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Studie ter evaluatie van de farmacokinetiek, werkzaamheid, verdraagbaarheid en veiligheid van subcutaan humaan immunoglobuline (Newnorm) bij patiënten met primaire immunodeficiëntieziekten

5 november 2026 bijgewerkt door: Octapharma

Prospectieve, open-label, eenarmige, multicentrische fase 3-studie ter evaluatie van de farmacokinetiek, werkzaamheid, verdraagbaarheid en veiligheid van subcutaan humaan immunoglobuline (Newnorm) bij patiënten met primaire immunodeficiëntieziekten

Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases

Studie Overzicht

Toestand

Actief, niet wervend

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

50

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • Duitsland
      • Leipzig, Duitsland, 04129
        • Octapharma Research Site
      • Munich, Duitsland, 80337
        • Octapharma Research Site
  • Hongarije
      • Budapest, Hongarije, 1097
        • Octapharma Research Site
      • Debrecen, Hongarije, 4032
        • Octapharma Research Site
  • Italië
      • Naples, Italië, 80131
        • Octapharma Research Site
      • Roma, Italië, 00161
        • Octapharma Research Site
      • Roma, Italië, 133
        • Octapharma Research Site
      • Treviso, Italië, 31100
        • Octapharma Research Site
  • Oekraïne
      • Kyiv, Oekraïne, 04209
        • Octapharma Research Site
      • Lviv, Oekraïne, 79010
        • Octapharma Research Site
      • Lviv, Oekraïne, 79035
        • Octapharma Research Site
  • Polen
      • Krakow, Polen, 30-663
        • Octapharma Research Site
  • Slowakije
      • Bratislava, Slowakije, 833 40
        • Octapharma Research Site
  • Verenigde Staten
    • California
      • Irvine, California, Verenigde Staten, 92697
        • Octapharma Research Site
    • Colorado
      • Centennial, Colorado, Verenigde Staten, 80112
        • Octapharma Research Site
    • Florida
      • Port Saint Lucie, Florida, Verenigde Staten, 34986
        • Octapharma Research Site
      • St. Petersburg, Florida, Verenigde Staten, 33701
        • Octapharma Research Site
    • Illinois
      • Chicago, Illinois, Verenigde Staten, 60612
        • Octapharma Research Site
    • Kansas
      • Overland Park, Kansas, Verenigde Staten, 66211
        • Octapharma Research Site
    • Kentucky
      • Louisville, Kentucky, Verenigde Staten, 40217
        • Octapharma Research Site
    • Maryland
      • White Marsh, Maryland, Verenigde Staten, 21162
        • Octapharma Research Site
    • Missouri
      • Kansas City, Missouri, Verenigde Staten, 64111
        • Octapharma Research Site
    • Nebraska
      • Omaha, Nebraska, Verenigde Staten, 68046
        • Octapharma Research Site

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

2 jaar tot 75 jaar (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria:

  1. Age of ≥2 years and ≤75 years
  2. Documented and confirmed diagnosis of PID as defined by European Society of Immunodeficiencies (ESID) and the Pan American Group for Immune Deficiency (PAGID) and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The exact type of PID must be recorded.
  3. At least 12 weeks of regular treatment before the screening visit (i.e., with a stable dosing interval) with any IVIG, SCIG, or fSCIG, with a stable IgG dose between 200 and 800 mg/kg/month. A stable dose is defined as one that deviates less than ±25% from the mean dose for all infusions within this 12-week period before screening.
  4. Trough level of IgG ≥5 g/L at screening and documentation of an IgG trough level of ≥5 g/L at least once within the previous 12 weeks.
  5. Freely given written informed consent from adult patients or freely given written informed consent from the patient's parent(s)/legal guardian(s) and written informed assent from paediatric or adolescent patients in accordance with the applicable regulatory requirements, before any study-specific procedure takes place.
  6. Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.

