Clinical study to evaluate the efficacy, pharmacokinetics and safety of immunoglobulin intravenous (human) 10% (NewGam) in patients with primary immunodeficiency diseases

ISRCTN	ISRCTN05425999
DOI	https://doi.org/10.1186/ISRCTN05425999
Secondary identifying numbers	NGAM-01

Submission date: 09/11/2009
Registration date: 11/11/2009
Last edited: 12/11/2009

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Ms Barbara Pyringer
Scientific

Oberlaaerstrasse 235
Vienna
1100
Austria

Study information

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
Study setting(s)	Hospital
Study type	Treatment
Participant information sheet	Not available in web format, please use the contact details below to request a patient information sheet
Scientific title
Study objectives	To assess the efficacy of NewGam in preventing serious bacterial infections compared to historical control data.
Ethics approval(s)	Saint Louis University Biomedical Institutional Review Board approved on the 15th September 2009 (ref: 16291)
Health condition(s) or problem(s) studied	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
Pharmaceutical study type(s)
Phase	Phase III
Drug / device / biological / vaccine name(s)	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 study start date	01/12/2009
Completion date	01/04/2011

Eligibility

Participant type(s)	Patient
Age group	Other
Sex	Both
Target number of participants	50
Key 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
Key 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
Date of first enrolment	01/12/2009
Date of final enrolment	01/04/2011

Locations

Countries of recruitment

Austria
Germany
Poland
United States of America

Study participating centre

Oberlaaerstrasse 235

Vienna
1100
Austria

Sponsor information

Octapharma AG (Switzerland)
Industry

Seidenstrasse 2
Leiden
CH-8853
Switzerland

Website	http://www.octapharma.com
"ROR"	https://ror.org/002k5fe57

Funders

Funder type

Industry

Octapharma AG (Switzerland)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan