Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease

Summary

This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.

Timeline

Start

2015-06-17

Primary completion

2019-05-03

Completion

2019-06-27

Outcome

Missed primary endpoint

registry analysis (superiority test); Rivipansel Treatment Arm vs Placebo Treatment Arm; p = 0.7944; Hazard Ratio (HR) 0.97 (95% CI 0.77 to 1.22); Log Rank NCT02187003 ↗

paper the primary end point, was not different between rivipansel and placebo (-5.7 hours, P = .79; hazard ratio, 0.97) PMID 35981565 ↗

release “RESET pivotal study did not meet its primary or key secondary efficacy endpoints.” pfizer.com ↗

Publications

• Dampier CD, Telen MJ, Wun T, Brown RC, Desai P, El Rassi F, Fuh B, Kanter J, Pastore Y, Rothman J, Taylor JG, Readett D, Sivamurthy KM, Tammara B, Tseng LJ, Lozier JN, Thackray H, Magnani JL, Hassell KL; RESET Investigators. A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis. Blood. 2023 Jan 12;141(2):168-179. doi: 10.1182/blood.2022015797.
• 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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Rivipansel	Small molecule	20 mg/kg	Intravenous
Subject	Rivipansel	Small molecule	840 mg	Intravenous

Indications