Clinical trial NCT03896932: Minipooled-IVIG in Primary Immunodeficiency Disease
Official title: Study of Safety and Efficacy of Mini-pool Intravenous Immunoglobulin (MP-IVIG) Prepared by Assiut University Hospital Blood Bank in Primary Immunodeficiency Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Primary Immunodeficiency
Lead sponsor: Assiut University
Sponsor class: OTHER
First posted: 2019-04-01
Enrollment: 15
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single Group Assignment
Primary purpose: TREATMENT

Brief summary: 1. study the pharmacokinetics of mini-pooled intravenous immunoglobulin( MP-IVIG)
2. 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
3. Compare the efficacy of MP-IVIG to standard IVIG in children with primary immunodeficiency (PID).

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Efficacy of MP-IVIG assessed by the incidence of acute Serious Bacterial infections(SBIs)
timeFrame: 1 year
measure: Safty of MP-IVIG assessed by percentage of adverse Events
timeFrame: 72 hour after adminstration of MP-IVIG and betwen infusions period
measure: Study the pharmacokinetics- MP-IVIG trough levels
timeFrame: predose sample
measure: Study the pharmacokinetics MP-IVIG plasma concentration -time curve
timeFrame: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
measure: Study the pharmacokinetics MP-IVIG half-life
timeFrame: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
measure: Study the pharmacokinetics MP-IVIG area under the curve
timeFrame: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
measure: Study the pharmacokinetics MP-IVIG Cmax
timeFrame: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
measure: Study the pharmacokinetics of MP-IVIG-Tmax.
timeFrame: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
measure: Study the pharmacokinetics of MP-IVIG elimination rate constant(s).
timeFrame: (1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
Secondary outcomes:
measure: Compare efficacy of MP-IVIG vs standard IVIG by compare incidence of SBIs of both
timeFrame: 1 year
Other outcomes:
Locations:
city: Assiut
country: Egypt
facility: Faculty of Medicine
Study officials:
affiliation: Assiut University
name: Maha A Mohammed, professor
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.