Clinical Trial
Study Details
A single dose of intravenous immune globulin administered within 24 hours of hospital presentation. The maximum dose in Phase I was 800 mg/kg. The dose for Phase II is 400mg/kg.
A single dose of normal saline administered within 24 hours of hospital admission for uncomplicated pain crisis.
The purpose of this study is to determine whether Intravenous Immunoglobulin (IVIG) is safe and effective in the acute treatment of pain crises in sickle cell disease.
Funding Source: Food and Drug Administration (FDA), Office of Orphan Products Development (OOPD)
Study Overview
Patients will be randomized to a single dose of IVIG versus normal saline placebo during an uncomplicated pain crisis. Length of vaso-occlusive crisis (VOC) and other secondary endpoints will be monitored.
Phase I: To determine the tolerability and obtain preliminary data on the clinical efficacy of IVIG treatment in a randomized, double-blind, placebo-controlled, dose escalation Phase I clinical study of sickle cell disease patients, ages 12-65, admitted for acute vaso-occlusive crisis.
Phase II: To evaluate the effect of a single dose of 400mg/kg of intravenous (IV) Gamunex on length of VOC in subjects 6-13.99 years of age (initially 8-65 years of age, see "NOTES/CLARIFICATION below) hospitalized for sickle cell VOC in a randomized, double blind placebo-controlled Phase II trial. To further evaluate safety of a single dose of 400mg/kg of IV Gamunex in subjects 6-65 years of age hospitalized for sickle cell VOC.
NOTES/CLARIFICATION:
The following is a timeline of the 'evolution' of the required Age Range as per eligibility criteria for this study:
Initial Age Range: 8-65 years of age Effective 1/2/2013: 12-65 years of age Effective 3/31/2015: 8-21 years of age Effective 6/22/2018: 8-13 years of age Effective 7/11/2019: 6-13.99 years of age
Contacts and Locations
Montefiore Medical Center
The Bronx, New York 10467
United States