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Sickle Cell DiseasePhase 2NCT01119833

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

A Phase 2 study testing GMI-1070 in people with Sickle Cell Disease and Vaso-occlusive Crisis, run by GlycoMimetics Incorporated at 22 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 12 to 60 years
What is being tested?
GMI-1070
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
81 participants
Where
22 sitesin 2 countries
Study dates
May 2010to Dec 2013
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 60 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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)

Who cannot join

  • 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)
Show all 34Show fewer
  • 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
  • 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)
  • 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)
  • Greater than 5 episodes of hospitalization for VOC in the past 6 months (180 days)
  • 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
  • Currently receiving, or has received within the previous 4 weeks, any other investigational agent
  • Previous administration of GMI-1070
  • Expectation that the subject will not be able to be followed for the duration of the study
  • 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
  • Active use of illicit drugs and/or alcohol dependence, as determined by the investigator

Trial locations and contacts

22 sites in 2 countries.

22 sites in 2 countries

United States21 sites

  • Georgia Health Sciences University

    Augusta, Georgia, 30912

  • University of Colorado

    Aurora, Colorado, 80045

  • The Johns Hopkins School of Medicine

    Baltimore, Maryland, 21205

  • Alta Bates Summit Medical Center

    Berkeley, California, 94705

  • University of Alabama Hospital

    Birmingham, Alabama, 35294

  • Boston Medical Center

    Boston, Massachusetts, 02118

  • New York Methodist Hospital

    Brooklyn, New York, 11215

  • University of Illinois, Chicago

    Chicago, Illinois, 60612

  • Cincinnati Childrens' Hospital

    Cincinnati, Ohio, 45229

  • UT Southwestern Medical Center at Dallas

    Dallas, Texas, 75235

  • Karmanos Cancer Institute

    Detroit, Michigan, 48201

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • University of Connecticut Health Center

    Farmington, Connecticut, 06030

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216

  • University of Miami Miller School of Medicine

    Miami, Florida, 33136

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

  • Virginia Commonwealth University

    Richmond, Virginia, 23298

  • University of California, Davis Medical Center

    Sacramento, California, 95817

  • Children's Hospital at Montefiore

    The Bronx, New York, 10467

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

Canada1 site

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1XB

Other trials in Canada

About this study

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

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.

Read the 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.

Being tested
GMI-1070, Placebo
Keywords
Sickle Cell Disease
Medical subject headings
Anemia, Sickle Cell, Vaso-Occlusive Crises

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GMI-1070Drug

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

  • PlaceboDrug

    Also called saline placebo

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

Study groups in NCT01119833
Study groupRoleWhat they receive
GMI-1070ExperimentalNot described
Receives: Drug: GMI-1070
PlaceboPlacebo comparatorNot described
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: Up to 7 days or resolution

Secondary outcomes (3)

  1. Safety during the study

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

    Measured: Up to 28 days post last dose

  2. Pharmacokinetics

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

    Measured: Baseline thru 36 hrs post last dose

  3. Markers of inflammation and cell stickiness in the blood

    Measured: Up thru 28 days post last dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 May 2010 (estimated)
  2. Study startMay 2010
  3. Primary completionDec 2012
  4. Study completionDec 2013

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
81 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01119833
Sponsor's protocol ID
GMI-1070-201
Status last verified
May 2020
First submitted
5 May 2010
Last updated
13 May 2020
Results posted
No
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 35120836

  • 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.

    PubMed 25733584

  • 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.

    PubMed 22129980

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing GMI-1070 in people with Sickle Cell Disease and Vaso-occlusive Crisis, run by GlycoMimetics Incorporated at 22 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 34 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 May 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Reduction in time to resolution of vaso-occlusive crisis (time frame: Up to 7 days or resolution). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01119833, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2020.

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