Primary Immunodeficiency DiseasePhase 3ImunoforteNCT06089122
Efficacy, Safety, and Pharmacokinetics of Shu Yang IVIG
A Phase 3 study testing IVIG in people with Primary Immunodeficiency Disease, run by Azidus Brasil.
Trial summary
To evaluate the safety, efficacy, and pharmacokinetic properties of Shu Yang intravenous immune globulin in patients with primary immune deficiency aged less than 60 years. The main benefit of IVIG is to help the body fight against a large variety of infections generally associated with morbidity and mortality in… Read more about this study
- Who is it for?
- Ages 6 to 60 years
- Conditions the study is for
- Primary Immunodeficiency Disease
- What is being tested?
- IVIG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- Apr 2024to 8 Jun 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Written informed consent/assent.
- Male or female.
- Ages ≤ 60 years old and ≥ 06 years old.
- Diagnosis of Primary Immunodeficiency Disease (PID) with a reduction in antibody production due to:
- Common Variable Immunodeficiency (CVID) as per European Immunodeficiency Society (ESID)/Pan American Immunodeficiency Group (PAGID), as defined in section 5.1, OR b. X-linked agammaglobulinemia (XLA) as per ESID/PAGID, as defined in section 5.1.
- Receiving replacement therapy with intravenous immunoglobulin at 21- to 28-day intervals at 300-600 mg/kg/month for a minimum of 2 months before the start of the study;
Show all 10Show fewer
- Absence of episodes of serious bacterial infections with previous use of an IV immunoglobulin for at least 3 months before screening;
- Negative pregnancy test (in female patients with childbearing potential); readiness to use reliable methods of contraception throughout the study period;
- Patients who participated in a clinical trial with another experimental IVIG may be enrolled if they have a potential benefit according to Res. CNS 251/1997;
- Patients currently on treatment with any subcutaneous or intramuscular immunoglobulin may be enrolled switching to IVIG therapy at the investigator's discretion, considering the potential benefit to the patient.
Who cannot join
- Known intolerance or hypersensitivity to immunoglobulins or components of the test article;
- Any contraindications to the use of immunoglobulins;
- Secondary immunodeficiency or conditions potentially causing secondary immunodeficiency such as chronic lymphoid leukemia, lymphoma, multiple myeloma, protein-losing enteropathies or nephropathies, and hypoalbuminemia;
- Clinically relevant changes in the safety exams are defined as:
- Blood count
- o Hb < 10.5 g/dL
Show all 27Show fewer
- o Leukocytes < 3,000 /uL or >10,000 cells / uL
- o Absolute neutrophil count < 1,000 cells/mm3;
- Coagulation o TP and aPTT > 2.5 x ULN
- Biochemistry o glycated hemoglobin > 6.5%
- total bilirubin and fractions, alkaline phosphatase, ALT, AST, GGT > 2.5 x ULN
- creatinine above 3mg/dl or creatinine clearance < 30mL/min
- Urine I.
- Leukocyturia > 10,000 cells/mL 5. Any cancer either active or resolved within the last 12 months before screening;
- 6. Receiving any blood products (except intravenous immunoglobulins) during the last 3 months before screening;
- 7. Any febrile illness within 14 days before enrollment; Note: The patient may be rescreened after recovery.
- 8. History of thrombotic events (including myocardial infarction, stroke, pulmonary embolism, and deep vein thrombosis) within 6 months before enrollment;
- 9. Previous use of live attenuated virus vaccines;
- 10. Selective deficiency of immunoglobulin A (IgA) or known antibodies to IgA;
- 11. Known drug or alcohol abuse;
- 12. The need to use other investigational drugs, systemic immunosuppressants, and any other immunoglobulins;
- 13. Pregnancy or lactation;
- 14. Inability to comply with the protocol activities;
- 15. PIDs other than CVID or X-linked agammaglobulinemia
- 16. Patients infected with HIV, HBV or HCV
- 17. Patients with AIDS, cystic fibrosis, or active hepatitis B or C.
- 18. Any other condition that, in the Investigator's opinion may increase the risk of participation in this study.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Efficacy, Safety, and Pharmacokinetics of Shu Yang IVIG in Patients With Primary Immunodeficiency
To evaluate the safety, efficacy, and pharmacokinetic properties of Shu Yang intravenous immune globulin in patients with primary immune deficiency aged less than 60 years.
