Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
NGAM-01
Study information
Scientific title
Acronym
Study hypothesis
To assess the efficacy of NewGam in preventing serious bacterial infections compared to historical control data.
Ethics approval
Saint Louis University Biomedical Institutional Review Board approved on the 15th September 2009 (ref: 16291)
Study design
Prospective open-label non-controlled non-randomised multi-centre phase III study
Primary study design
Interventional
Secondary study design
Non randomised controlled trial
Trial setting
Hospitals
Trial type
Treatment
Patient information sheet
Not available in web format, please use the contact details below to request a patient information sheet
Condition
Primary immunodeficiency diseases (PID)
Intervention
The treatment intervals with NewGam will be documented over 12 months: every 3 or every 4 weeks (+/- 3 days) following the same dosing interval as the previous commercial IVIG infusions. Therefore, it is anticipated that each patient will be administered either 17 (at 3-week intervals) or 13 (at 4-week intervals) infusions of NewGam.
Intervention type
Drug
Phase
Phase III
Drug names
NewGam
Primary outcome measure
To assess the efficacy of NewGam in preventing serious bacterial infections compared to historical control data, measured throughout the 12-month treatment period.
Secondary outcome measures
1. To evaluate the safety of NewGam, measured throughout the 12-month treatment period
2. To determine the pharmacokinetic (PK) profile of NewGam, measured on the 9th (or soonest subsequent) NewGam infusion day for patients on the 3-week schedule, or on the 7th (or soonest subsequent) NewGam infusion day for patients on the 4-week schedule
3. To assess the effect of NewGam on quality of life (QoL) measures, measured using the Child Health Questionnaire (CHQ-PF50) (completed by a parent or guardian of patients less than 14 years of age) or the 36-item short form health survey (SF-36) in patients greater than or equal to 14 years of age. These will be completed at the first and last infusion visits and at intervals of 3 months, i.e. at the 5th, 10th and 14th infusion days for patients on the 3-week schedule, or on the 4th, 8th and 11th infusion days for patients on the 4-week schedule.
Overall trial start date
01/12/2009
Overall trial end date
01/04/2011
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. Aged greater than or equal to 2 years and less than or equal to 75 years, either sex
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 manoeuvre) 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 common variable immunodeficiency (CVID) or X-linked agammaglobulinaemia (XLA)
4. Previously treated with a commercial immune globulin intravenous (human) every 21 - 28 days for at least 6 infusion intervals at a constant dose between 200 and 800 mg/kg body weight
5. Availability of the immunoglobulin G (IgG) trough levels of the two previous infusions before enrolment, and maintenance of at least 5.5 g/l in the trough levels of these two infusions
6. Negative result on a pregnancy test (human chorionic gonadotrophin [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
Participant type
Patient
Age group
Other
Gender
Both
Target number of participants
50
Participant 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 immunoglobulin A (IgA) in other products
3. Exposure to blood or any blood product or derivative, other than commercially available intravenous immunoglobulin (IVIG), within the past 3 months prior to enrolment
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 pre-medication for IVIG infusion
6. History of congenital impairment of pulmonary function
7. Severe liver function impairment (alanine aminotransferase [ALAT] 3 x upper limit of normal)
8. Presence of renal function impairment (creatinine greater than 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)
9. History of autoimmune haemolytic anaemia
10. History of diabetes mellitus
11. Congestive heart failure New York Heart Association (NYHA) class III or IV
12. Non-controlled arterial hypertension (systolic blood pressure greater than 160 mmHg or diastolic blood pressure greater than 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: human immunodeficiency virus (HIV), hepatitis C virus (HCV), hepatitis B virus (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, greater than or equal to 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 enrolment
19. Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to enrolment
20. Pregnant or nursing women
Recruitment start date
01/12/2009
Recruitment end date
01/04/2011
Locations
Countries of recruitment
Germany, Poland, United States of America
Trial participating centre
Oberlaaerstrasse 235
Vienna
1100
Austria
Sponsor information
Organisation
Octapharma AG (Switzerland)
Sponsor details
Seidenstrasse 2
Leiden
CH-8853
Switzerland
Sponsor type
Industry
Website
Funders
Funder type
Industry
Funder name
Octapharma AG (Switzerland)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list