Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA

• Register: EUCTR
• Last refreshed on: 11 February 2013
• Main ID: EUCTR2007-002611-27-DE
• Date of registration: 14/12/2007
• Prospective Registration: Yes
• Primary sponsor: Octapharma AG
• Public title: Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA
• Scientific title: Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NA
• Date of first enrolment: 27/10/2008
• Target sample size: 45
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-002611-27
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:

Countries of recruitment

France, Germany, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
Inclusion criteria:
• Age of = 2 years and = 75 years.
• For minor patients, above a minimum weight based on the amount of blood
required for testing: per individual, the trial-related blood loss (including any
losses in the manoeuvre) should not exceed 3% of the total blood volume during a
period of four weeks and should not exceed 1% at any single time (the total
volume of blood is estimated at 80 ml/kg body weight).
• Confirmed diagnosis of primary immunodeficiency as stated by the World Health
Organisation and requiring immunoglobulin replacement therapy due to hypogammaglobulinemia or agammaglobulinemia. The exact type
of PID should be recorded.
• Previously treated with commercial Octagam 5% every 21-28 days for at least 6 infusion intervals at a constant dose between 200-800 mg/kg body weight
• Availability of the IgG trough levels of the two previous infusions before enrollment, and maintenance of at least 5.5 g/L in the trough levels of these two
infusions.
• Negative result on a pregnancy test (HCG-based assay in blood or urine) forwomen of child-bearing potential and use of a reliable method of contraception for
the duration of the study.
• For adult patients: freely given written informed consent. For minor patients:
freely given written informed consent from both parents/legal guardians and
written informed assent from the child/adolescent = 8 years of age, according to
his/her age and capacity of understanding.
• Willingness to comply with all aspects of the protocol, including blood sampling,
for the duration of the study

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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Exclusion criteria:
• Acute infection requiring intravenous antibiotic treatment within two weeks before
screening.
• Known history of adverse reactions to IgA in other products.
• Exposure to blood or any blood product or derivative, other than a commercially
available Octagam 5%, within the past 3
months.
• Ongoing history of hypersensitivity or persistent reactions to blood or plasma
derived products, or any component of the investigational product, such as
maltose.
• Requirement of any routine pre-medication for IGIV infusion.
• History of congenital impairment of pulmonary function.
• Severe liver function impairment (ALAT 3x > normal value)
• Severe renal function impairment (creatinine > 120 µmol/L), or predisposition for
acute renal failure (e.g. any degree of pre-existing renal insufficiency or routine
treatment with known nephritic drugs).
• History of autoimmune haemolytic anemia.
• History of diabetes mellitus.
• Congestive heart failure NYHA III or IV.
• None-controlled arterial hypertension (systolic blood pressure > 160 mm Hg
and/or diastolic blood pressure > 90 mm Hg).
• History of DVT or thrombotic complications of IGIV therapy.
• Known to be infected with HIV, HCV or HBV.
• Presence of any clinically relevant disease or unstable condition beside those
concerning study indication at screening which in the opinion of the investigator
may interfere with the conduct of the study.
• Treatment with steroids, immunosuppressive or immunomodulatory drugs.
• Planned vaccination during the study period.
• Treatment with any investigational agent within the prior 3 months.
• Known or suspected to abuse alcohol, drugs, psychotropic agents or other
chemicals within the last 12 months.
• Pregnant and/or nursing women.


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

Health Condition(s) or Problem(s) studied

Primary Immundeficiency Diseases (PID)
MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome

Intervention(s)

Trade Name: Octagam 10%
Product Name: Octagam 10%
Pharmaceutical Form: Solution for infusion
Current Sponsor code: GAM10
Concentration unit: % (W/V) percent weight/volume
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

Primary end point(s): Primary endpoint is the rate of Octagam® 10% infusions with one or more adverse
events occurring during or within 72 hours after end of the infusion, including adverse events that are determined not to be product-related.
Second primary endpoint is the comparison of the pharmacokinetic profile of
Octagam® 10% with that of the previously used commercial Octagam® 5%.

Secondary Objective: The secondary objective of the study is to investigate the efficacy of Octagam® 10% in replacement therapy in PID by monitoring the rate of occurrence of serious bacterial infections, the rate of other infections, the trough (pre-next-dose) levels of total serum IgG and IgG subclasses (IgG1, IgG2, IgG3, IgG4), the trough (pre-next-dose) levels of selected antigen specific antibodies, use of antibiotics, rate of absence from work/school, number and days of hospitalizations.

Main Objective: The primary objective of the study is to investigate the safety of Octagam® 10% in
replacement therapy in PID and to compare the pharmacokinetic profile of Octagam® 10%with that of the previously used commercial Octagam® 5% (Pharmacokinetic sub-study).
Safety outcome parameters will be:
• Occurrence of adverse events.
• Short term tolerance parameters including vital signs (blood pressure, heart rate,
temperature, respiratory rate).
• Laboratory parameters (hematology, clinical chemistry, direct Coombs’test,
urinalysis) and tests for viral safety.
Pharmacokinetic outcome parameters will be the parameter Cmax, Cmin, t ½, Tmax, AUC, volume of distribution, and incremental recovery of serum total IgG; of IgG subclasses (IgG1, IgG2, IgG3, IgG4); of specific antibodies against Haemophilus influenzae, Streptococcus pneumoniae (types 4, 6B, 9V, 14, 18C, 19F, 23F), CMV, VZV, tetanus, measles; and of glucose and maltose.

Secondary ID(s)

GAM10-03

ISRCTN63491981