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

• ClinicalTrials.gov Identifier: NCT02433158
• Recruitment Status: Terminated
• First Posted: May 4, 2015
• Last Update Posted: February 10, 2021
• Sponsor: GlycoMimetics Incorporated
• Information provided by (Responsible Party): GlycoMimetics Incorporated

Tracking Information

• First Submitted Date: April 29, 2015
• First Posted Date: May 4, 2015
• Last Update Posted Date: February 10, 2021
• Actual Study Start Date: December 16, 2015
• Actual Primary Completion Date: November 15, 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 29, 2015)

• Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study. [ Time Frame: 18 months ]
  Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study, number of TEAEs over the study and rate of TEAEs per subject per Vaso-Occlusive Crisis (VOC) will be summarized overall, by system organ class and by preferred term.
• Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study. [ Time Frame: 18 months ]
  Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study, number of events of adjudicated ACS and the rates of these events per subject per Vaso-Occlusive Crisis (VOC) will be summarized.
• Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study [ Time Frame: 18 months ]
  Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study, number of events of adjudicated severe and/or generalized cutaneous manifestations and the rates of these events per subject per Vaso-Occlusive Crisis (VOC) will be summarized.
• Number (%) of subjects with serious adverse events (SAEs) over the study. [ Time Frame: 18 months ]
  Number (%) of subjects with serious adverse events (SAEs) over the study, number of SAEs over the study and rate of SAEs per subject per VOC will be summarized overall, by system organ class and by preferred term.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: April 29, 2015)

Subject re hospitalization [ Time Frame: 18 months ]

Number (%) of subjects re hospitalized for VOC within 7, 14, and 30 days of most recent discharge will be provided overall. Number of re hospitalizations for VOC within 7, 14, and 30 days of most recent discharge and the rate of re hospitalization for VOC within 7, 14, and 30 days of most recent discharge per subject per VOC will be provided as well.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
• Official Title: An Open-Label Extension Study to Evaluate the Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
• Brief Summary: This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).
• Detailed 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.
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

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

Intervention

Drug: Rivipansel

Rivipansel will be infused intravenously every 12 hours up to a maximum of 15 doses.

Other Name: GMI-1070

Study Arms

• Experimental: Cohort 1
  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.
  Intervention: Drug: Rivipansel
• Experimental: Cohort 2
  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).
  Intervention: Drug: Rivipansel

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: February 18, 2020): 154
• Original Estimated Enrollment (submitted: April 29, 2015): 250
• Actual Study Completion Date: November 15, 2019
• Actual Primary Completion Date: November 15, 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, United States

Administrative Information

• NCT Number: NCT02433158
• Other Study ID Numbers: B5201003
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: GlycoMimetics Incorporated
• Study Sponsor: GlycoMimetics Incorporated
• Collaborators: Not Provided

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

• PRS Account: GlycoMimetics Incorporated
• Verification Date: February 2021