IV Gammaglobulin for Sickle Cell Pain Crises
Recruiting in Palo Alto (17 mi)
Overseen byDeepa G Manwani, M.D
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: Deepa Manwani
Must be taking: Voxelotor
Must not be taking: Estrogen, Illicit drugs
Disqualifiers: Renal dysfunction, Stroke, Pregnancy, others
Approved in 4 Jurisdictions
Trial Summary
What is the purpose of this trial?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)
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but if you are prescribed Voxelotor, you must either continue using it consistently or not have used it in the past week.
What data supports the effectiveness of the drug IV Gammaglobulin for Sickle Cell Pain Crises?
Intravenous gammaglobulin (IVIG) has been shown to be effective in improving immune function in patients with primary immunodeficiency diseases and preventing the progression of HIV-related conditions. It contains antibodies that help fight infections, which might suggest potential benefits in managing sickle cell pain crises, although direct evidence for this specific use is not provided.
12345Is IV Gammaglobulin generally safe for humans?
IV Gammaglobulin, also known as IVIG, is generally considered safe for humans, but it can cause mild side effects like headaches, fever, and nausea. Rarely, it may lead to more serious issues like kidney problems or blood clots, especially in people with certain risk factors. Ensuring patients are well-hydrated and using a slow infusion rate can help minimize these risks.
26789How does the drug IV Gammaglobulin differ from other treatments for sickle cell pain crises?
IV Gammaglobulin (IVIG) is unique because it is administered intravenously (through a vein) and is primarily used to boost the immune system in various conditions, including immunodeficiencies and autoimmune diseases. Unlike typical pain management for sickle cell crises, which often involves painkillers, IVIG may help by modulating immune responses, potentially offering a novel approach to managing the condition.
1351011Eligibility Criteria
This trial is for individuals aged 12-65 with sickle cell disease experiencing a pain crisis needing hospital care. It's open to those not on chronic transfusion or at high stroke risk, without recent live vaccines, illicit drug abuse history, certain blood levels (Hb >10 g/dL or <5 g/dL), pregnancy, thrombosis risks like estrogen use, suspected infections, kidney issues, other drug trials participation, IgA deficiency or gamma globulin allergies.Inclusion Criteria
I have been diagnosed with sickle cell disease.
I was hospitalized for severe pain that needed strong painkillers given through an IV.
I am between 12 and 65 years old for Phase 1, or between 6 and 13.99 years old for Phase 2.
Exclusion Criteria
I have not received a live vaccine in the last 6 weeks.
I have had blood clots before or am currently using estrogen.
I currently have a fever over 101.3°F possibly due to an infection.
+10 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive a single dose of IVIG or normal saline placebo during an uncomplicated pain crisis
Average 4 days, maximum 30 days
1 visit (in-person)
Follow-up
Participants are monitored for safety and effectiveness after treatment, including changes in Mac-1, LDH, Hb, and hsCRP levels
24 hours post-infusion
Extended Monitoring
Participants are monitored for length of hospitalization and rate of transfer to ICU
Average 4 days, maximum 30 days
Participant Groups
The study tests if intravenous immune globulin can safely and effectively treat acute pain crises in sickle cell disease compared to normal saline. Participants are hospitalized and receive either the immune therapy or a saline solution as part of the treatment process.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Intravenous Immune Globulin (IVIG)Experimental Treatment1 Intervention
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.
Group II: Normal salinePlacebo Group1 Intervention
An equivalent volume (weight-based) of normal saline
Immune Globulin Intravenous is already approved in European Union, United States, Canada, Japan for the following indications:
🇪🇺 Approved in European Union as Immune Globulin Intravenous for:
- Primary immunodeficiency
- Immune thrombocytopenic purpura
- Chronic inflammatory demyelinating polyneuropathy
- Kawasaki disease
- Guillain–Barré syndrome
🇺🇸 Approved in United States as IVIG for:
- Primary immunodeficiency
- Immune thrombocytopenic purpura
- Chronic inflammatory demyelinating polyneuropathy
- Multifocal motor neuropathy
🇨🇦 Approved in Canada as Human Normal Immunoglobulin for:
- Primary immunodeficiency
- Immune thrombocytopenic purpura
- Chronic inflammatory demyelinating polyneuropathy
🇯🇵 Approved in Japan as Gammaglobulin for:
- Primary immunodeficiency
- Immune thrombocytopenic purpura
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Montefiore Medical CenterBronx, NY
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Who Is Running the Clinical Trial?
