Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease

GlycoMimetics

Status and phase: Terminated; Phase 3

Conditions: Vaso-occlusive Crisis; Pain Crisis; Sickle Cell Disorders; Sickle Cell Anemia; Sickle Cell Disease

Treatments: Drug: Rivipansel

Study type: Interventional

Funder types: Industry

Identifiers

NCT02433158

B5201003

Details and patient eligibility

About

This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).

Full description

This is an open label extension study in subjects who are 6 years of age or older with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002). This study is designed to evaluate the safety and describe the efficacy of rivipansel as treatment for one or more vaso-occlusive crisis (VOC) events in hospitalized subjects with SCD.

Enrollment: 154 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Completion of Study B5201002.
• Documented diagnosis of SCD.
• At least 6 years of age.
• Male and female subjects of childbearing potential and at risk for pregnancy must agree to use a highly effective method of contraception throughout the study.
• Diagnosis of VOC necessitating IV opioids and admission to the hospital.
• Able to receive the first dose of rivipansel within 24 hours from administration of the first dose of IV opioids for this hospitalization.

Exclusion criteria

• Non-compliance with study procedures in the double blind study (B5201002).
• Occurrence of any severe and/or generalized cutaneous manifestation or any other adverse event during participation in Study B5201002 that was related to study drug and which would therefore make it inappropriate for the subject to receive rivipansel in the current study.
• Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration or may interfere with the interpretation of study results.
• Clinically significant deterioration in renal function in Study B5201002.
• Pregnant female subjects, breastfeeding female subjects and male and female subjects of childbearing potential who are unwilling or unable to use a highly effective method of contraception.
• Active use of illicit drugs and/or alcohol dependence.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

154 participants in 2 patient groups

Cohort 1

Experimental group

Description:

Includes one adult stratum (>18 years old) and one pediatric stratum (12-17 years old). Subjects aged 12 and over who weigh >40 kg, will receive a loading dose of 1680 mg followed by a maintenance dose of 840 mg.

Treatment:

Drug: Rivipansel

Cohort 2

Experimental group

Description:

Includes one pediatric stratum (6-11 years old). Subjects 6 to 11 years of age or subjects who weigh 40 kg, will receive a loading dose of 40 mg/kg (maximum of 1680 mg) followed by a maintenance dose of 20 mg/kg (maximum of 840 mg).

Treatment:

Drug: Rivipansel