Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases

NCT04640142 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: NORM-01
• Study Type: interventional
• Target: 50
• Locations: 7 countries
• Sites: 24

Brief Summary

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

Conditions

Primary Immune Deficiency

Outcome Measures

Primary Outcomes (2)

• Rate of Serious Bacterial Infections

  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

  Average total IgG concentration (Cav) on steadystate dosing.

  16 weeks

Secondary Outcomes (10)

• Infections of any type of seriousness

  52 weeks

• Time to resolution of infections

  52 weeks

• Use of antibiotics

  52 weeks

• Hospitalisations due to infection

  52 weeks

• Episodes of fever

  52 weeks

Study Arms (1)

Newnorm

EXPERIMENTAL

Newnorm is a 20% human normal immunoglobulin for SC infusion

Biological: Newnorm

Interventions

Newnorm BIOLOGICAL

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

Newnorm

Eligibility Criteria

• Age: 2 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age of ≥2 years and ≤75 years
• 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.
• 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.
• 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.
• 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.
• Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.

You may not qualify if:

• 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.
• The patient has isolated specific antibody deficiency disorder, isolated IgG subclass deficiency, or transient hypogammaglobulinaemia of infancy.
• 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).
• Known history of ADRs to IgA contained in other products.
• Body mass index \>40 kg/m2.
• Exposure to blood or any blood product or plasma derivative other than IgG for PID within 3 months before the first infusion of Newnorm.
• 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).
• Severe liver dysfunction (alanine aminotransferase \[ALT\] \>3 times the upper limit of normal for the expected normal range for the testing laboratory) at screening.
• Known protein-losing enteropathies or proteinuria (known urinary protein loss of \>1 g/24 h, or dipstick proteinuria of ≥3+).
• 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).
• Uncontrolled diabetes mellitus (HbA1c \> 7% / \>53 mmol/mol).
• 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).
• 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.
• 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 \[e.g. fondaparinux\]).
• Treatment with oral or parenteral steroids either

Sponsors & Collaborators

• Octapharma: lead

Study Sites (24)

Octapharma Research Site

Irvine, California, 92697, United States

Location

Octapharma Research Site

Centennial, Colorado, 80112, United States

Location

Octapharma Research Site

Hollywood, Florida, 33021, United States

Location

Octapharma Research Site

Port Saint Lucie, Florida, 34986, United States

Location

Octapharma Research Site

St. Petersburg, Florida, 33701, United States

Location

Octapharma Research Site

Chicago, Illinois, 60612, United States

Location

Octapharma Research Site

Overland Park, Kansas, 66211, United States

Location

Octapharma Research Site

Louisville, Kentucky, 40217, United States

Location

Octapharma Research Site

White Marsh, Maryland, 21162, United States

Location

Octapharma Research Site

Kansas City, Missouri, 64111, United States

Location

Octapharma Research Site

Omaha, Nebraska, 68046, United States

Location

Octapharma Research Site

Leipzig, 04129, Germany

Location

Octapharma Research Site

Munich, 80337, Germany

Location

Octapharma Research Site

Budapest, 1097, Hungary

Location

Octapharma Research Site

Debrecen, 4032, Hungary

Location

Octapharma Research Site

Napoli, 80131, Italy

Location

Octapharma Research Site

Roma, 00161, Italy

Location

Octapharma Research Site

Roma, 133, Italy

Location

Octapharma Research Site

Treviso, 31100, Italy

Location

Octapharma Research Site

Krakow, 30-663, Poland

Location

Octapharma Research Site

Bratislava, 833 40, Slovakia

Location

Octapharma Research Site

Kyiv, 04209, Ukraine

Location

Octapharma Research Site

Lviv, 79010, Ukraine

Location

Octapharma Research Site

Lviv, 79035, Ukraine

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2020
• First Posted: November 23, 2020
• Study Start: August 4, 2021
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: October 3, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (11); DE (2); HU (2); IT (4); SL (1); UA (3); PL (1)