Deepa ManwaniLead Sponsor
Albert Einstein College of MedicineLead Sponsor
Food and Drug Administration (FDA)Collaborator
Case Western Reserve UniversityCollaborator
Grifols Therapeutics LLCIndustry Sponsor
References
High dose intact-immunoglobulin treatment for an HIV-infected asymptomatic carrier with hemophilia. [2019]Intravenous administration of a high dose of intact-immunoglobulin to a human immunodeficiency virus (HIV) infected patient with hemophilia A, whose total lymphocyte and CD4 positive lymphocyte counts showed a decreasing tendency, resulted in a gradual rise in these cell counts. The results were quite reproducible on another occasions. Therefore, intravenous administration of gammaglobulin (IVGG) was thought to be effective for preventing further deterioration of cellular immunity and progression from asymptomatic carrier (AC) to AIDS related complex (ARC)/AIDS in hemophilia patients.
Arthritis associated with circulating immune complexes following administration of intravenous immunoglobulin therapy in a patient with chronic inflammatory demyelinating polyneuropathy. [2019]Intravenous immunoglobulin is viewed as a relatively safe treatment for several neurologic disorders. I report arthritis associated with elevated circulating immune complexes and abnormalities of serum complement components which is a rare complication with modern preparations of immunoglobulin-gamma.
[Treatment of primary immunodeficiencies with intravenous gamma globulin]. [2006]Thirty five patients affected by primary immunodeficiency diseases were treated with a polyethylenglicol treated intravenous gammaglobulin. Number of infusions administered during a period of 665 months/patients, was 110. In those cases previously treated with intramuscular gammaglobulin, serum IgG levels were higher after intravenous gammaglobulin administration. Therapeutic response was favourable in most cases. Intravenous gammaglobulin was well tolerated, noticing only two serious and two mild adverse reactions. Patients that suffered adverse reactions with intramuscular gammaglobulin tolerated well intravenous preparation used, except for one patient that after two years in treatment developed IgE mediated antibodies against IgA. Need of an individualized dosage is emphasized.
Intravenous immunoglobulin in oncology nursing practice. [2013]Intravenous immunoglobulin (IVIG) is a concentrated form of IgG, also known as gamma globulin, that is derived from the pooled serum of a large number of donors. IVIG contains many types of antibacterial and antiviral antibodies. While its use in certain clinical conditions (e.g., severe combined immunodeficiency) is well-established, other indications still are under investigation. Along with nursing implications for use in inpatient and outpatient settings, the role of IVIG in treating immune thrombocytopenic purpura, chronic lymphocytic leukemia, treatment-induced neutropenia and thrombocytopenia, bone marrow transplantation, and AIDS will be discussed.
[Chronic kidney disease in adults with primary immunodeficiency diseases in treatment with intravenous immunoglobulin]. [2021]Intravenous immunoglobulin (IVIG) is the treatment of choice for humoral primary immunodeficiency diseases (PIDs). A third of the patients who receive intravenous immunoglobulin have adverse reactions, such as osmotic nephrosis.
Intravenous immunoglobulin therapy: a snapshot for the internist. [2022]Intravenous immunoglobulins are the cornerstone for the treatment of primary humoral immunodeficiencies and may be used for a great number of other autoimmune, neurological and hematological conditions as well. Given their wide application, the possibility of running across a patient who needs this kind of therapy is becoming increasingly common. Generally, intravenous immunoglobulins are well tolerated. However, numerous adverse reactions ranging from mild to severe have been reported and linked to patient- and product-related factors. For all these reasons, we present herein a comprehensive review of the on- and off-label applications of intravenous immunoglobulins and provide a guide for the internist how to minimize the risk of adverse reactions and manage them.
