Minipooled-IVIG in Primary Immunodeficiency Disease
Study of Safety and Efficacy of Mini-pool Intravenous Immunoglobulin (MP-IVIG) Prepared by Assiut University Hospital Blood Bank in Primary Immunodeficiency Patients
- study the pharmacokinetics of mini-pooled intravenous immunoglobulin( MP-IVIG)
Study the safety and efficacy of a newly developed preparation of MP-IVIG in children with primary immunodeficiency (PID) :
- Adverse reaction of MP-IVIG(anaphylaxis and haemolysis)( no or mild or moderate)
- Prevention of severe bacterial infection
- Improvement of general health(weight gain and mentality)
- Integration in to social live
- Compare the efficacy of MP-IVIG to standard IVIG in children with primary immunodeficiency (PID).
Přehled studie
Postavení
Podmínky
Intervence / Léčba
Detailní popis
Primary immunodeficiency diseases (PID) are a heterogeneous group of inherited disorders of the immune system, predisposing individuals to recurrent infections, allergy, autoimmunity, and malignancies. Clinical descriptions have already been made for more than 200 PIDs, for which over 150 forms of PID have been molecularly characterized .
A population prevalence of diagnosed PID in the United States at approximately 1 in 1,200 persons.
A part from local registration in some centres there is no national registry of PID in Egypt, and hence, the prevalence of these disorders in the investigator's population is still unknown .
An increasing number of PID are recognized, and effective treatments are possible. Early use of prophylactic antibiotics and replacement immunoglobulin can prevent significant end organ damage and improve long quality of life in these patients .
Immunoglobulin G (IgG) is an essential plasma derived medicine that is lacking in developing countries .IgG shortages leave immune deficient patients without treatment, exposing them to devastating recurrent infections from local pathogens. A simple and practical method for producing IgG from normal plasma collected in developing countries is needed to provide better, faster access to IgG for patients .
Magdy EL-Ekiaby, et al 2010 introduce the concept of small-scale ("minipool") plasma processing methods implementable with minimum infrastructural requirements. They developed viral inactivation and protein purification technologies in single-use equipment to prepare virally safe solvent/detergent-filtered (S/D-F) plasma Producing a 90%pure immunoglobulin fraction in disposable single-use devices for transfusion as well as minipool S/D-F cryoprecipitate to treat bleeding disorders.
Typ studie
Zápis (Aktuální)
Fáze
- Nelze použít
Kontakty a umístění
Studijní místa
-
-
-
Assiut, Egypt
- Faculty of Medicine
-
-
Kritéria účasti
Kritéria způsobilosti
Věk způsobilý ke studiu
Přijímá zdravé dobrovolníky
Pohlaví způsobilá ke studiu
Popis
Inclusion Criteria:
- Age group: children patients under 18 years.
- The study will include patient diagnosed as primary immunodeficiency disease (PID) in Assiut university hospital on standard IVIG therapy.
Exclusion Criteria:
- Patient has SCID.
- Patient with history of severe IVIG side effect.
- Patient with severe immunodeficiency and has severe disseminated infection.
- Patient with renal impairment
- Patient with hepatic cell failure
- Patient with endocrinal abnormalities
- patient with secondary immunodeficiency diseases
Studijní plán
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: N/A
- Intervenční model: Přiřazení jedné skupiny
- Maskování: Žádné (otevřený štítek)
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
|---|---|
|
Experimentální: minipooled- Intravenous immunoglobulin(MP-IVIG)
• MP-IVIG equivalent to 1 g/ kg of standard IVIG over a 6-hour to 8-hour period monthly alternated by standard IVIG for a period of 12 months follow up and the newly diagnosed cases admitted to AUH in the follow up period will be included.
|
The process of MP-IVIG preparation will involve the use of caprylic acid for purification and virus inactivation of Igs from mini-pools of 20 plasma donations collected in our CBTS in AUH.
The equipment used for the process comprised disposable blood bags, hemodialyzers, and purification and microbial filters.
|
Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Efficacy of MP-IVIG assessed by the incidence of acute Serious Bacterial infections(SBIs)
Časové okno: 1 year
|
The rate of Acute SBIs for each participant per 1 year will be assessed by questionnaire (Serious Bacterial Infections) include sign and symptoms of acute serious bacterial infections, i.e. bacterial pneumonia, bacteremia/sepsis, bacterial meningitis, osteomyelitis/ septic arthritis, visceral abscess.
