Minipooled-IVIG in Primary Immunodeficiency Disease

Not Applicable

Completed

• Conditions: Primary Immunodeficiency
• Interventions: Other: minipooled- Intravenous immunoglobulin(MP-IVIG)

• Registration Number: NCT03896932
• Lead Sponsor: Assiut University

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.

Study Timeline

• Study First Submitted: 2019-03-26
• Study First Submitted QC: 2019-03-28
• Study First Posted: 2019-04-01
• Study Start: 2020-01-01
• Status Verified: 2020-05
• Primary Completion: 2020-05-01
• Study Completion: 2020-05-01
• Last Update Submitted: 2020-05-10
• Last Update Posted: 2020-05-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
minipooled- Intravenous immunoglobulin(MP-IVIG)	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.

Primary Outcome Measures

Name	Time	Method
Study the pharmacokinetics MP-IVIG Cmax	(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
Efficacy of MP-IVIG assessed by the incidence of acute Serious Bacterial infections(SBIs)	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.
Study the pharmacokinetics- MP-IVIG trough levels	predose sample	MP-IVIG trough level concentration values of serum total IgG pre the MP-IVIG infusion; (if applicable).
Study the pharmacokinetics MP-IVIG plasma concentration -time curve	(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
Safty of MP-IVIG assessed by percentage of adverse Events	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.
Study the pharmacokinetics MP-IVIG half-life	(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
Study the pharmacokinetics MP-IVIG area under the curve	(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
Study the pharmacokinetics of MP-IVIG-Tmax.	(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
Study the pharmacokinetics of MP-IVIG elimination rate constant(s).	(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

Secondary Outcome Measures

Name	Time	Method
Compare efficacy of MP-IVIG vs standard IVIG by compare incidence of SBIs of both	1 year	• Compare the efficacy of MP-IVIG to standard IVIG in children with Primary immunodeficiency disease (PID).

Trial Locations

Locations (1)

Faculty of Medicine; 🇪🇬 Assiut, Egypt