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

• Register: EUCTR
• Last refreshed on: 15 July 2013
• Main ID: EUCTR2012-000792-16-DE
• Date of registration: 10/05/2012
• Prospective Registration: Yes
• Primary sponsor: OCTAPHARMA AG
• Public title: Study to evaluate the efficacy, metabolism and safety of human immune globulin in patients with primary immunodeficiency diseases
• Scientific title: “CLINICAL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF OCTAGAM 5% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES”
• Date of first enrolment: 03/09/2012
• Target sample size: 23
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-000792-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no 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

Czech Republic, Germany, Hungary

Contacts

• Name: Clinical Research Department
• Affiliation: Octapharma Pharmazeutika Produktionsgesellschaft

• Name: Clinical Research Department
• Affiliation: Octapharma Pharmazeutika Produktionsgesellschaft

Key inclusion & exclusion criteria

Inclusion criteria:
1. Age of >= 18 years and <= 75 years.
2. Primary immunodeficiency syndromes with significant component hypogamma-globulinaemia or antibody deficiency.
3. Previously treated with a commercial IVIG
a) approximately every 21–28 days for at least 6 infusion intervals b) at a constant dose between 200 and 800 mg/kg body weight (+/- 20% of the mean dose for the last 6 infusions).
4. Availability of the IgG trough levels of the 2 previous infusions before enrolment, and maintenance of more than 5.0 g/L in the trough levels of these 2 infusions.
5. Negative result on a pregnancy test for women of childbearing potential and use of a reliable method of contraception for the duration of the study.
6. Freely given written informed consent.
7. Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
Are the trial subjects under 18? no
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 22
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion criteria:
8. Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
9. Known history of adverse reactions to IgA in other products.
10. Exposure to blood or any blood product or derivative, other than commercially available IVIG, within the past 3 months prior to enrolment.
11. Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product.
12. Requirement of any routine premedication for IVIG infusion.
13. History of congenital impairment of pulmonary function.
14. Severe liver function impairment
15. Presence of renal function impairment, or predisposition for acute renal failure.
16. History of autoimmune haemolytic anaemia.
17. History of diabetes mellitus.
18. Congestive heart failure NYHA class III or IV.
19. Non-controlled arterial hypertension.
20. History of deep vein thrombosis or thrombotic complications of IVIG therapy.
21. Known HIV, HCV, or HBV infection.
22. 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.
23. Treatment with steroids , immunosuppressive or immunomodulatory drugs.
24. Planned vaccination during the study period except for “killed” influenza vaccines (incl. H1N1).
25. Participating in another clinical trial or planned participation in another trial for the duration of this study.
26. Treatment with any investigational agent within 3 months prior to enrolment.
27. Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to enrolment.
28. Pregnant or nursing women.
29. Unable or unwilling to comply with the study protocol.


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

Health Condition(s) or Problem(s) studied

primary immunodeficiency disease (PID)
MedDRA version: 14.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency System Organ Class: 10021428 - Immune system disorders
MedDRA version: 14.1 Level: LLT Classification code 10049485 Term: Bruton's agammaglobulinemia System Organ Class: 10010331 - Congenital, familial and genetic disorders

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

Intervention(s)

Trade Name: Octagam 5%
Product Name: Octagam 5%
Pharmaceutical Form: Solution for infusion
CAS Number: 308067-58-5
Current Sponsor code: Octagam 5%
Other descriptive name: IMMUNOGLOBULIN G
Concentration unit: % percent
Concentration type: equal
Concentration number: 5-

Primary Outcome(s)

Secondary Objective: • To evaluate the efficacy of Octagam 5% in preventing serious bacterial infections compared to historical control data.
• To evaluate safety and tolerability of Octagam 5%.

Timepoint(s) of evaluation of this end point: continuously, see protocol

Main Objective: To determine the pharmacokinetic (PK) profile of Octagam 5% at steady state on standard prophylactic treatment of Primary Immunodeficiency Disorders (PID).

Primary end point(s): PK profile of Octagam with respect to total IgG, IgG subclasses (IgG1, IgG2, IgG3, IgG4), and antigen-specific antibodies at steady state on standard prophylactic treatment of PID

Secondary Outcome(s)

Secondary end point(s): • Trough levels of serum total IgG.
• Rate of serious bacterial infections (defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess) per person-year on treatment.
• The occurrence of all infections of any kind or seriousness.
• Non-serious infections (total and by category).
• Time to resolution of infections.
• Use of antibiotics.
• Hospitalisations due to infection.
• Episodes of fever.
• Days missed from school or work due to infections and their treatment.
• Occurrence of AEs.
• Occurrence of temporally associated AEs.
• Proportion of infusions with 1 or more temporally associated AEs.
• AEs by infusion rate.
• Short term tolerance parameters including vital signs (blood pressure, heart rate, temperature, respiratory rate).
• Laboratory parameters (haematology, clinical chemistry, and urinalysis).

Timepoint(s) of evaluation of this end point: continuously, see protocol

Secondary ID(s)

2012-000792-16-HU

GAMr-29

Source(s) of Monetary Support

Octapharma AG