Study to evaluate the efficacy, metabolism and safety of human immune globulin in patients with primary immunodeficiency diseases.

• Conditions: Primary immunodeficiency diseases; MedDRA version: 14.1Level: LLTClassification code 10049485Term: Bruton's agammaglobulinemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 14.1Level: LLTClassification code 10010112Term: Common variable immunodeficiencySystem Organ Class: 10021428 - Immune system disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2009-011434-10-Outside-EU/EEA
• Lead Sponsor: OCTAPHARMA AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-01-27
• Last Update Posted: 24 April 2012

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 51

Inclusion Criteria

Patients who meet the following criteria can be included:
1. Age of = 2 years and = 75 years.
2. 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 maneuver) should not exceed 3% of the total blood volume during a period of 4 weeks and should not exceed 1% at any single time (the total volume of blood is estimated at 80 mL/kg body weight).
3. Confirmed diagnosis of CVID or XLA.
4. Previously treated with a commercial immune globulin intravenous (human)
a) every 21–28 days for at least 6 infusion intervals (± 3 days for the last three infusions and ± 7 days for the three infusions before the last three infusions)
b) at a constant dose between 200 and 800 mg/kg body weight (± 20% of the mean dose for the last 6 infusions).
5. Availability of the IgG trough levels of the 2 previous infusions before enrollment, and maintenance of at least 5.5 g/L in the trough levels of these 2 infusions.
6. Negative result on a pregnancy test (HCG-based assay in urine) for women of childbearing potential and use of a reliable method of contraception for the duration of the study.
7. For adult patients: freely given written informed consent. For minor patients: freely given written informed consent from parents/legal guardians and written informed assent from the child/adolescent in accordance with the applicable approvals.
8. 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: 24
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 1

Exclusion Criteria

1. Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
2. Known history of adverse reactions to IgA in other products.
3. Exposure to blood or any blood product or derivative, other than commercially available IVIG, within the past 3 months prior to enrollment.
4. Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product.
5. Requirement of any routine premedication for IVIG infusion.
6. History of congenital impairment of pulmonary function.
7. Severe liver function impairment (ALAT 3x > upper limit of normal).
8. Presence of renal function impairment (creatinine > 120 micromol/L), or predisposition for acute renal failure (e.g. any degree of pre-existing renal insufficiency or routine treatment with known nephritic drugs).
9. History of autoimmune hemolytic anemia.
10. History of diabetes mellitus.
11. Congestive heart failure NYHA class III or IV.
12. Non-controlled arterial hypertension (systolic blood pressure > 160 mmHg or diastolic blood pressure > 90 mmHg).
13. History of deep vein thrombosis or thrombotic complications of IVIG therapy.
14. A positive result at screening on any of the following viral markers: HIV, HCV, HBV.
15. Presence of any clinically relevant disease or unstable condition at screening, other than PID, which in the opinion of the investigator could interfere with the conduct of the study.
16. Treatment with steroids (oral or parenteral, long-term, i.e. 30 days or more, not intermittent or burst, daily, = 0.15 mg of prednisone or equivalent/kg/day), immunosuppressive or immunomodulatory drugs.
17. Planned vaccination during the study period.
18. Treatment with any investigational agent within 3 months prior to enrollment.
19. Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to enrollment.
20. Pregnant or nursing women.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified