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Studie for å evaluere farmakokinetikken, effektiviteten, toleransen og sikkerheten til subkutan humant immunglobulin (Newnorm) hos pasienter med primære immunsviktsykdommer

5. november 2026 oppdatert av: Octapharma

Prospektiv, åpen, enkeltarm, multisenter fase 3-studie for å evaluere farmakokinetikken, effekten, toleransen og sikkerheten til subkutan humant immunoglobulin (Newnorm) hos pasienter med primære immunsviktsykdommer

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

Studieoversikt

Status

Aktiv, ikke rekrutterende

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Faktiske)

50

Fase

  • Fase 3

Kontakter og plasseringer

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

Studiesteder

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

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

2 år til 75 år (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

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

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: Newnorm

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

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

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Rate of Serious Bacterial Infections
Tidsramme: 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
Tidsramme: 16 weeks

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

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Infections of any type of seriousness
Tidsramme: 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
Tidsramme: 52 weeks

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

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

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

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

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

Samarbeidspartnere og etterforskere

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

Sponsor

Octapharma

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

4. august 2021

Primær fullføring (Antatt)

1. november 2026

Studiet fullført (Antatt)

1. november 2026

Datoer for studieregistrering

Først innsendt

17. november 2020

Først innsendt som oppfylte QC-kriteriene

17. november 2020

Først lagt ut (Faktiske)

23. november 2020

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

9. november 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

5. november 2026

Sist bekreftet

1. november 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • NORM-01

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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Abonnere