Minipooled-IVIG in Primary Immunodeficiency Disease

Assiut University

Status: Completed

Conditions: Primary Immunodeficiency

Treatments: Other: minipooled- Intravenous immunoglobulin(MP-IVIG)

Study type: Interventional

Funder types: Other

Identifiers

NCT03896932

IVIG in PID

Details and patient eligibility

About

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).

Full 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.

Enrollment: 15 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

minipooled- Intravenous immunoglobulin(MP-IVIG)

Experimental group

Description:

• 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.

Treatment:

Other: minipooled- Intravenous immunoglobulin(MP-IVIG)