The main benefit of IVIG is to help the body fight against a large variety of infections generally associated with morbidity and mortality in patients with primary immunodeficiency diseases, particularly in CVID and XLA. In addition, a decrease in the number of infections, a reduction in medications and hospitalizations, and a better quality of life are expected.
Throughout treatment, approximately one-fourth of persons may experience a side effect. These are usually mild or bothersome but not dangerous. Very rarely, more serious side effects like allergic reactions or low blood counts (anemia) can occur. One of the most common side effects is headache. Other side effects include chills, fever, flushing, flu-like muscle pains or joint pains, feeling tired, nausea, vomiting, and rash. For the most part, these reactions typically happen with the first dose of IVIG or because change to a different brand of IVIG. All IVIG products have similar warnings and contraindications, such as the potential for renal failure, thrombotic events, aseptic meningitis, hemolysis, and anaphylactic reactions.
Read the detailed description
This is a Phase III, open-label, prospective, single-arm, multicenter trial to evaluate the efficacy of IVIG in maintaining the average of severe bacterial infections in less than one per year. The safety and pharmacokinetics (PK) of the investigational product will also be evaluated. Fifty male or female patients aged up to 60 years old will be selected. At least 20 patients must be up to 17 years old. During the trial, at least 20 adult patients will be invited to make up the PK assessment subgroups.
After obtaining the signed Informed Consent/Assent Form , the screening procedures will be performed including the immune deficiency history from the medical records and safety exams. Patients will start the trial with a run-in period to stabilize the IgG trough levels. This period could last two to six visits with posological adjustments until the last two IgG trough levels are above 4 (5) g/L.
After the run-in, the one-year test period will start at the V0. Depending on the treatment regimen, the patients will receive IVIG every 21 days (±3 days) up to day 378 or every 28 days (±4 days) up to day 364 when the close-out visit will occur. In all visits from all patients, a blood sample will be collected immediately before each IVIG administration to assess the IgG trough levels.
To assess the investigational product PK properties, a group of 20 patients will collect additional blood samples for dosing IgG levels between Visit 4 and Visit 5. Those taking IVIG every 21 days, will collect six additional blood samples at the following times after the injection 30 min, 2h, 24h, 72h, 7 days, and 14 days. Those taking IVIG every 28 days will collect seven additional blood samples at the times 30 min, 2h, 24h, 72h, 7 days, 14 days, and 21 days.
Adverse events will be collected at all visits to fulfill the safety endpoints. Moreover, the patients will have continuous access to the investigator's team to report adverse events, be instructed about how to proceed, or even perform Extra visits for presential medical evaluation.
Additionally, the patient will receive a diary to record AEs, any medication taken, infections of any kind, days of hospitalization, and days missed from major activities due to infections.
Trial duration: Each patient can participate in the trial for a maximum period of approximately 540 days from the time of signing the ICF or IAF until the close-out visit, including a Run-in period, depending on treatment regimen.
Blinding and Randomization: This is an open-label trial; no blinding and randomization procedures will be applied.
- Conditions
- Primary Immunodeficiency Disease
- Being tested
- IVIG
- Keywords
- IVIG, IMUNOFORTE, PID
- Medical subject headings
- Primary Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IVIGBiological
Also called Shu Yang IVIG
Patients with primary immunodeficiency will switch to Shu Yang IVIG and optimize the posology to IgG 300 to 600 mg every 21 or 28 days in a run-in period of 2 to 6 administrations, aiming at keeping the IgG trough levels above 5 g/L. At Visit 4, at least 20 patients will perform the PK assessment, collecting additional blood samples.
| Study group | Role | What they receive |
|---|---|---|
| IVIG | Experimental | Patients with primary immunodeficiency will switch to Shu Yang IVIG and optimize the posology to IgG 300 to 600 mg every 21 or 28 days in a run-in period of 2 to 6 administrations, aiming at keeping the IgG trough levels above 5 g/L. In the one-year test period, the patients will receive the test IVIG at the same dose/intervals of the optimization. However, the IgG trough levels will be monitored every visit, and new adjustments/dose optimization will be performed whenever needed to keep the IgG trough levels above 5g/L. Receives: Biological: IVIG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Efficacy Objective (average of acute serious bacterial infections)
The incidence of serious bacterial infections (septicemia, meningitis, visceral abscess, osteomyelitis, and pneumonia) within the 1-year follow-up is less than 1.0 per patient/year in the average of the population.
