Primary Immunofediciency (PID) syndromes MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870
Conditions
Interventions
Product Name: Human Normal Immunoglobulin for subcutaneous administration
Product Code: LFB-IgSC
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human Normal immunoglobulin
Other descr
Sponsors
LFB Biotechnologies
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female aged from 2 to 70 years old - Primary immunodeficiency syndrome with predominant antibody deficiency as: X-linked agammaglobulinaemia (XLA), Common variable immunodeficiency (CVID), Other PID syndrome in which the main immunological defect is deficiency in IgG production. - Stable IgG therapy for at least 5 months before the study, with a constant dose ranging from 0.2 to 0.8 g/kg per month and with regular intervals of 3 to 4 weeks for IVIg and one week for SCIg. - At least 2 documented serum IgG trough levels = 5 g/l with the previous IgG dosage regimen. - For women of childbearing potential, negative blood pregnancy test at enrolment and agreement to use a medically-acceptable method of contraception throughout the study. Are the trial subjects under 18? yes Number of subjects for this age range: 25 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 25 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: - History of severe allergy or serious adverse reaction to any IVIg, SCIg or an excipient of LFB-IgSC. - Patient with known antibodies to IgA. - Glomerular filtration rate < 80 ml/min/1.73m2 measured according to the Modified Diet in Renal Disease (MDRD) calculation. - Progressive hepatic disease that could worsen during the study. - Refusal of PK study (for adults only). - History of cardiac ischemia, cardiac insufficiency, cerebral ischemia, stroke, thrombotic events or pulmonary embolism. - Any additional cause of immunodeficiency, other than a primary immunodeficiency, (such as acquired immunodeficiency or malignancy of lymphoid cells). - Allogenic haematopoietic stem cell transplantation. - Need for routine premedication before SCIg infusions (excluding dermal anaesthetics). - Need for long-term therapy with corticosteroids or prophylactic antibiotics during the study. - Immunosuppressive agents, including anti-CD20 antibodies, during the last 6 months.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to assess the efficacy of LFB-IgSC.;Secondary Objective: There are two secondary objectives: - To assess the safety of LFB-IgSC, - To assess the pharmacokinetic profile of LFB-IgSC over a one-week injection interval. ;Primary end point(s): The primary endpoint is the number of serious bacterial infections (SBI) per patient and per year (annualized rate) as defined: - Bacteraemia or sepsis - Bacterial meningitis - Osteomyelitis / septic arthritis - Bacterial pneumonia - Visceral abscess ;Timepoint(s) of evaluation of this end point: Throughout the course of the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy endpoints - Rate of infections (serious or non-serious) per patient and per year - Number of days missed from work or school due to infections, - Number of days of hospitalization related to infection, - Number of days with use of antibiotics, - Number of fever episodes (>38°C) related to infections, - Total IgG trough levels will be assessed every month over a period of 6 months starting at week 21. These IgG trough levels will be compared to trough levels obtained with the previous immunoglobulin (IVIg or SCIg). In addition, distribution of IgG sub-classes will be described. Safety endpoints - Number of patients who have presented an AE and number of AEs: For all AEs reported during the study For IMP related AEs For infusional AEs i.e., AEs that begin during or within 72 hours after an infusion For local reactions to IMP For serious AEs - Percentage of site-infusions with site reaction - Changes in vital signs from before infusions to after infusions. ;Timepoint(s) of evaluation of this end point: - Total IgG trough levels: every month over a period of 6 months starting at W21. - All other secondary endpoints: throughout the course of the study | — |
Countries
France, Germany, Hungary, Italy, Poland, Ukraine, United Kingdom
Contacts
Public ContactClinical trial Information Desk
LFB Biotechnologies
Outcome results
None listed