Newnorm for Primary Immunodeficiency

Recruiting at 19 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Octapharma

Must be taking: Immunoglobulin therapy

Must not be taking: Anticoagulants, Immunosuppressants, Steroids

Disqualifiers: Acute infection, Cancer, Liver dysfunction, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial is testing a new antibody treatment called Newnorm, which is given through an injection under the skin. It aims to help people with primary immunodeficiency diseases, whose immune systems are weak. The treatment provides extra antibodies to help their bodies fight infections.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, certain medications like systemic immunosuppressants, high-dose steroids, and some anticoagulants may not be allowed. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the drug Newnorm for treating primary immunodeficiency?

Research shows that subcutaneous immunoglobulin treatments, like Newnorm, are commonly used and effective for managing primary immunodeficiency. Studies such as the FIGARO study and others have demonstrated their real-world effectiveness and safety in similar conditions. ^{1 2 3 4 5}

What makes the drug Newnorm unique for treating primary immunodeficiency?

Newnorm is unique because it is a subcutaneous (under the skin) form of human immunoglobulin G (IgG), which allows for more convenient administration compared to intravenous (into the vein) options, potentially improving patient comfort and adherence to treatment. ^{6 7 8 9 10}

Research Team

Eligibility Criteria

This trial is for people aged 2 to 75 with Primary Immunodeficiency Diseases (PID) who need immunoglobulin therapy. They must have been on stable treatment doses for at least 12 weeks and agree to protocol requirements, including blood sampling. Excluded are those with severe allergies to IgA, obesity (BMI >40), liver dysfunction, uncontrolled diabetes or hypertension, certain heart conditions, recent blood product exposure other than IgG for PID, or active infections.

Inclusion Criteria

Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.

My IgG levels have been above 5 g/L recently and at screening.

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.

Exclusion Criteria

Body mass index >40 kg/m2.

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.

I have a condition like leukemia or lymphoma that weakens my immune system.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous human immunoglobulin (Newnorm) to evaluate pharmacokinetics, efficacy, tolerability, and safety

52 weeks

Regular visits for treatment and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Newnorm (Immunoglobulin)

Trial Overview The study is testing 'Newnorm,' a subcutaneous human immunoglobulin treatment for patients with PID. It's an open-label Phase 3 trial assessing how the body processes the drug and its effectiveness and safety across multiple centers.

Participant Groups

Group I: Newnorm Experimental Treatment 1 Intervention

Newnorm is a 20% human normal immunoglobulin for SC infusion

Newnorm is already approved in Canada for the following indications:

Approved in Canada as Human Immunoglobulin for:

• Primary Immunodeficiency Diseases

Who Is Running the Clinical Trial?

Octapharma

Lead Sponsor

Trials

86

Recruited

11,300+

Wolfgang Marguerre

Octapharma

Chief Executive Officer since 1983

MBA from INSEAD

Wolfgang Frenzel

Octapharma

Chief Medical Officer since 2010

MD from University of Vienna

Findings from Research

The FIGARO study, involving 156 patients with primary or secondary immunodeficiency diseases, demonstrated that facilitated subcutaneous immunoglobulin (fSCIG) is feasible and well-tolerated, with most patients self-administering their treatment at home.

At the 12-month follow-up, 85.8% of patients were infusing at home and 88.2% were self-administering, with a low incidence of acute severe bacterial infections (0%-9.1%), indicating effective infection control and safety across different age groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Facilitated Subcutaneous Immunoglobulin Treatment in Patients with Immunodeficiencies: the FIGARO Study. Borte, M., Hanitsch, LG., Mahlaoui, N., et al.[2023]

In a real-world study of 85 patients receiving IgPro20 for hypogammaglobulinemia, 45.9% experienced adverse events, with injection site reactions being the most common, but the treatment was generally considered safe.

The effectiveness of IgPro20 was demonstrated by a reduction in infection rates from 0.54 per patient before treatment to 0.39 during treatment, indicating it helps protect against infections in patients with primary and secondary immunodeficiencies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-world results with IgPro20 for hypo- or agammaglobulinemia in Japan. Imai, K., Ishii, T., Nonoyama, S., et al.[2023]

Gammanorm® 16.5% (subcutaneous immunoglobulin) was found to be effective in providing protection against infections in patients with primary immunodeficiencies, with a mean dose of 28.7 g/month and only 7 out of 33 patients experiencing serious bacterial infections.

The treatment was well-tolerated, with no serious adverse events related to SCIG and only minor drug-related reactions in 2 out of 43 patients, indicating a favorable safety profile and improved quality of life for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy, safety and quality of life in patients receiving subcutaneous IgG treatment: experience in Bogotá, Colombia. Ortega-López, MC., Garay, J., Pinilla, ML.[2019]

A survey of 300 patients with primary immunodeficiency disease (PID) revealed that while 76% were satisfied with their immunoglobulin treatments, their overall physical and mental well-being remained below normal health standards.

