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 range
6 Years – 13 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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