|
1 year
|
|
Safty of MP-IVIG assessed by percentage of adverse Events
Časové okno: 72 hour after adminstration of MP-IVIG and betwen infusions period
|
Overall percentage of adverse events as hemolysis and anaphylaxis headache and other complains that occur during 72 hours of following an infusion of MP-IVIG will be assessed by1) vital sign(pulse,blood pressure,Respiratory rate and temprature 2)Hemolysis by hemoglobin level,LDH,billirubin level.2)lbetwen
infusions by home diaries.
|
72 hour after adminstration of MP-IVIG and betwen infusions period
|
|
Study the pharmacokinetics- MP-IVIG trough levels
Časové okno: predose sample
|
MP-IVIG trough level concentration values of serum total IgG pre the MP-IVIG infusion (if applicable). |
predose sample
|
|
Study the pharmacokinetics MP-IVIG plasma concentration -time curve
Časové okno: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
Blood samples for analysis of pharmacokinetics MP-IVIG plasma concentration -time curve were obtained and analysed
|
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
|
Study the pharmacokinetics MP-IVIG half-life
Časové okno: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
Blood samples for analysis of pharmacokinetics MP-IVIG haf-life were obtained and analysed
|
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
|
Study the pharmacokinetics MP-IVIG area under the curve
Časové okno: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
Blood samples for analysis of pharmacokinetics MP-IVIG haf-life were obtained and analysed
|
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
|
Study the pharmacokinetics MP-IVIG Cmax
Časové okno: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
Blood samples for analysis of pharmacokinetics MP-IVIG Cmax were obtained and analysed
|
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
|
Study the pharmacokinetics of MP-IVIG-Tmax.
Časové okno: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
Blood samples for analysis of pharmacokinetics MP-IVIG Tmax were obtained and analysed
|
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
|
Study the pharmacokinetics of MP-IVIG elimination rate constant(s).
Časové okno: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
Blood samples for analysis of pharmacokinetics MP-IVIG elimination rate constant(s) were obtained and analysed
|
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
|
Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Compare efficacy of MP-IVIG vs standard IVIG by compare incidence of SBIs of both
Časové okno: 1 year
|
• Compare the efficacy of MP-IVIG to standard IVIG in children with Primary immunodeficiency disease (PID).
|
1 year
|
Spolupracovníci a vyšetřovatelé
Sponzor
Vyšetřovatelé
- Ředitel studie: Maha A Mohammed, professor, Assiut University
Publikace a užitečné odkazy
Obecné publikace
- El-Ekiaby M, Sayed MA, Caron C, Burnouf S, El-Sharkawy N, Goubran H, Radosevich M, Goudemand J, Blum D, de Melo L, Soulie V, Adam J, Burnouf T. Solvent-detergent filtered (S/D-F) fresh frozen plasma and cryoprecipitate minipools prepared in a newly designed integral disposable processing bag system. Transfus Med. 2010 Feb;20(1):48-61. doi: 10.1111/j.1365-3148.2009.00963.x. Epub 2009 Sep 23.
- Boyle JM, Buckley RH. Population prevalence of diagnosed primary immunodeficiency diseases in the United States. J Clin Immunol. 2007 Sep;27(5):497-502. doi: 10.1007/s10875-007-9103-1. Epub 2007 Jun 19.
- Reda SM, Afifi HM, Amine MM. Primary immunodeficiency diseases in Egyptian children: a single-center study. J Clin Immunol. 2009 May;29(3):343-51. doi: 10.1007/s10875-008-9260-x. Epub 2008 Nov 11.
- Piguet D, Tosi C, Luthi JM, Andresen I, Juge O; Study investigators. Redimune NF Liquid, a ready-to-use, high-concentration intravenous immunoglobulin therapy preparation, is safe and typically well tolerated in the routine clinical management of a broad range of conditions. Clin Exp Immunol. 2008 Apr;152(1):45-9. doi: 10.1111/j.1365-2249.2008.03597.x. Epub 2008 Jan 28.
Termíny studijních záznamů
Hlavní termíny studia
Začátek studia (Aktuální)
Primární dokončení (Aktuální)
Dokončení studie (Aktuální)
Termíny zápisu do studia
První předloženo
První předloženo, které splnilo kritéria kontroly kvality
První zveřejněno (Aktuální)
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Naposledy ověřeno
Více informací
Termíny související s touto studií
Další relevantní podmínky MeSH
Další identifikační čísla studie
- IVIG in PID
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Studuje produkt zařízení regulovaný americkým úřadem FDA
Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .
Klinické studie na minipooled- Intravenous immunoglobulin(MP-IVIG)
-
Assiut UniversityNeznámýGuillain-Barre syndrom
-
North American Consortium for HistiocytosisHistiocyte SocietyNáborHistiocytóza z Langerhansových buněkSpojené státy