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

• Register: EUCTR
• Last refreshed on: 12 May 2014
• Main ID: EUCTR2013-000620-34-IT
• Date of registration: 30/07/2013
• Prospective Registration: Yes
• Primary sponsor: LFB Biotechnologies
• Public title: 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
• Scientific title: A Multicentre Phase III Study on the Efficacy, Safety and Pharmacokinetics of LFB-IgSC in Patients with Primary Immunodeficiency (PID) Syndromes - NA
• Date of first enrolment: 05/08/2013
• Target sample size: 55
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-000620-34
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

France, Germany, Hungary, Italy, Poland, Ukraine, United Kingdom

Contacts

• Name: Clinical trial Information Desk
• Affiliation: LFB Biotechnologies

• Name: Clinical trial Information Desk
• Affiliation: LFB Biotechnologies

Key inclusion & exclusion 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:
- 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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Primary Immunofediciency (PID) syndromes
MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Intervention(s)

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 descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 250-

Primary Outcome(s)

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

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.

Main Objective: The primary objective of the study is to assess the efficacy of LFB-IgSC.

Timepoint(s) of evaluation of this end point: Throughout the course of the study

Secondary Outcome(s)

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

Secondary ID(s)

IGSC-1103

Source(s) of Monetary Support

LFB Biotechnologies