A clinical trial with a subcutaneous immunoglobulin (LFB-IgSC) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Primary Immunodeficiency (PID) syndromes

Conditions: Primary Immunofediciency (PID) syndromes
MedDRA version: 14.1Level: HLTClassification code 10036700Term: Primary immunodeficiency syndromesSystem Organ Class: 100000004870
Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Registration Number: EUCTR2013-000620-34-DE

Lead Sponsor: FB Biotechnologies

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 55

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

- 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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the study is to assess the efficacy of LFB-IgSC.;Secondary Objective: There are two secondary objectives: <br>- To assess the safety of LFB-IgSC,<br>- To assess the pharmacokinetic profile of LFB-IgSC over a one-week injection interval.<br>;Primary end point(s): The primary endpoint is the number of serious bacterial infections (SBI) per patient and per year (annualized rate) as defined:<br>- Bacteraemia or sepsis<br>- Bacterial meningitis<br>- Osteomyelitis / septic arthritis<br>- Bacterial pneumonia<br>- Visceral abscess<br>;Timepoint(s) of evaluation of this end point: Throughout the course of the study

Secondary Outcome Measures