Safety of intravenous immunoglobulin (IVIG) therapy. [2022]Intravenous immunoglobulin (IVIg) is administered both for the treatment of immunodeficiencies and for an expanding list of autoimmune diseases. Most adverse effects are mild and transient including headaches, flushing, fever, chills, fatigue, nausea, diarrhea, blood pressure changes and tachycardia. IgA deficiency-related anaphylactic reactions are largely preventable. Late adverse events are rare and include acute renal failure and thromboembolic events. Acute renal failure, usually oliguric and transient, occurs generally in insufficiently hydrated patients and with sucrose-stabilized products due to osmotic injury. Thromboembolic complications occur due to hyperviscosity especially in patients having risk factors including advanced age, previous thromboembolic events, immobilization, diabetes mellitus, hypertension, dyslipidemia or those receiving high-dose IVIg in a rapid infusion rate or excessive dose. Slow infusion rate and good hydration may prevent renal failure, thromboembolic events and aseptic meningitis. In our experience in more than 200 patients receiving IVIg for different autoimmune diseases and near 10000 infusions for relapsing-remitting multiple sclerosis patients, the occurrence of adverse effects was 24-36% after high dose IVIg, most were headaches and all were mild adverse events. We conclude that IVIg is a safe therapy when given in a slow infusion rate in well-hydrated patients, better avoiding patients with known risk factors.
Intravenous immunoglobulin G use and pharmacovigilance in a tertiary care children's hospital. [2021]Intravenous immunoglobulin G (IVIG) is a blood product from polyvalent and polyclonal immunoglobulin G. It covers a broad range of indications as immunomodulator or replacement therapy. In addition, although it is considered a safe therapy, the incidence of adverse reactions reported in the bibliography ranges from 1 % to 81 %. The objective of this study was to assess IVIG use and describe related adverse events in a tertiary care children's hospital.
Human immunoglobulin (KIOVIG®/GAMMAGARD LIQUID®) for immunodeficiency and autoimmune diseases: an observational cohort study. [2022]To document the therapeutic efficacy and safety of Human Normal Immunoglobulin 10% Liquid (KIOVIG(®)/GAMMAGARD LIQUID(®) [IVIG 10%]) under clinical routine conditions.
Haemolysis after treatment with intravenous immunoglobulin due to anti-A. [2011]Intravenous immunoglobulin (IVIG) is used to treat an increasing number of conditions. IVIG contains immunoglobulin G (IgG) directed against many targets, including red blood cell (RBC) antigens.
Historic aspects of intravenous immunoglobulin therapy. [2019]The earliest preparations of immunoglobulins (Ig) decreased the susceptibility of agammaglobulinemic patients to infections caused by pneumococci, Haemophilus influenzae, meningococci, streptococci, and Pseudomonas aeruginosa. Intramuscular administration of such preparations was painful and traumatic, especially for children. Ethanol-fractionated Ig could not be administered intravenously (IV) because the IgG molecules tended to aggregate and thus were more likely to produce anaphylactoid reactions. New Ig preparations, isolated at low pH (e.g., pH 4) in the presence of traces of pepsin to inhibit reaggregation, were well tolerated when administered IV. Thus a new era of treatment and prophylaxis of disease using IV Ig (IVIG) was launched. The IVIG preparations revolutionized the management of virtually all immunodeficiency syndromes characterized by failure of antibody responses. Amelioration of antibody deficiency secondary to certain chronic diseases or surgical trauma can be achieved with these preparations. Newer uses of IVIG include treatment of some autoimmune diseases; in some conditions, the beneficial influences may be attributable to antiidiotype antibodies present in the IVIG. Another likely explanation is that IVIG inhibits damage to cells and tissues by antibody-mediated cellular cytotoxicity or blocks phagocytosis that is facilitated by Fc receptor mechanisms. The value of IVIG in preventing infection in patients undergoing bone marrow or organ transplantation and in the treatment and prophylaxis of life-threatening infections in neonates and premature infants also is reviewed.