Patients expressed a strong preference for improvements in treatment, including the option for 4-weekly infusions, home administration, self-administration, shorter infusion times, and fewer needle sticks, indicating a need for more patient-centered treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Improving current immunoglobulin therapy for patients with primary immunodeficiency: quality of life and views on treatment. Espanol, T., Prevot, J., Drabwell, J., et al.[2022]

In a study of 262 patients with common variable immunodeficiency, severe adverse reactions to immunoglobulin therapy were rare but could occur even after long-term tolerance to treatment.

Subcutaneous immunoglobulin therapy was generally safe for patients who switched from intravenous treatment, but those with a history of severe reactions to intravenous therapy are at higher risk for adverse reactions with subcutaneous administration, necessitating careful monitoring during the transition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prospective study on CVID patients with adverse reactions to intravenous or subcutaneous IgG administration. Quinti, I., Soresina, A., Agostini, C., et al.[2022]

A study analyzing 456 serum samples from PID-free children established reliable reference intervals for IgG and IgG subclasses using Optilite® reagents, which are crucial for diagnosing primary immunodeficiency (PID) in pediatric patients.

The findings highlight the importance of using manufacturer-specific reference intervals for IgG subclass measurements, as variations in calibration can lead to differences in reported levels compared to existing literature.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunoglobulin G (IgG) and IgG subclass reference intervals in children, using Optilite® reagents. Grunewald, O., Lopez, B., Brabant, S., et al.[2019]

A new flow cytometry-based assay was developed to detect various immunoglobulin classes and subclasses in the serum of 47 HIV-1-infected pregnant women, revealing that IgG1 was the most frequently reactive antibody class against HIV-1 proteins.

Mothers who transmitted HIV-1 to their infants had a broader distribution of antibody classes compared to non-transmitting mothers, with IgG2-gp160 being the most common antibody-antigen combination found in transmitting mothers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunoglobulin class and subclass antibodies to HIV proteins in maternal serum: association with perinatal transmission. Mann, DL., Hamlin-Green, G., Willoughby, A., et al.[2020]

In a study of 49 neonates with various gastrointestinal conditions, those with gastrointestinal anomalies and necrotizing enterocolitis showed a significant increase in IgA levels, indicating a potential immune response (p < 0.001).

Neonates with low anorectal anomalies and spina bifida had normal or undetectable IgA levels, suggesting that these conditions may not trigger the same immune response as gastrointestinal issues.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Significance of elevated IgA antibody levels in neonates with gastrointestinal conditions. Reen, DJ., Puri, P.[2019]

A male patient with common variable hypogammaglobulinemia experienced a significant decline in serum IgA levels, dropping to undetectable levels over one year, while his IgG and IgM levels remained low but stable.

Despite the lack of IgA, the patient's B lymphocytes still showed some ability to secrete IgA in laboratory tests, indicating that there may be residual functionality in his immune response.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development of a serologically complete IgA deficiency in a patient with common variable hypogammaglobulinemia. Smith, CI., Hammarström, L., Palmblad, J.[2006]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Facilitated Subcutaneous Immunoglobulin Treatment in Patients with Immunodeficiencies: the FIGARO Study. [2023]

2. Australia pubmed.ncbi.nlm.nih.gov

Real-world results with IgPro20 for hypo- or agammaglobulinemia in Japan. [2023]

3. England pubmed.ncbi.nlm.nih.gov

Efficacy, safety and quality of life in patients receiving subcutaneous IgG treatment: experience in Bogotá, Colombia. [2019]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Improving current immunoglobulin therapy for patients with primary immunodeficiency: quality of life and views on treatment. [2022]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Prospective study on CVID patients with adverse reactions to intravenous or subcutaneous IgG administration. [2022]

6. Germany pubmed.ncbi.nlm.nih.gov

Immunoglobulin G (IgG) and IgG subclass reference intervals in children, using Optilite® reagents. [2019]

7. United States pubmed.ncbi.nlm.nih.gov

Immunoglobulin class and subclass antibodies to HIV proteins in maternal serum: association with perinatal transmission. [2020]

8. United States pubmed.ncbi.nlm.nih.gov

Significance of elevated IgA antibody levels in neonates with gastrointestinal conditions. [2019]

9. United States pubmed.ncbi.nlm.nih.gov

Characterization of adult patients with IgG subclass deficiency and subnormal IgG2. [2020]

10. Scotland pubmed.ncbi.nlm.nih.gov

Development of a serologically complete IgA deficiency in a patient with common variable hypogammaglobulinemia. [2006]