Minipooled-IVIG in Primary Immunodeficiency Disease
10 de mayo de 2020 actualizado por: Alshaimaa Mokhtar Selim mohamed, Assiut University
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).
Descripción general del estudio
Estado
Terminado
Condiciones
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Intervencionista
Inscripción (Actual)
15
Fase
- No aplica
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Ubicaciones de estudio
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Assiut, Egipto
- Faculty of Medicine
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Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
No mayor que 18 años (Niño, Adulto)
Acepta Voluntarios Saludables
No
Géneros elegibles para el estudio
Todos
Descripción
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
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
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Experimental: 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.
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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.
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Efficacy of MP-IVIG assessed by the incidence of acute Serious Bacterial infections(SBIs)
Periodo de tiempo: 1 year
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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.
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1 year
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Safty of MP-IVIG assessed by percentage of adverse Events
Periodo de tiempo: 72 hour after adminstration of MP-IVIG and betwen infusions period
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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.
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72 hour after adminstration of MP-IVIG and betwen infusions period
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Study the pharmacokinetics- MP-IVIG trough levels
Periodo de tiempo: predose sample
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MP-IVIG trough level concentration values of serum total IgG pre the MP-IVIG infusion (if applicable). |
predose sample
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Study the pharmacokinetics MP-IVIG plasma concentration -time curve
Periodo de tiempo: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Blood samples for analysis of pharmacokinetics MP-IVIG plasma concentration -time curve were obtained and analysed
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(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Study the pharmacokinetics MP-IVIG half-life
Periodo de tiempo: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Blood samples for analysis of pharmacokinetics MP-IVIG haf-life were obtained and analysed
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(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Study the pharmacokinetics MP-IVIG area under the curve
Periodo de tiempo: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Blood samples for analysis of pharmacokinetics MP-IVIG haf-life were obtained and analysed
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(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Study the pharmacokinetics MP-IVIG Cmax
Periodo de tiempo: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Blood samples for analysis of pharmacokinetics MP-IVIG Cmax were obtained and analysed
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(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Study the pharmacokinetics of MP-IVIG-Tmax.
Periodo de tiempo: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Blood samples for analysis of pharmacokinetics MP-IVIG Tmax were obtained and analysed
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(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Study the pharmacokinetics of MP-IVIG elimination rate constant(s).
Periodo de tiempo: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Blood samples for analysis of pharmacokinetics MP-IVIG elimination rate constant(s) were obtained and analysed
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(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Compare efficacy of MP-IVIG vs standard IVIG by compare incidence of SBIs of both
Periodo de tiempo: 1 year
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• Compare the efficacy of MP-IVIG to standard IVIG in children with Primary immunodeficiency disease (PID).
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1 year
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Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Investigadores
- Director de estudio: Maha A Mohammed, professor, Assiut University
Publicaciones y enlaces útiles
La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.
Publicaciones Generales
- 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.
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Actual)
1 de enero de 2020
Finalización primaria (Actual)
1 de mayo de 2020
Finalización del estudio (Actual)
1 de mayo de 2020
Fechas de registro del estudio
Enviado por primera vez
26 de marzo de 2019
Primero enviado que cumplió con los criterios de control de calidad
28 de marzo de 2019
Publicado por primera vez (Actual)
1 de abril de 2019
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
12 de mayo de 2020
Última actualización enviada que cumplió con los criterios de control de calidad
10 de mayo de 2020
Última verificación
1 de mayo de 2020
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Enfermedades del sistema inmunológico
- Enfermedades Genéticas Congénitas
- Síndromes de deficiencia inmunológica
- Enfermedades de inmunodeficiencia primaria
- Efectos fisiológicos de las drogas
- Factores inmunológicos
- Anticuerpos
- Inmunoglobulinas
- Inmunoglobulinas, Intravenosas
- gamma-globulinas
- Inmunoglobulina Rho(D)
Otros números de identificación del estudio
- IVIG in PID
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
No
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
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