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Minipooled-IVIG in Primary Immunodeficiency Disease

10. mai 2020 oppdatert av: 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

  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).

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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.

Studietype

Intervensjonell

Registrering (Faktiske)

15

Fase

  • Ikke aktuelt

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Egypt
      • Assiut, Egypt
        • Faculty of Medicine

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

Ikke eldre enn 18 år (Barn, Voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

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

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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.
Annen: 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.

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Efficacy of MP-IVIG assessed by the incidence of acute Serious Bacterial infections(SBIs)
Tidsramme: 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.
1 year
Safty of MP-IVIG assessed by percentage of adverse Events
Tidsramme: 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.
72 hour after adminstration of MP-IVIG and betwen infusions period
Study the pharmacokinetics- MP-IVIG trough levels
Tidsramme: predose sample

MP-IVIG trough level concentration values of serum total IgG pre the MP-IVIG infusion

(if applicable).
predose sample
Study the pharmacokinetics MP-IVIG plasma concentration -time curve
Tidsramme: (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
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
Study the pharmacokinetics MP-IVIG half-life
Tidsramme: (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
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
Study the pharmacokinetics MP-IVIG area under the curve
Tidsramme: (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
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
Study the pharmacokinetics MP-IVIG Cmax
Tidsramme: (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
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
Study the pharmacokinetics of MP-IVIG-Tmax.
Tidsramme: (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
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose
Study the pharmacokinetics of MP-IVIG elimination rate constant(s).
Tidsramme: (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
(1 hour, 2 hours and 1, 2, 3, 7, 14 and 21 days) post-dose

Sekundære resultatmål

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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Assiut University

Etterforskere

  • Studieleder: Maha A Mohammed, professor, Assiut University

Publikasjoner og nyttige lenker

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Generelle publikasjoner

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Studierekorddatoer

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Studer hoveddatoer

Studiestart (Faktiske)

1. januar 2020

Primær fullføring (Faktiske)

1. mai 2020

Studiet fullført (Faktiske)

1. mai 2020

Datoer for studieregistrering

Først innsendt

26. mars 2019

Først innsendt som oppfylte QC-kriteriene

28. mars 2019

Først lagt ut (Faktiske)

1. april 2019

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

12. mai 2020

Siste oppdatering sendt inn som oppfylte QC-kriteriene

10. mai 2020

Sist bekreftet

1. mai 2020

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • IVIG in PID

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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