Minipooled-IVIG in Primary Immunodeficiency Disease

• Sponsor: Assiut University (Other)
• Overall Status: Completed
• CT.gov ID: NCT03896932
• Collaborator: (none)
• Enrollment: 15
• Location: 1
• Arm: 1
• Actual Duration (Months): 4
• Patients Per Site Per Month: 3.8

Study Details

Study Description

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

1. Compare the efficacy of MP-IVIG to standard IVIG in children with primary immunodeficiency (PID).

Condition or Disease	Intervention/Treatment	Phase
Primary Immunodeficiency	Other: minipooled- Intravenous immunoglobulin(MP-IVIG)	N/A

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 Design

• Study Type: Interventional
• Actual Enrollment: 15 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention Model Description: Single Group AssignmentSingle Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Jan 1, 2020
• Actual Primary Completion Date: May 1, 2020
• Actual Study Completion Date: May 1, 2020

Arms and Interventions

Arm	Intervention/Treatment
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.	Other: minipooled- Intravenous immunoglobulin(MP-IVIG); 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.

Outcome Measures

Primary Outcome Measures

1. 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.

2. 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.

3. 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).

4. 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

5. 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

6. 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

7. 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

8. 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

9. 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

1. 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).

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 18 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Faculty of Medicine	Assiut		Egypt

Sponsors and Collaborators

• Assiut University

Investigators

• Study Director: Maha A Mohammed, professor, Assiut University

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Gathmann B, Grimbacher B, Beauté J, Dudoit Y, Mahlaoui N, Fischer A(2009):. ESID Registry Working Party. The European internet based patient and research database for primary immunodeficiency: results 2006-2008. Clin Exp Immuno.

Publications

• Boyle JM, Buckley RH. Population prevalence of diagnosed primary immunodeficiency diseases in the United States. J Clin Immunol. 2007 Sep;27(5):497-502. Epub 2007 Jun 19.
• El-Ekiaby M, Sayed MA, Caron C, Burnouf S, El-Sharkawy N, Goubran H, Radosevich M, Goudemand J, Blum D, de Melo L, Soulié 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.
• Piguet D, Tosi C, Lüthi 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.
• 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.

• Responsible Party: Alshaimaa Mokhtar Selim mohamed, Principal investigator, Assiut University
• ClinicalTrials.gov Identifier: NCT03896932
• Other Study ID Numbers: IVIG in PID
• First Posted: Apr 1, 2019
• Last Update Posted: May 12, 2020
• Last Verified: May 1, 2020

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes; Immunoglobulins; Immunoglobulins, Intravenous; Antibodies; gamma-Globulins; Rho(D) Immune Globulin

Study Results

No Results Posted as of May 12, 2020