Using intravenous gammaglobulin to treat pain crises in sickle cell disease
Phase 1-2 Trial of Gamunex (intravenous Gammaglobulin) for Sickle Cell Acute Pain
This study is testing if a treatment called intravenous gammaglobulin can help relieve pain during sickle cell disease crises for people of different ages.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 300 (estimated) |
| Ages | 6 Years to 65 Years |
| Sex | All |
| Sponsor | Albert Einstein College of Medicine Academic / other |
| Locations | 1 site (Bronx, New York) |
| Trial ID | NCT01757418 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the safety and effectiveness of intravenous immune globulin (IVIG) in treating acute pain crises associated with sickle cell disease. Participants will be randomly assigned to receive either a single dose of IVIG or a normal saline placebo during a vaso-occlusive crisis. The study will monitor the duration of the pain crisis and other secondary outcomes to assess the treatment's impact. The trial includes both Phase 1 and Phase 2 components, focusing on different age groups.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 6 to 65 with documented sickle cell disease experiencing uncomplicated acute pain crises.
Not a fit: Patients with acute complications such as acute chest syndrome or significant infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for managing pain crises in patients with sickle cell disease.
How similar studies have performed: While the use of IVIG in this context is novel, other studies have explored immune therapies for sickle cell disease with varying degrees of success.
Eligibility criteria
Show full inclusion / exclusion criteria
Each subject must fulfill each of the following Inclusion/Exclusion criteria at screening and continue to fulfill these criteria prior to dosing: Inclusion Criteria: * Documented 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) * Normal stroke risk as assessed by transcranial Doppler (TCD). A normal TCD in subjects 16 years of age and younger within the year prior to study drug administration are required * Uncomplicated acute vaso-occlusive crisis requiring hospital admission and parenteral narcotic analgesics * If prescribed Voxelotor: Consistent daily use of voxelotor in the past week AND able to continue Voxelotor inpatient OR no reported use in prior week Exclusion Criteria: * Concomitant acute process, including acute chest syndrome, potential serious infection, or clinically significant bleeding * Fever \> 38.5° C and clinical suspicion of infection * Serum alanine aminotransferase \>4x Upper Limit of Normal (ULN) * Serum creatinine ≥1.3 mg/dL (or \> than 95th percentile for age) or \>300 mg/dL protein in spot urinalysis * Known condition associated with renal dysfunction including but not limited to diabetes mellitus, uncontrolled hypertension, multiple myeloma, and congestive heart failure * Any clinical evidence of prior stroke * Prior thromboses or current estrogen use * Current estrogen use * Hb \< 5 g/dL or \> 10 g/dL * Known Immunoglobulin A (IgA) deficiency or known allergy to gamma globulin * Pregnancy or breastfeeding * Current participation in another investigational drug study * Current enrollment in a hypertransfusion program * Previous participation in current study less than 3 months ago * Current treatment with chronic transfusion * Vaccination with a live attenuated virus in the preceding 6 weeks * Documented history of illicit (e.g., heroin, cocaine) drug abuse * Subject is otherwise not an appropriate study candidate, in the investigator's judgement, such as concern for opioid addiction or comorbid psychiatric diagnoses that may contribute to secondary gain in prolonged use of opioids or hospital stay * Greater than 24 hours from time of presentation to the hospital for VOC * Atrial fibrillation * Right to left cardiac shunting due to patent foramen ovale or other anatomic cause * Known magnetic resonance imaging/angiography (MRI/A) evidence of stroke or clinically significant central nervous system (CNS) vasculopathy at any age (Imaging done if clinically indicated)
Where this trial is running
Bronx, New York
- Montefiore Medical Center — Bronx, New York, United States (Recruiting)
Study contacts
- Principal investigator: Deepa G Manwani, M.D — Albert Einstein College of Medicine
- Study coordinator: Deepa G Manwani, M.D
- Email: dmanwani@montefiore.org
- Phone: 718-741-2342
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.