Intravenous Gammaglobulin for Sickle Cell Pain Crises
Purpose
The purpose of this study is to determine whether intravenous immune globulin 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)
Conditions
- Sickle Cell Disease
- Pain
Eligibility
- Eligible Ages
- Between 6 Years and 65 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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 to gamma globulin - Pregnancy or breastfeeding - Current participation in another investigational drug study - Current enrollment in a hypertransfusion program - Previous participation in current study less than 3 months ago - Current treatment with chronic transfusion - Vaccination with a live attenuated virus in the preceding 6 weeks - Documented history of illicit (e.g., heroin, cocaine) drug abuse - Subject is otherwise not an appropriate study candidate, in the investigator's judgement, such as concern for opioid addiction or comorbid psychiatric diagnoses that may contribute to secondary gain in prolonged use of opioids or hospital stay - Greater than 24 hours from time of presentation to the hospital for VOC - Atrial fibrillation - Right to left cardiac shunting due to patent foramen ovale or other anatomic cause - Known magnetic resonance imaging/angiography (MRI/A) evidence of stroke or clinically significant central nervous system (CNS) vasculopathy at any age (Imaging done if clinically indicated)
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Intravenous Immune Globulin (IVIG) |
IVIG used in the trial is the GAMUNEX brand, at doses up through 800 mg/kg in Phase 1 and at 400mg/kg in Phase 2. |
|
|
Placebo Comparator Normal saline |
An equivalent volume (weight-based) of normal saline |
|
Recruiting Locations
Montefiore Medical Center
Bronx, New York 10467
Bronx, New York 10467
More Details
- Status
- Recruiting
- Sponsor
- Deepa Manwani
Detailed Description
Patients will be randomized to a single dose of IVIG versus normal saline placebo during an uncomplicated pain crisis. Length of VOC and other secondary endpoints will be monitored.