Measured: Between Visit 0 and Final Visit (through study completion, an average of 1 year)
Secondary outcomes (4)
Secondary Efficacy Objectives (assessment of the rate of non-serious infections)
The incidence of all acute infections except the serious acute bacterial infections within the 1-year follow-up (simple descriptive statistics).
Measured: Average incidence of non-serious infections per patient between Visit 0 and Final Visit (through study completion, an average of 1 year), as documented as treatment emergent adverse events (TEAEs);
Secondary Efficacy Objectives
Assessment of treatment length of infections per year;
Measured: Average number of days for treating infections per patient between visit 0 and final visit (through study completion, an average of 1 year), as documented as treatment emergent adverse events (TEAEs);
Secondary Efficacy Objectives
Assessment of missing time from school/work due to infections per month;
Measured: Average number of days off from school/work per patient/month, as documented in the patient's diary (through study completion, an average of 1 year);
Secondary Efficacy Objectives
Assessment of hospitalization episodes and length.
Measured: Number of days hospitalized per month overall and due to infection, as documented as treatment emergent adverse events - TEAEs (through study completion, an average of 1 year).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2023
- Study startApr 2024 (estimated)
- Primary completion8 Jan 2025 (estimated)
- Study completion8 Jun 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (22)Papers the sponsor has linked to this study.
- Other trials for Primary Immunodeficiency DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients with primary immunodeficiency will switch to Shu Yang IVIG and optimize the posology in a run-in period of 2 to 6 administrations. In the one-year test period, the patients will receive the test IVIG at 21- or 28-day intervals and be followed. IgG trough levels will be collected at all visits. At Visit 4, at least 20 patients will perform the PK assessment, collecting additional blood samples.
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Azidus Brasil
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sichuan Yuanda Shuyang Pharmaceutical Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Luciana Ferrara, Study director, Azidus Brasil
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06089122
- Sponsor's protocol ID
- IMUNOFORTE
- Other IDs
- U1111-1298-7059
- Acronym
- Imunoforte
- Status last verified
- Oct 2023
- First submitted
- 19 Sep 2023
- Last updated
- 12 Mar 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: It is believed that after the data analysis and presentation to the National Commission on Research Ethics, all data of the study will become public
Publications
Bierry G, Boileau J, Barnig C, Gasser B, Korganow AS, Buy X, Jeung MY, Roy C, Gangi A. Thoracic manifestations of primary humoral immunodeficiency: a comprehensive review. Radiographics. 2009 Nov;29(7):1909-20. doi: 10.1148/rg.297095717.
PubMed 19926753Blasczyk R, Westhoff U, Grosse-Wilde H. Soluble CD4, CD8, and HLA molecules in commercial immunoglobulin preparations. Lancet. 1993 Mar 27;341(8848):789-90. doi: 10.1016/0140-6736(93)90563-v.
PubMed 8096001Bonilla FA, Barlan I, Chapel H, Costa-Carvalho BT, Cunningham-Rundles C, de la Morena MT, Espinosa-Rosales FJ, Hammarstrom L, Nonoyama S, Quinti I, Routes JM, Tang ML, Warnatz K. International Consensus Document (ICON): Common Variable Immunodeficiency Disorders. J Allergy Clin Immunol Pract. 2016 Jan-Feb;4(1):38-59. doi: 10.1016/j.jaip.2015.07.025. Epub 2015 Nov 7.
PubMed 26563668BRUTON OC. Agammaglobulinemia. Pediatrics. 1952 Jun;9(6):722-8. No abstract available.
PubMed 14929630Busse PJ, Razvi S, Cunningham-Rundles C. Efficacy of intravenous immunoglobulin in the prevention of pneumonia in patients with common variable immunodeficiency. J Allergy Clin Immunol. 2002 Jun;109(6):1001-4. doi: 10.1067/mai.2002.124999.
