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

NCT01119833 | Completed Phase 2 DMC

• 1 other identifier: GMI-1070-201
• Study Type: interventional
• Target: 81
• Locations: 2 countries
• Sites: 22

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.

Conditions

Sickle Cell Disease; Vaso-occlusive Crisis; Pain Crisis

Keywords

Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Reduction in time to resolution of vaso-occlusive crisis

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

  Up to 7 days or resolution

Secondary Outcomes (3)

• Safety during the study

  Up to 28 days post last dose

• Pharmacokinetics

  Baseline thru 36 hrs post last dose

• Markers of inflammation and cell stickiness in the blood

  Up thru 28 days post last dose

Study Arms (2)

GMI-1070

EXPERIMENTAL

Drug: GMI-1070

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

GMI-1070 DRUG

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

GMI-1070

Placebo DRUG

Intravenous Placebo given twice a day during hospital stay for sickle cell pain crisis

Also known as: saline placebo

Placebo

Eligibility Criteria

• Age: 12 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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:

Sponsors & Collaborators

• GlycoMimetics Incorporated: lead

Study Sites (22)

University of Alabama Hospital

Birmingham, Alabama, 35294, United States

Location

Alta Bates Summit Medical Center

Berkeley, California, 94705, United States

Location

University of California, Davis Medical Center

Sacramento, California, 95817, United States

Location

University of Colorado

Aurora, Colorado, 80045, United States

Location

University of Connecticut Health Center

Farmington, Connecticut, 06030, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

Georgia Health Sciences University

Augusta, Georgia, 30912, United States

Location

University of Illinois, Chicago

Chicago, Illinois, 60612, United States

Location

The Johns Hopkins School of Medicine

Baltimore, Maryland, 21205, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

New York Methodist Hospital

Brooklyn, New York, 11215, United States

Location

Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Childrens' Hospital

Cincinnati, Ohio, 45229, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

UT Southwestern Medical Center at Dallas

Dallas, Texas, 75235, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1XB, Canada

Location

Related Publications (3)

• Rebelo AL, Chevalier MT, Russo L, Pandit A. Role and therapeutic implications of protein glycosylation in neuroinflammation. Trends Mol Med. 2022 Apr;28(4):270-289. doi: 10.1016/j.molmed.2022.01.004. Epub 2022 Feb 1.

  PMID: 35120836 DERIVED

• Telen MJ, Wun T, McCavit TL, De Castro LM, Krishnamurti L, Lanzkron S, Hsu LL, Smith WR, Rhee S, Magnani JL, Thackray H. Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use. Blood. 2015 Apr 23;125(17):2656-64. doi: 10.1182/blood-2014-06-583351. Epub 2015 Mar 2.

  PMID: 25733584 DERIVED

• Deal watch: Pfizer deal for selectin inhibitor highlights potential of glycomimetic drugs. Nat Rev Drug Discov. 2011 Dec 1;10(12):890. doi: 10.1038/nrd3622. No abstract available.

  PMID: 22129980 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell; Vaso-Occlusive Crises

Interventions

rivipansel

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Marilyn J Telen, MD

  Duke University

  PRINCIPAL INVESTIGATOR

• Helen Thackray, MD

  GlycoMimetics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2010
• First Posted: May 10, 2010
• Study Start: May 1, 2010
• Primary Completion: December 1, 2012
• Study Completion: December 1, 2013
• Last Updated: May 13, 2020

Record last verified: 2020-05

Locations

US (21); CA (1)