NCT01757418

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

Completed Phase 1/Phase 2 Last updated 27 March 2026

What this trial tests

Phase 1/Phase 2 trial testing Immune Globulin Intravenous (IVIG) in Sickle Cell Disease in 300 participants. Completed in 18 December 2024.

Timeline

1 November 2008

Primary endpoint
18 December 2024

18 December 2024

Quick facts

Lead sponsor	Albert Einstein College of Medicine
Phase	Phase 1/Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	300
Start date	1 November 2008
Primary completion	18 December 2024
Estimated completion	18 December 2024
Sites	1 location across United States

Drugs / interventions tested

• Immune Globulin Intravenous (IVIG) — full drug profile →
• Normal saline

Conditions studied

• Sickle Cell Disease — all drugs for Sickle Cell Disease →
• Pain — all drugs for Pain →

Sponsor

Albert Einstein College of Medicine

Who can join

Adults 6 to 13, any sex, with Sickle Cell Disease or Pain. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Length of vaso-occlusive crisis (VOC)
  Time frame: Number of days from time of presentation to emergency room to end of crisis, average 4 days and maximum 30 days
  Length (duration) 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. Group results will be summarized in number of days using univariate statistics.

Sponsor's own description

The purpose of this study is to determine whether Intravenous Immunoglobulin (IVIG) 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)

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Neutrophils, platelets, and inflammatory pathways at the nexus of sickle cell disease pathophysiology.
   Zhang D, Xu C, Manwani D, Frenette PS. · · 2016 · cited 295× · PMID 26758915 · DOI 10.1182/blood-2015-09-618538
2. Therapeutic strategies for sickle cell disease: towards a multi-agent approach.
   Telen MJ, Malik P, Vercellotti GM. · · 2019 · cited 136× · PMID 30514970 · DOI 10.1038/s41573-018-0003-2
3. Beyond hydroxyurea: new and old drugs in the pipeline for sickle cell disease.
   Telen MJ. · · 2016 · cited 90× · PMID 26758919 · DOI 10.1182/blood-2015-09-618553
4. Emerging disease-modifying therapies for sickle cell disease.
   Carden MA, Little J. · · 2019 · cited 60× · PMID 31413089 · DOI 10.3324/haematol.2018.207357
5. Sickle cell disease biochip: a functional red blood cell adhesion assay for monitoring sickle cell disease.
   Alapan Y, Kim C, Adhikari A, Gray KE, et al · · 2016 · cited 58× · PMID 27063958 · DOI 10.1016/j.trsl.2016.03.008
6. Innate immune cells, major protagonists of sickle cell disease pathophysiology.
   Allali S, Maciel TT, Hermine O, de Montalembert M. · · 2020 · cited 49× · PMID 31919091 · DOI 10.3324/haematol.2019.229989
7. Ischemia-Reperfusion Injury in Sickle Cell Disease: From Basics to Therapeutics.
   Ansari J, Gavins FNE. · · 2019 · cited 49× · PMID 30904156 · DOI 10.1016/j.ajpath.2018.12.012
8. Drug Therapies for the Management of Sickle Cell Disease.
   Rai P, Ataga KI. · · 2020 · cited 32× · PMID 32765834 · DOI 10.12688/f1000research.22433.1