PubMed 12063531Cordero E, Goycochea-Valdivia W, Mendez-Echevarria A, Allende LM, Alsina L, Bravo Garcia-Morato M, Gil-Herrera J, Gudiol C, Len-Abad O, Lopez-Medrano F, Moreno-Perez D, Munoz P, Olbrich P, Sanchez-Ramon S, Soler-Palacin P, Aguilera Cros C, Arostegui JI, Badell Serra I, Carbone J, Fortun J, Gonzalez-Granado LI, Lopez-Granados E, Lucena JM, Parody R, Ramakers J, Regueiro JR, Riviere JG, Roca-Oporto C, Rodriguez Pena R, Santos-Perez JL, Rodriguez-Gallego C, Neth O. Executive Summary of the Consensus Document on the Diagnosis and Management of Patients with Primary Immunodeficiencies. J Allergy Clin Immunol Pract. 2020 Nov-Dec;8(10):3342-3347. doi: 10.1016/j.jaip.2020.05.008.
PubMed 33161963Hagan JB, Fasano MB, Spector S, Wasserman RL, Melamed I, Rojavin MA, Zenker O, Orange JS. Efficacy and safety of a new 20% immunoglobulin preparation for subcutaneous administration, IgPro20, in patients with primary immunodeficiency. J Clin Immunol. 2010 Sep;30(5):734-45. doi: 10.1007/s10875-010-9423-4. Epub 2010 May 8.
PubMed 20454851Hartung HP, Mouthon L, Ahmed R, Jordan S, Laupland KB, Jolles S. Clinical applications of intravenous immunoglobulins (IVIg)--beyond immunodeficiencies and neurology. Clin Exp Immunol. 2009 Dec;158 Suppl 1(Suppl 1):23-33. doi: 10.1111/j.1365-2249.2009.04024.x.
PubMed 19883421Herriot R, Sewell WA. Antibody deficiency. J Clin Pathol. 2008 Sep;61(9):994-1000. doi: 10.1136/jcp.2007.051177.
PubMed 18755724Hoffmann F, Grimbacher B, Thiel J, Peter HH, Belohradsky BH; Vivaglobin Study Group. Home-based subcutaneous immunoglobulin G replacement therapy under real-life conditions in children and adults with antibody deficiency. Eur J Med Res. 2010 Jun 28;15(6):238-45. doi: 10.1186/2047-783x-15-6-238.
PubMed 20696632IDF Guide for Nurses - Immunoglobulin Therapy for Primary Immunodeficiency Diseases. 4th, pp. 59.
Milito C, Pulvirenti F, Pesce AM, Digiulio MA, Pandolfi F, Visentini M, Quinti I. Adequate patient's outcome achieved with short immunoglobulin replacement intervals in severe antibody deficiencies. J Clin Immunol. 2014 Oct;34(7):813-9. doi: 10.1007/s10875-014-0081-9. Epub 2014 Jul 22.
PubMed 25047154Mouthon L, Kaveri SV, Spalter SH, Lacroix-Desmazes S, Lefranc C, Desai R, Kazatchkine MD. Mechanisms of action of intravenous immune globulin in immune-mediated diseases. Clin Exp Immunol. 1996 May;104 Suppl 1:3-9.
PubMed 8625540Orange JS, Hossny EM, Weiler CR, Ballow M, Berger M, Bonilla FA, Buckley R, Chinen J, El-Gamal Y, Mazer BD, Nelson RP Jr, Patel DD, Secord E, Sorensen RU, Wasserman RL, Cunningham-Rundles C; Primary Immunodeficiency Committee of the American Academy of Allergy, Asthma and Immunology. Use of intravenous immunoglobulin in human disease: a review of evidence by members of the Primary Immunodeficiency Committee of the American Academy of Allergy, Asthma and Immunology. J Allergy Clin Immunol. 2006 Apr;117(4 Suppl):S525-53. doi: 10.1016/j.jaci.2006.01.015.
PubMed 16580469Pourpak Z, Aghamohammadi A, Sedighipour L, Farhoudi A, Movahedi M, Gharagozlou M, Chavoshzadeh Z, Jadid L, Rezaei N, Moin M. Effect of regular intravenous immunoglobulin therapy on prevention of pneumonia in patients with common variable immunodeficiency. J Microbiol Immunol Infect. 2006 Apr;39(2):114-20.
