CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES

• Register: EUCTR
• Last refreshed on: 24 April 2012
• Main ID: EUCTR2009-011434-10-DE
• Date of registration: 27/10/2009
• Prospective Registration: Yes
• Primary sponsor: OCTAPHARMA AG
• Public title: CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES
• Scientific title: CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES
• Date of first enrolment: 02/03/2010
• Target sample size: 50
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-011434-10
• 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

Germany

Key inclusion & exclusion criteria

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:
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:
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.



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

Health Condition(s) or Problem(s) studied

Primary immunodeficiency diseases
MedDRA version: 12.0 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency
MedDRA version: 12.0 Level: LLT Classification code 10049485 Term: Bruton's agammaglobulinemia

Intervention(s)

Product Name: NewGam
Product Code: NewGam
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: immunoglobulin G
Other descriptive name: NewGam
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-

Primary Outcome(s)

Main Objective: The primary objective of the study is to assess the efficacy of NewGam in preventing
serious bacterial infections compared to historical control data.

Primary end point(s): The primary endpoint is the rate of serious bacterial infections (defined as
bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial
pneumonia, and visceral abscess) per person-year on treatment.

Secondary Objective: The secondary objectives of the study are:
• To evaluate the safety of NewGam.
• To determine the PK profile of NewGam.
• To assess the effect of NewGam on QoL measures.

Secondary ID(s)

NGAM-01