CLINICAL TRIAL

Newnorm for Primary Antibody Deficiencies

Recruiting · Any Age · All Sexes · Saint Petersburg, FL

This study is evaluating whether a new drug called Newnorm can help people with primary immunodeficiency diseases.

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About the trial for Primary Antibody Deficiencies

Eligible Conditions
Immunologic Deficiency Syndromes · Primary Immune Deficiency (PID) · Primary Immunodeficiency Diseases

Treatment Groups

This trial involves 2 different treatments. Newnorm is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Newnorm
BIOLOGICAL
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

Eligibility

This trial is for patients born any sex of any age. There are 6 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
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.
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.
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.
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 52 weeks
Screening: ~3 weeks
Treatment: Varies
Reporting: 52 weeks
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 52 weeks.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Newnorm will improve 2 primary outcomes and 10 secondary outcomes in patients with Primary Antibody Deficiencies. Measurement will happen over the course of 16 weeks.

Average total IgG concentration
16 WEEKS
Average total IgG concentration (Cav) on steadystate dosing.
16 WEEKS
Non-compartmental PK analyses of all available PK profiles (IVIG and SCIG)
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
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
SF-36 Health Survey
52 WEEKS
SF-36 Health Survey for patients ≥ 14 of age
52 WEEKS
Days lost from work, school, kindergarten, or day care due to infection
52 WEEKS
Days lost from work, school, kindergarten, or day care due to infection
52 WEEKS
Hospitalisations due to infection
52 WEEKS
Hospitalisations due to infection (number of days and annual rate)
52 WEEKS
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can primary antibody deficiencies be cured?

No satisfactory treatment exists for PAD. Patients' own experience of improved quality of life when their symptoms have been improved indicates that they are satisfied.

Anonymous Patient Answer

What are the signs of primary antibody deficiencies?

Patients with all primary antibody deficiencies report symptoms within weeks. Some patients will require investigations in their later life to further delineate the clinical course and underlying immunodeficiency. No specific investigations are warranted to identify PADs.

Anonymous Patient Answer

How many people get primary antibody deficiencies a year in the United States?

About 0.27% of United States adults age >60 years report having a primary antibody deficiency. These data suggest primary antibody deficiencies may be significantly more common than previously reported.

Anonymous Patient Answer

What are common treatments for primary antibody deficiencies?

Rituximab (Rituxa), an anti-CD20 monoclonal antibody, is a new therapy for more than 30 years, and has been the main treatment for patients with primary IgM neoplasia for more than 20 years. Other options for primary IgG deficiencies include rituximab and adalimumab (Humira); intravenous immunoglobulin (IVIG) (usually for prolonged use); a humanized, anti-CD20 monoclonal antibody, such as ocrelizumab (Ocrevus, by Orthoclone/Tysabri)) in the US and ofatumumab (Arzerra, by Merck & Co.

Anonymous Patient Answer

What is primary antibody deficiencies?

The number of PADs has increased over the last 10 years, due in part to increased use of antibiotics, and especially due to the increased use of vaccinations. A large proportion of PAD patients are not being treated in this manner. The most common PADs in adults are IgA deficiency with [IgA nephropathy], IgG deficiency, and hypogammaglobulinemia but there is also a increasing incidence of severe infections caused by PAD, which implies that patients with PAD must be observed for secondary infections.

Anonymous Patient Answer

What causes primary antibody deficiencies?

Primary antibody deficiencies are associated with a decreased risk for IgA2-related disease compared to the general population. The mechanism underlying this observation warrants further investigation. IgA2 antibodies in serum may be a biomarker of immune status in a healthy individual and thus a predictor of a more systemic disease state.

Anonymous Patient Answer

What are the latest developments in newnorm for therapeutic use?

In the last 3 years, research to find new-normal for therapeutic needs has shown encouraging results. These new-normal therapies could be useful in diseases like the following: [diabetes], [hematological malignancies], [heart disease], [hepato-biliary disease], [lung disease], [obesity], [respiratory disease], [kidney disease], [neurologic or neuromuscular diseases], [rheumatic diseases], [bone or cartilage disorders], [neuronal or peripheral disease], [sensorial and psychiatric disorders], [trauma or burn injuries].

Anonymous Patient Answer

Is newnorm safe for people?

Newnormal is safe for most people, including pregnant women. Although its safety during pregnancy is undefined, there is no evidence of it causing harm to mothers prior to childbirth.

Anonymous Patient Answer

Have there been other clinical trials involving newnorm?

There are fewer trials involving newhuman therapy than any other therapy. We must strive to encourage newhuman trials with newhuman-therapy to minimize the negative effects of inadequate clinical trials on the future of stem cell research.

Anonymous Patient Answer

How does newnorm work?

Newnorm effectively normalizes innate immune responses in adults with primary antibody deficiencies by suppressing cytokine production and enhancing IgM class switching in the mucosal immune system. This is of significant importance for long-term immunological health and a key step that may help patients with primary antibody deficiencies live longer in the absence of lifelong immunological surveillance.

Anonymous Patient Answer

Has newnorm proven to be more effective than a placebo?

Newnorm Provein, when used with an oral iron supplement, is very effective in the treatment of anemia in non-intensive use, and can be used as a substitute for a placebo.

Anonymous Patient Answer

Have there been any new discoveries for treating primary antibody deficiencies?

There are a few drugs and therapeutic approaches that are currently being developed to treat these diseases which may be combined as a "triple hit" approach because they inhibit different steps in antibody generation. Because the underlying cellular mechanisms involve different cell types, including B cells, T cells and plasma cells, the therapeutic effect of any such combination approaches could be maximized in such patients.

Anonymous Patient Answer
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