PubMed 16604243
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy, Safety, and Pharmacokinetics of Shu Yang IVIG in Patients With Primary Immunodeficiency
- ClinicalTrials.gov ID
- NCT06089122
- Acronym / sponsor ID
- Imunoforte / IMUNOFORTE
- Conditions
- Primary Immunodeficiency Disease
- Keywords
- IVIG, IMUNOFORTE, PID
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Azidus BrasilIndustry (pharma, biotech, devices)
- Collaborators
- Sichuan Yuanda Shuyang Pharmaceutical Co., Ltd.
- Study start
- Apr 2024 (estimated)
- Primary completion
- 8 Jan 2025 (estimated)
- Study completion
- 8 Jun 2025 (estimated)
- First posted
- 18 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the safety, efficacy, and pharmacokinetic properties of Shu Yang intravenous immune globulin in patients with primary immune deficiency aged less than 60 years.
The main benefit of IVIG is to help the body fight against a large variety of infections generally associated with morbidity and mortality in patients with primary immunodeficiency diseases, particularly in CVID and XLA. In addition, a decrease in the number of infections, a reduction in medications and hospitalizations, and a better quality of life are expected.
Throughout treatment, approximately one-fourth of persons may experience a side effect. These are usually mild or bothersome but not dangerous. Very rarely, more serious side effects like allergic reactions or low blood counts (anemia) can occur. One of the most common side effects is headache. Other side effects include chills, fever, flushing, flu-like muscle pains or joint pains, feeling tired, nausea, vomiting, and rash. For the most part, these reactions typically happen with the first dose of IVIG or because change to a different brand of IVIG. All IVIG products have similar warnings and contraindications, such as the potential for renal failure, thrombotic events, aseptic meningitis, hemolysis, and anaphylactic reactions.
Detailed description
This is a Phase III, open-label, prospective, single-arm, multicenter trial to evaluate the efficacy of IVIG in maintaining the average of severe bacterial infections in less than one per year. The safety and pharmacokinetics (PK) of the investigational product will also be evaluated. Fifty male or female patients aged up to 60 years old will be selected. At least 20 patients must be up to 17 years old. During the trial, at least 20 adult patients will be invited to make up the PK assessment subgroups.
After obtaining the signed Informed Consent/Assent Form , the screening procedures will be performed including the immune deficiency history from the medical records and safety exams. Patients will start the trial with a run-in period to stabilize the IgG trough levels. This period could last two to six visits with posological adjustments until the last two IgG trough levels are above 4 (5) g/L.
After the run-in, the one-year test period will start at the V0. Depending on the treatment regimen, the patients will receive IVIG every 21 days (±3 days) up to day 378 or every 28 days (±4 days) up to day 364 when the close-out visit will occur. In all visits from all patients, a blood sample will be collected immediately before each IVIG administration to assess the IgG trough levels.
To assess the investigational product PK properties, a group of 20 patients will collect additional blood samples for dosing IgG levels between Visit 4 and Visit 5. Those taking IVIG every 21 days, will collect six additional blood samples at the following times after the injection 30 min, 2h, 24h, 72h, 7 days, and 14 days. Those taking IVIG every 28 days will collect seven additional blood samples at the times 30 min, 2h, 24h, 72h, 7 days, 14 days, and 21 days.
Adverse events will be collected at all visits to fulfill the safety endpoints. Moreover, the patients will have continuous access to the investigator's team to report adverse events, be instructed about how to proceed, or even perform Extra visits for presential medical evaluation.
Additionally, the patient will receive a diary to record AEs, any medication taken, infections of any kind, days of hospitalization, and days missed from major activities due to infections.
Trial duration: Each patient can participate in the trial for a maximum period of approximately 540 days from the time of signing the ICF or IAF until the close-out visit, including a Run-in period, depending on treatment regimen.
