Intravenous Gammaglobulin for Sickle Cell Pain Crises

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2016 by Albert Einstein College of Medicine of Yeshiva University
Sponsor:
Information provided by (Responsible Party):
Deepa Manwani, Albert Einstein College of Medicine of Yeshiva University
ClinicalTrials.gov Identifier:
NCT01757418
First received: November 8, 2012
Last updated: January 26, 2016
Last verified: January 2016

November 8, 2012
January 26, 2016
November 2008
September 2019   (final data collection date for primary outcome measure)
Time to end of vaso-occlusive crisis [ Time Frame: Number of days from start of study drug infusion to end of crisis, average 4 days and maximum 30days ] [ Designated as safety issue: No ]
Time to end of vaso-occlusive crisis as measured from start of study drug infusion to end of VOC end of VOC defined as 12 hours from the last dose of parenteral opioid analgesia for the treatment of VOC prior to hospital discharge
Duration of pain crisis [ Time Frame: Number of days from study drug infusion to end of crisis, average 4 days and maximum 30days ] [ Designated as safety issue: No ]
End of crisis defined as either 1) Pain score consistently ≤ 5 (on the visual analog or Wong-Baker FACES scale) AND off of IV opioids or 2) Hospital discharge
Complete list of historical versions of study NCT01757418 on ClinicalTrials.gov Archive Site
Total opioid use in equivalent of mg of IV morphine [ Time Frame: From study drug infusion to end of crisis, average 4 days and maximum 30days ] [ Designated as safety issue: No ]
End of VOC end of VOC defined as 12 hours from the last dose of parenteral opioid analgesia for the treatment of VOC prior to hospital discharge
Total opioid use in equivalent of mg of IV morphine [ Time Frame: From study drug infusion to end of crisis, average 4 days and maximum 30days ] [ Designated as safety issue: No ]
End of crisis defined as either: 1)Pain score consistently ≤ 5 (on the visual analog or Wong-Baker FACES scale AND off of IV opioids or 2) Hospital discharge
Not Provided
Not Provided
 
Intravenous Gammaglobulin for Sickle Cell Pain Crises
Phase 1-2 Trial of Gamunex (Intravenous Gammaglobulin) for Sickle Cell Acute Pain

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)

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.
Interventional
Phase 1
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Sickle Cell Disease
  • Pain
  • 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 Name: GAMUNEX (Talecris Biotherapeutics)
  • Drug: Normal saline
    A single dose of normal saline administered within 24 hours of hospital admission for uncomplicated pain crisis.
    Other Name: Placebo
  • 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.
    Intervention: Drug: Immune Globulin Intravenous
  • Placebo Comparator: Normal saline
    An equivalent volume (weight-based)of normal saline
    Intervention: Drug: Normal saline

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
94
July 2020
September 2019   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Documented diagnosis of sickle cell disease (SS or S-β thalassemia genotype)
  • Age 12-65 years for Phase 1, 8-21 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%
  • 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
Both
12 Years to 65 Years   (Child, Adult)
No
Contact: Deepa G Manwani, M.D 718-741-2342 dmanwani@montefiore.org
Contact: Karen Ireland 718-741-2401 kireland@montefiore.org
United States
 
NCT01757418
FD-R-005341-01, FD-R-005341-01
Yes
Undecided
Not Provided
Deepa Manwani, Albert Einstein College of Medicine of Yeshiva University
Albert Einstein College of Medicine of Yeshiva University
Not Provided
Principal Investigator: Deepa G Manwani, M.D Albert Einstein College of Medicine of Yeshiva University
Albert Einstein College of Medicine of Yeshiva University
January 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP