Intravenous Gammaglobulin for Sickle Cell Pain Crises
United States300 participantsStarted 2008-11
Plain-language summary
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)
Who can participate
Age range6 Years – 13 Years
SexALL
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Each subject must fulfill each of the following Inclusion/Exclusion criteria at screening and continue to fulfill these criteria prior to dosing:
Inclusion Criteria:
* Documented Sickle Cell Disease (SS or S-β thalassemia genotype)
* Age 12-65 years for Phase 1 (Completed), 6-13.99 years for Phase 2 (Ongoing)
* Normal stroke risk as assessed by transcranial Doppler (TCD). A normal TCD in subjects 16 years of age and younger within the year prior to study drug administration are required
* Uncomplicated acute vaso-occlusive crisis requiring hospital admission and parenteral narcotic analgesics
* If prescribed Voxelotor: Consistent daily use of voxelotor in the past week AND able to continue Voxelotor inpatient OR no reported use in prior week
Exclusion Criteria:
* Concomitant acute process, including acute chest syndrome, potential serious infection, or clinically significant bleeding
* Fever \> 38.5° C and clinical suspicion of infection
* Serum alanine aminotransferase \>4x Upper Limit of Normal (ULN)
* Serum creatinine ≥1.3 mg/dL (or \> than 95th percentile for age) or \>300 mg/dL protein in spot urinalysis
* Known condition associated with renal dysfunction including but not limited to diabetes mellitus, uncontrolled hypertension, multiple myeloma, and congestive heart failure
* Any clinical evidence of prior stroke
* Prior thromboses or current estrogen use
* Current estrogen use
* Hb \< 5 g/dL or \> 10 g/dL
* Known Immunoglobulin A (IgA) deficiency or known allergy t…
What they're measuring
1
Length of vaso-occlusive crisis (VOC)
Timeframe: Number of days from time of presentation to emergency room to end of crisis, average 4 days and maximum 30 days