Intravenous Gammaglobulin for Sickle Cell Pain Crises

Sponsor

Albert Einstein College of Medicine (Other)

Overall Status

Recruiting

CT.gov ID

NCT01757418

Collaborator

(none)

Enrollment

Location

Arms

200

Duration (Months)

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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 or Disease	Intervention/Treatment	Phase
Sickle Cell Disease Pain	Drug: Immune Globulin Intravenous Other: Normal saline	Phase 1/Phase 2

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

94 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

Phase 1-2 Trial of Gamunex (Intravenous Gammaglobulin) for Sickle Cell Acute Pain

Study Start Date :

Nov 1, 2008

Anticipated Primary Completion Date :

Sep 1, 2024

Anticipated Study Completion Date :

Jul 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
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

Arm

Intervention/Treatment

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

Outcome Measures

Primary Outcome Measures

Length of vaso-occlusive crisis [Number of days from time of presentation to emergency room to end of crisis, average 4 days and maximum 30 days]
Length of vaso-occlusive crisis as measured from the time of presentation to the emergency room to 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 [From study drug infusion to end of crisis, average 4 days and maximum 30 days]
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
Time to end of vaso-occlusive crisis [Number of days from start of study drug infusion to end of crisis, average 4 days and maximum 30 days]
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
In vitro adhesion assays [Pre and 24 hours post study drug]
Activated Mac-1, Aged neutrophils

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Documented diagnosis of sickle cell disease (SS or S-β thalassemia genotype)
Age 12-65 years for Phase 1, 6-13.99 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