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
  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)


Condition Intervention Phase
Sickle Cell Disease
Pain
Drug: Immune Globulin Intravenous
Drug: Normal saline
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase 1-2 Trial of Gamunex (Intravenous Gammaglobulin) for Sickle Cell Acute Pain

Resource links provided by NLM:


Further study details as provided by Albert Einstein College of Medicine of Yeshiva University:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • 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


Estimated Enrollment: 94
Study Start Date: November 2008
Estimated Study Completion Date: July 2020
Estimated Primary Completion Date: September 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: GAMUNEX (Talecris Biotherapeutics)
Placebo Comparator: Normal saline
An equivalent volume (weight-based)of normal saline
Drug: Normal saline
A single dose of normal saline administered within 24 hours of hospital admission for uncomplicated pain crisis.
Other Name: Placebo

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.
  Eligibility

Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01757418

Contacts
Contact: Deepa G Manwani, M.D 718-741-2342 dmanwani@montefiore.org
Contact: Karen Ireland 718-741-2401 kireland@montefiore.org

Locations
United States, New York
Montefiore Medical Center Recruiting
Bronx, New York, United States, 10467
Contact: Deepa G Manwani, MD    718-741-2342    dmanwani@montefiore.org   
Principal Investigator: Deepa G Manwani, MD         
Sponsors and Collaborators
Albert Einstein College of Medicine of Yeshiva University
Investigators
Principal Investigator: Deepa G Manwani, M.D Albert Einstein College of Medicine of Yeshiva University
  More Information

Publications:
Responsible Party: Deepa Manwani, Director, Sickle Cell Disease Program, Children's Hospital at Montefiore, Albert Einstein College of Medicine of Yeshiva University
ClinicalTrials.gov Identifier: NCT01757418     History of Changes
Obsolete Identifiers: NCT00644865
Other Study ID Numbers: FD-R-005341-01  FD-R-005341-01 
Study First Received: November 8, 2012
Last Updated: January 26, 2016
Health Authority: United States: Food and Drug Administration
Individual Participant Data  
Plan to Share IPD: Undecided

Keywords provided by Albert Einstein College of Medicine of Yeshiva University:
Sickle Cell Disease
Pain
Immune Globulin

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Immunoglobulins
Antibodies
Gamma-Globulins
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on August 23, 2016