Exclusion Criteria:

  1. Any acute infection requiring IV antibiotic treatment within 2 weeks before the screening visit or during the screening period, or any SBI within the 3 months prior to the screening visit or during the screening period.

  2. The patient has isolated specific antibody deficiency disorder, isolated IgG subclass deficiency, or transient hypogammaglobulinaemia of infancy.

  3. Current medical condition or history of condition known to cause secondary immune deficiency, for example, chronic lymphocytic leukaemia, lymphoma, multiple myeloma, or chronic or recurrent neutropenia (absolute neutrophil count <1000/μL).

  4. Known history of ADRs to IgA contained in other products.

  5. Body mass index >40 kg/m2.

  6. Exposure to blood or any blood product or plasma derivative other than IgG for PID within 3 months before the first infusion of Newnorm.

  7. History of or ongoing severe hypersensitivity, e.g., anaphylaxis or severe systemic response, or persistent reactions to blood or plasma-derived products, or to any component of Newnorm (such as glycine).

  8. Severe liver dysfunction (alanine aminotransferase [ALT] >3 times the upper limit of normal for the expected normal range for the testing laboratory) at screening.

  9. Known protein-losing enteropathies or proteinuria (known urinary protein loss of >1 g/24 h, or dipstick proteinuria of ≥3+).

  10. Moderate to severe renal dysfunction (per investigator discretion based on estimated glomerular filtration rate [eGFR] ≤44 mL/min/1.73 m2, as defined by KDIGO Clinical Practice Guideline) or predisposition to acute renal failure (e.g., any degree of pre-existing renal dysfunction in presence of additional acute renal failure risk factors, e.g. routine treatment with known nephrotoxic drugs).

  11. Uncontrolled diabetes mellitus (HbA1c > 7% / >53 mmol/mol).

  12. Uncontrolled arterial hypertension (systolic blood pressure of ≥ 130 mmHg for the subject under 13 years of age, ≥ 140 mmHg for subject 13 to 17 years of age, and > 160 mmHg for adults).

  13. Dysrhythmia/Tachycardia (resting heart rate > 100 bpm for adults/adolescents and > 120 bpm for children) and symptomatic bradycardia (resting heart rate < 60 bpm for adults, < 50 bpm for adolescents, and < 75 bpm for children in presence of symptoms e.g., low blood pressure, abnormal rhythm, chest discomfort, shortness of breath). Physiological sinus bradycardia in physically active adults/children/athletes is NOT an exclusion criterion).

  14. The subject has a history of or current diagnosis of deep venous thrombosis or thromboembolism (e.g. myocardial infarction, cerebrovascular accident, or transient ischemic attack); history refers to an incident in the year prior to screening or 2 episodes over lifetime.

  15. The subject is currently receiving anti-coagulation therapy which would make SC administration inadvisable (vitamin K antagonist, nonvitamin K antagonist oral anticoagulants [e.g. dabigatran etexilate targeting Factor IIa, rivaroxaban, edoxaban, and apixaban targeting Factor Xa], parenteral anticoagulants [e.g. fondaparinux]).

  16. Treatment with oral or parenteral steroids either

    1. at daily doses >0.3 mg/kg of prednisone (or equivalent) within the last 12 weeks before screening or
    2. bolus treatment of a daily dose greater than 1 mg/kg of prednisone (or equivalent) for longer than 10 days within the last 12 weeks before screening. Courses of corticosteroids (intermittent) of not more than 10 days would not exclude a patient. Inhaled or topical corticosteroids are allowed.

  17. Treatment with systemic immunosuppressants including chemotherapeutic agents 1 year before screening or immunomodulatory drugs 12 weeks before the screening visit.

  18. Live viral vaccination (such as measles, rubella, mumps, or varicella) within 1 month before the first infusion of Newnorm, during the study period, and within 3 months after last infusion of Newnorm. Note: Seasonal inactivated (killed) influenza vaccines (incl. H1N1) are allowed. COVID vaccines (mRNA vaccine and a non-replicating viral vector vaccine) are allowed.

