NCT01119833

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 81 (actual)
• Sponsor: GlycoMimetics Incorporated · Industry
• Sex: All
• Age: 12 Years – 60 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Sickle Cell Disease
• Vaso-occlusive Crisis
• Pain Crisis

Interventions

Type	Name	Description
DRUG	GMI-1070	Intravenous GMI-1070 given twice a day during hospital stay for sickle cell pain crisis
DRUG	Placebo	Intravenous Placebo given twice a day during hospital stay for sickle cell pain crisis

Timeline

• Start date: 2010-05-01
• Primary completion: 2012-12-01
• Completion: 2013-12-01
• First posted: 2010-05-10
• Last updated: 2020-05-13

Locations

22 sites across 2 countries: United States, Canada