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, 8-13.99 years for Phase 2
  • 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

ArmDescriptionAssigned 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.
  • Drug: Immune Globulin Intravenous
    A single dose of intravenous immune globulin or saline placebo 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.
    Other names:
    • GAMUNEX (Talecris Biotherapeutics)
Placebo Comparator
Normal saline
An equivalent volume (weight-based)of normal saline
  • Other: Normal saline
    A single dose of normal saline administered within 24 hours of hospital admission for uncomplicated pain crisis.
    Other names:
    • Placebo

Recruiting Locations

Montefiore Medical Center
Bronx, New York 10467
Contact:
Deepa G Manwani, MD
718-741-2342
dmanwani@montefiore.org

More Details

NCT ID
NCT01757418
Status
Recruiting
Sponsor
Albert Einstein College of Medicine

Study Contact

Deepa G Manwani, M.D
718-741-2342
dmanwani@montefiore.org

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.