Intravenous Gammaglobulin for Sickle Cell Pain Crises

September 29, 2023 updated by: Deepa Manwani

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)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

300

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • New York
      • Bronx, New York, United States, 10467
        • Recruiting
        • Montefiore Medical Center
        • Contact:
        • Principal Investigator:
          • Deepa G Manwani, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Length of vaso-occlusive crisis
Time Frame: 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.
Number of days from time of presentation to emergency room to end of crisis, average 4 days and maximum 30 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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 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
From study drug infusion to end of crisis, average 4 days and maximum 30 days
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 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
Number of days from start of study drug infusion to end of crisis, average 4 days and maximum 30 days
In vitro adhesion assays
Time Frame: Pre and 24 hours post study drug
Activated Mac-1, Aged neutrophils
Pre and 24 hours post study drug

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Deepa Manwani

Investigators

  • Principal Investigator: Deepa G Manwani, M.D, Albert Einstein College of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2008

Primary Completion (Estimated)

December 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

November 8, 2012

First Submitted That Met QC Criteria

December 21, 2012

First Posted (Estimated)

December 31, 2012

Study Record Updates

Last Update Posted (Actual)

October 3, 2023

Last Update Submitted That Met QC Criteria

September 29, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 09-06-172
  • FD-R-005341-01 (Other Grant/Funding Number: FDA, OOPD)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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