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 12 Years and 65 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Documented diagnosis of 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) - Uncomplicated acute pain episode requiring hospital admission and parenteral narcotics
Exclusion Criteria
- Increased stroke risk as assessed by transcranial Doppler or magnetic resonance imaging (all subjects undergo testing) - Concomitant acute process, including fever > 38.5° C with clinical suspicion of infection - Increased ALT > 2X ULN - Serum creatinine ≥1.3 mg/dL, >300 mg/dL protein in spot urinalysis, or known condition associated with renal dysfunction - Hb > 10 g/dL and Hct > 30% - Hb< 5 g/dl - Known IgA deficiency or known allergy to gamma globulin - Pregnancy or breastfeeding - Vaccination with a live attenuated virus in the preceding 6 weeks - Documented history of illicit (eg. heroin, cocaine) drug abuse or drug-seeking behavior - Current participation in another investigational drug study - Current treatment with chronic transfusion - Prior thromboses or current estrogen use
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 Immune Globulin Intravenous |
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.