  19. Treatment with any investigational medicinal product (IMP) within 3 months before the screening visit.

  20. Presence of any condition likely to interfere with the evaluation of Newnorm or with the compliant conduct of the study.

  21. Known or suspected abuse of alcohol, drugs, and/or psychotropic agents within 12 months before screening.

  22. Known human immunodeficiency virus (HIV)-1/2, hepatitis B virus (HBV), or hepatitis C virus (HCV) infection or positive for HIV-1/2, HBV, or HCV at screening.

  23. Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method (refer to protocol Section 7.4.10.b) while on study and for 30 days following the last dose of study drug.

  24. Men who are unwilling to use birth control to prevent pregnancy for the duration of the study (unless the female partner

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: NVT
  • Interventioneel model: Opdracht voor een enkele groep
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Newnorm

Newnorm is a 20% human normal immunoglobulin for SC infusion
Biologisch: Newnorm

Newnorm is a 20% human normal immunoglobulin for SC infusion.

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Rate of Serious Bacterial Infections
Tijdsspanne: 52 weeks

Rate of SBIs (defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess) per person-year on treatment.
52 weeks
Average total IgG concentration
Tijdsspanne: 16 weeks

Average total IgG concentration (Cav) on steadystate dosing.
16 weeks

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Infections of any type of seriousness
Tijdsspanne: 52 weeks

Infections of any type or seriousness (including acute sinusitis, exacerbation of chronic sinusitis, acute otitis media, acute bronchitis, infectious diarrhoea, etc) (total and by category, annual rate)
52 weeks
Time to resolution of infections
Tijdsspanne: 52 weeks

Time to resolution of infections
52 weeks
Use of antibiotics
Tijdsspanne: 52 weeks

Use of antibiotics (number of days on antibiotics and annual rate of treatment episodes) characterised as therapeutic or prophylactic use
52 weeks
Hospitalisations due to infection
Tijdsspanne: 52 weeks

Hospitalisations due to infection (number of days and annual rate)
52 weeks
Episodes of fever
Tijdsspanne: 52 weeks

Episodes of fever
52 weeks
Days lost from work, school, kindergarten, or day care due to infection
Tijdsspanne: 52 weeks

Days lost from work, school, kindergarten, or day care due to infection
52 weeks
Child Health Questionnaire-Parent Form
Tijdsspanne: 52 weeks

Child Health Questionnaire-Parent Form (CHQPF50) for patients <14 years
52 weeks
SF-36 Health Survey
Tijdsspanne: 52 weeks

SF-36 Health Survey for patients ≥ 14 of age
52 weeks
Non-compartmental PK analyses of all available PK profiles (IVIG and SCIG)
Tijdsspanne: 16 weeks

Total IgG, IgG subclasses (IgG1, IgG2, IgG3, and IgG4), and antigen specific antibodies against Haemophilus influenzae B (capsular polysaccharide), Streptococcus pneumoniae (serotypes 4, 6B, 9V, 14, 18C, 19F, 23F), cytomegalovirus (CMV), tetanus, and measles
16 weeks
IgG Levels
Tijdsspanne: 16 weeks

Trough and peak levels of total IgG and IgG subclasses for weekly Newnorm relative to 3- or 4-weekly IVIG dosing
16 weeks

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Octapharma

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

4 augustus 2021

Primaire voltooiing (Geschat)

1 november 2026

Studie voltooiing (Geschat)

1 november 2026

Studieregistratiedata

Eerst ingediend

17 november 2020

Eerst ingediend dat voldeed aan de QC-criteria

17 november 2020

Eerst geplaatst (Werkelijk)

23 november 2020

Updates van studierecords

Laatste update geplaatst (Werkelijk)

9 november 2026

Laatste update ingediend die voldeed aan QC-criteria

5 november 2026

Laatst geverifieerd

1 november 2026

Meer informatie

Termen gerelateerd aan deze studie

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • NORM-01

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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