant or lactating female; or female of childbearing potential or male unable or unwilling to comply with birth control methods or abstinence during the course of the study

    11. Active use of illicit drugs and/or alcohol dependence, as determined by the investigator

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Alabama Hospital Birmingham Alabama United States 35294
    2 Alta Bates Summit Medical Center Berkeley California United States 94705
    3 University of California, Davis Medical Center Sacramento California United States 95817
    4 University of Colorado Aurora Colorado United States 80045
    5 University of Connecticut Health Center Farmington Connecticut United States 06030
    6 Children's National Medical Center Washington District of Columbia United States 20010
    7 University of Miami Miller School of Medicine Miami Florida United States 33136
    8 Georgia Health Sciences University Augusta Georgia United States 30912
    9 University of Illinois, Chicago Chicago Illinois United States 60612
    10 The Johns Hopkins School of Medicine Baltimore Maryland United States 21205
    11 Boston Medical Center Boston Massachusetts United States 02118
    12 Karmanos Cancer Institute Detroit Michigan United States 48201
    13 University of Mississippi Medical Center Jackson Mississippi United States 39216
    14 Children's Hospital at Montefiore Bronx New York United States 10467
    15 New York Methodist Hospital Brooklyn New York United States 11215
    16 Duke University Medical Center Durham North Carolina United States 27710
    17 Cincinnati Childrens' Hospital Cincinnati Ohio United States 45229
    18 The Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    19 Children's Hospital of Pittsburgh Pittsburgh Pennsylvania United States 15224
    20 UT Southwestern Medical Center at Dallas Dallas Texas United States 75235
    21 Virginia Commonwealth University Richmond Virginia United States 23298
    22 The Hospital for Sick Children Toronto Ontario Canada M5G 1XB

    Sponsors and Collaborators

    • GlycoMimetics Incorporated

    Investigators

    • Principal Investigator: Marilyn J Telen, MD, Duke University
    • Study Director: Helen Thackray, MD, GlycoMimetics, Inc.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    GlycoMimetics Incorporated
    ClinicalTrials.gov Identifier:
    NCT01119833
    Other Study ID Numbers:
    • GMI-1070-201
    First Posted:
    May 10, 2010
    Last Update Posted:
    May 13, 2020
    Last Verified:
    May 1, 2020
    Keywords provided by GlycoMimetics Incorporated
    Sickle Cell Disease
    Additional relevant MeSH terms:
    Anemia, Sickle Cell

    Study Results

    No Results Posted as of May 13, 2020