a primary immunodeficiency as defined by the ESID and validated by a reference centre : • X-linked agammaglobulinemia (XLA) • Common variable immunodeficiency (CVID) MedDRA version: 14.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency System Organ Class: 100000004870 MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870 MedDRA version: 14.1 Level: LLT Classification code 10001471
Conditions
Interventions
Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR
Product Code: I10E
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE
Concentration unit: mg/
Sponsors
LFB BIOTECHNOLOGIES
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Both genders 2. Age between 2 to 65 year old 3. Has an humoral primary immunodeficiency as defined by the ESIDs1 and validated by the reference centers : · X-linked agammaglobulinemia (XLA) · Common variable immunodeficiency (CVID) 4. The requirement of IVIG therapy at a dose between 0.2 to 0.8 g/kg and at administration interval of 3-weeks or 4-weeks may be considered medically necessary for the treatment of the patient condition 5. Patient already under IgG replacement therapy with a stable dosage and with 3 documented trough levels ³ 4 g/l within 6 months prior to the introduction of I10E (trough levels should have been performed at 3 to 8 weeks interval between samples or naïve. All these patients must be stabilized under I10E during the first 6 months during the study with IgG trough levels ³ 6 g/L). 6. Written informed consent obtained prior to any study-related procedure and study product administration from either the patient or the patient's legal representative 7. If required by national regulation, registered with a social security or health insurance system Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 34 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: 1. Patient already under IgG replacement therapy with no 3 documented trough levels ³ 4 g/l within 6 months prior to the introduction of I10E or patient with 3 documented Ig G trough level not performed within defined 3 to 8 weeks interval between samples. 2. Known allergy or history of serious adverse reaction to any IgG 3. Known hypersensitivity to the active substance or to any of the excipients 4. Patient with known antibodies to IgA 5. Chronic renal insufficiency or creatinine clearance values 12 years) or breastfeeding woman, or woman of childbearing potential with no effective contraception (injectable, patch or combined oral estro-progestative or progestative contraceptives, intra-uterine devices of type 'copper T' and levonorgest releasing IU systems, depot intramuscular medroxyprogesteron, subcutaneous implants of progestative contraceptives implants). 21. Blood levels of total bilirubin > 2 x upper limit of normal range, alanine aminotransferase (ALT) or aspartate amino transferase (AST) > 3 x upper limit of normal range. 22. Anticipated poor compliance of patient with study procedures
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of I10E in preventing acute serious bacterial infections;Secondary Objective: To assess the safety, efficacy, pharmacokinetic of I10E;Primary end point(s): The primary endpoint is the number of acute serious bacterial infections (aSBI) per patient and per year (annualized rate) as defined by the FDA guidance 70 FR 72124 and evaluated by the DSMB: • bacteraemia or sepsis, • bacterial meningitis, • osteomyelitis / septic arthritis, • bacterial pneumonia, • visceral abscess.;Timepoint(s) of evaluation of this end point: see protocol | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: see protocol ;Secondary end point(s): 1) Efficacy criteria Serum total IgG trough levels: • Total IgG trough concentrations will be measured by nephelemetry (central lab) in serum obtained from blood samples collected prior to each infusion from all included patients during the 12 month-study period. In addition, distribution of IgG sub-classes and content of clinically relevant antibodies will be assessed. • Descriptive relationship between total IgG trough levels and serious all infections. Other efficacy criteria will be assessed both by investigator and patients follow-up diaries (annualized rate) : • Number of all infections per patient and per year: annualized rate of documented of serious and non-serious bacterial infections • Number of days missed from work or school due to infections, • Number of days hospitalized related to infections, • Number of days with use of antibiotics related to infections, • Number of fever episodes related to infections 2) Safety criteria Adverse events (AEs), serious (SAEs) and non serious adverse events from all subjects followed throughout the clinical studies will be recorded and reported regardless of whether the AE is determined to be related to the product or not by the investigator (Pre-medication is discouraged to avoid masking AEs). Safety evaluation will include the monitoring of the following items: • Vital signs (blood pressure, heart rate, temperature) will be monitored by the study nurse during infusions within 30 mn after the end of the infusion • Laboratory tests including direct Coombs' test will be monitored at each visit • Adverse events that occurred during infusions will be noted by the study nurse or the investigator • Adverse events that occurred during intervals between infusions will be recorded by the patients in their diaries and reviewed by the investigator at each visit. • Renal function, hepatic function and blood cells | — |
Countries
Czech Republic, France, Hungary, Lithuania, Poland, Serbia, Ukraine
Contacts
Public ContactClinical trial information desk
LFB BIOTECHNOLOGIES
Outcome results
None listed