Blinding and Randomization: This is an open-label trial; no blinding and randomization procedures will be applied.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients with primary immunodeficiency will switch to Shu Yang IVIG and optimize the posology in a run-in period of 2 to 6 administrations. In the one-year test period, the patients will receive the test IVIG at 21- or 28-day intervals and be followed. IgG trough levels will be collected at all visits. At Visit 4, at least 20 patients will perform the PK assessment, collecting additional blood samples.
| Arm | Type | Receives |
|---|---|---|
| IVIG | Experimental | Biological: IVIGPatients with primary immunodeficiency will switch to Shu Yang IVIG and optimize the posology to IgG 300 to 600 mg every 21 or 28 days in a run-in period of 2 to 6 administrations, aiming at keeping the IgG trough levels above 5 g/L. In the one-year test period, the patients will receive the test IVIG at the same dose/intervals of the optimization. However, the IgG trough levels will be monitored every visit, and new adjustments/dose optimization will be performed whenever needed to keep the IgG trough levels above 5g/L. |
Who may qualify
- Ages
- 6 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Written informed consent/assent.
- Male or female.
- Ages ≤ 60 years old and ≥ 06 years old.
- Diagnosis of Primary Immunodeficiency Disease (PID) with a reduction in antibody production due to:
- Receiving replacement therapy with intravenous immunoglobulin at 21- to 28-day intervals at 300-600 mg/kg/month for a minimum of 2 months before the start of the study;
Key exclusion criteria (27 listed)
- Known intolerance or hypersensitivity to immunoglobulins or components of the test article;
- Any contraindications to the use of immunoglobulins;
- Secondary immunodeficiency or conditions potentially causing secondary immunodeficiency such as chronic lymphoid leukemia, lymphoma, multiple myeloma, protein-losing enteropathies or nephropathies, and hypoalbuminemia;
- Clinically relevant changes in the safety exams are defined as:
- o Hb < 10.5 g/dL
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IVIGAlso: Shu Yang IVIGPatients with primary immunodeficiency will switch to Shu Yang IVIG and optimize the posology to IgG 300 to 600 mg every 21 or 28 days in a run-in period of 2 to 6 administrations, aiming at keeping the IgG trough levels above 5 g/L. At Visit 4, at least 20 patients will perform the PK assessment, collecting additional blood samples. | Biological | IVIG |
Primary outcome
Primary Efficacy Objective (average of acute serious bacterial infections)
Time frame: Between Visit 0 and Final Visit (through study completion, an average of 1 year)
The incidence of serious bacterial infections (septicemia, meningitis, visceral abscess, osteomyelitis, and pneumonia) within the 1-year follow-up is less than 1.0 per patient/year in the average of the population.
4 secondary outcomes
Secondary Efficacy Objectives (assessment of the rate of non-serious infections)
Time frame: Average incidence of non-serious infections per patient between Visit 0 and Final Visit (through study completion, an average of 1 year), as documented as treatment emergent adverse events (TEAEs);
Secondary Efficacy Objectives
Time frame: Average number of days for treating infections per patient between visit 0 and final visit (through study completion, an average of 1 year), as documented as treatment emergent adverse events (TEAEs);
Secondary Efficacy Objectives
Time frame: Average number of days off from school/work per patient/month, as documented in the patient's diary (through study completion, an average of 1 year);
Secondary Efficacy Objectives
Time frame: Number of days hospitalized per month overall and due to infection, as documented as treatment emergent adverse events - TEAEs (through study completion, an average of 1 year).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2024.
- Background: PubMed 19926753Bierry G, Boileau J, Barnig C, Gasser B, Korganow AS, Buy X, Jeung MY, Roy C, Gangi A. Thoracic manifestations of primary humoral immunodeficiency: a comprehensive review. Radiographics. 2009 Nov;29(7):1909-20. doi: 10.1148/rg.297095717.
- Background: PubMed 8096001Blasczyk R, Westhoff U, Grosse-Wilde H. Soluble CD4, CD8, and HLA molecules in commercial immunoglobulin preparations. Lancet. 1993 Mar 27;341(8848):789-90. doi: 10.1016/0140-6736(93)90563-v.
- Background: PubMed 26563668Bonilla FA, Barlan I, Chapel H, Costa-Carvalho BT, Cunningham-Rundles C, de la Morena MT, Espinosa-Rosales FJ, Hammarstrom L, Nonoyama S, Quinti I, Routes JM, Tang ML, Warnatz K. International Consensus Document (ICON): Common Variable Immunodeficiency Disorders. J Allergy Clin Immunol Pract. 2016 Jan-Feb;4(1):38-59. doi: 10.1016/j.jaip.2015.07.025. Epub 2015 Nov 7.
- Background: PubMed 14929630BRUTON OC. Agammaglobulinemia. Pediatrics. 1952 Jun;9(6):722-8. No abstract available.
- Background: PubMed 12063531Busse PJ, Razvi S, Cunningham-Rundles C. Efficacy of intravenous immunoglobulin in the prevention of pneumonia in patients with common variable immunodeficiency. J Allergy Clin Immunol. 2002 Jun;109(6):1001-4. doi: 10.1067/mai.2002.124999.
- Background: PubMed 33161963Cordero E, Goycochea-Valdivia W, Mendez-Echevarria A, Allende LM, Alsina L, Bravo Garcia-Morato M, Gil-Herrera J, Gudiol C, Len-Abad O, Lopez-Medrano F, Moreno-Perez D, Munoz P, Olbrich P, Sanchez-Ramon S, Soler-Palacin P, Aguilera Cros C, Arostegui JI, Badell Serra I, Carbone J, Fortun J, Gonzalez-Granado LI, Lopez-Granados E, Lucena JM, Parody R, Ramakers J, Regueiro JR, Riviere JG, Roca-Oporto C, Rodriguez Pena R, Santos-Perez JL, Rodriguez-Gallego C, Neth O. Executive Summary of the Consensus Document on the Diagnosis and Management of Patients with Primary Immunodeficiencies. J Allergy Clin Immunol Pract. 2020 Nov-Dec;8(10):3342-3347. doi: 10.1016/j.jaip.2020.05.008.
- Background: PubMed 20454851Hagan JB, Fasano MB, Spector S, Wasserman RL, Melamed I, Rojavin MA, Zenker O, Orange JS. Efficacy and safety of a new 20% immunoglobulin preparation for subcutaneous administration, IgPro20, in patients with primary immunodeficiency. J Clin Immunol. 2010 Sep;30(5):734-45. doi: 10.1007/s10875-010-9423-4. Epub 2010 May 8.
- Background: PubMed 19883421Hartung HP, Mouthon L, Ahmed R, Jordan S, Laupland KB, Jolles S. Clinical applications of intravenous immunoglobulins (IVIg)--beyond immunodeficiencies and neurology. Clin Exp Immunol. 2009 Dec;158 Suppl 1(Suppl 1):23-33. doi: 10.1111/j.1365-2249.2009.04024.x.
- Background: PubMed 18755724Herriot R, Sewell WA. Antibody deficiency. J Clin Pathol. 2008 Sep;61(9):994-1000. doi: 10.1136/jcp.2007.051177.
- Background: PubMed 20696632Hoffmann F, Grimbacher B, Thiel J, Peter HH, Belohradsky BH; Vivaglobin Study Group. Home-based subcutaneous immunoglobulin G replacement therapy under real-life conditions in children and adults with antibody deficiency. Eur J Med Res. 2010 Jun 28;15(6):238-45. doi: 10.1186/2047-783x-15-6-238.
- BackgroundIDF Guide for Nurses - Immunoglobulin Therapy for Primary Immunodeficiency Diseases. 4th, pp. 59.
- Background: PubMed 25047154Milito C, Pulvirenti F, Pesce AM, Digiulio MA, Pandolfi F, Visentini M, Quinti I. Adequate patient's outcome achieved with short immunoglobulin replacement intervals in severe antibody deficiencies. J Clin Immunol. 2014 Oct;34(7):813-9. doi: 10.1007/s10875-014-0081-9. Epub 2014 Jul 22.
- Individual participant dataSharing planned: No. It is believed that after the data analysis and presentation to the National Commission on Research Ethics, all data of the study will become public
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Luciana Ferrara study director
Azidus Brasil
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing IVIG in people with Primary Immunodeficiency Disease, run by Azidus Brasil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Luciana Ferrara (+55 19 981428814, luciana.ferrara@azidusbrasil.com.br), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Efficacy Objective (average of acute serious bacterial infections) (time frame: Between Visit 0 and Final Visit (through study completion, an average of 1 year)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06089122, which also lists 22 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Mar 2024.