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Safety Of Rivipansel (GMI-1070) In The Treatment Of One or More Vaso-occlusive Crises In Hospitalized Subjects With Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT02433158
Recruitment Status : Recruiting
First Posted : May 4, 2015
Last Update Posted : October 4, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

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).

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Sickle Cell Disease Sickle Cell Disorders Pain Crisis Vaso-occlusive Crisis Drug: Rivipansel Phase 3

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  (Clinical Trial)
Estimated Enrollment : 250 participants
Allocation: Non-Randomized
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : December 16, 2015
Estimated Primary Completion Date : August 18, 2021
Estimated Study Completion Date : August 18, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Cohort 1
includes one adult stratum (>18 years old) and one pediatric stratum (12-17 years old)
Drug: Rivipansel
Rivipansel will be infused intravenously every 12 hours up to a maximum of 15 doses. 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. Subjects 6 to 11 years of age or subject who weighs 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).
Other Name: GMI-1070

Experimental: Cohort 2
includes one pediatric stratum (6-11 years old).
Drug: Rivipansel
Rivipansel will be infused intravenously every 12 hours up to a maximum of 15 doses. 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. Subjects 6 to 11 years of age or subject who weighs 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).
Other Name: GMI-1070




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.


Secondary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02433158


Contacts
Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

  Show 109 Study Locations
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT02433158     History of Changes
Other Study ID Numbers: B5201003
First Posted: May 4, 2015    Key Record Dates
Last Update Posted: October 4, 2018
Last Verified: October 2018

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

Keywords provided by Pfizer:
Rivipansel
GMI-1070
Selectin Inhibitor
SCD
VOC

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn