Clinical trial NCT01012323: A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases
Official title: Clinical Study to Evaluate the Efficacy, Pharmacokinetics and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Patients With Primary Immunodeficiency Diseases
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Immunodeficiency Diseases
Keywords: PID
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
Collaborators: name: Premier Research Group plc
First posted: 2009-11-13
Enrollment: 51
Sex: ALL
Minimum age (years): 2
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam. The safety of NewGam and its effect on quality of life were also evaluated.

Detailed description: NewGam is a new 10% human normal immunoglobulin (IVIG) solution developed by Octapharma for intravenous administration. It is supplied as a liquid formulation ready to use. The primary therapeutic use of immunoglobulins is to provide antibodies to prevent viral and bacterial diseases (replacement therapy). IVIG has proved to be useful in a variety of clinical conditions other than for replacement of immunoglobulins; IVIG exhibits an immunomodulatory effect. Children and adults with a Primary Immunodeficiency Disease (PID) have an increased risk of recurrent bacterial and viral infections that typically attack the respiratory tract (sinusitis, bronchitis, pneumonia) but can also affect the gastrointestinal tract (gastroenteritis). Theses diseases can be severe and can lead to substantial morbidity. Responses to antibacterial therapy are often poor. At present, most primary immune deficiencies are not curable, but IVIGs have been shown to decrease the total number of severe infections and the duration of hospitalization.

Eligibility criteria: Inclusion Criteria:

* Age of ≥ 2 years and ≤ 75 years.
* Confirmed diagnosis of common variable immunodeficiency (CVID) or X-linked agammaglobulinemia (XLA).
* 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.

Exclusion Criteria:

* Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
* Exposure to blood or any blood product or derivative, other than commercially available intravenous immunoglobulin (IVIG), within the past 3 months prior to enrollment.
* Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product.
* Requirement of any routine pre-medication for IVIG infusion.
* Severe liver function impairment (alanine aminotransferase \[ALAT\] 3x \> upper limit of normal).
* Presence of renal function impairment (creatinine \> 120 μmol/L), or predisposition for acute renal failure (eg, any degree of pre-existing renal insufficiency or routine treatment with known nephritic drugs).
* History of autoimmune hemolytic anemia.
* History of diabetes mellitus.
* Congestive heart failure New York Heart Association (NYHA) class III or IV.
* Non-controlled arterial hypertension (systolic blood pressure \> 160 mmHg or diastolic blood pressure \> 90 mmHg).
* History of deep vein thrombosis or thrombotic complications of IVIG therapy.
* A positive result at screening on any of the following viral markers: human immunodeficiency virus (HIV), hepatitis C virus (HCV), hepatitis B virus (HBV).
* Treatment with steroids (oral or parenteral, long-term, ie, 30 days or more, not intermittent or burst, daily, ≥ 0.15 mg of prednisone or equivalent/kg/day), immunosuppressive or immunomodulatory drugs.
* Planned vaccination during the study period.
* Treatment with any investigational agent within 3 months prior to enrollment.
* Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to enrollment.
* Pregnant or nursing women.

Primary outcomes:
measure: Number of Serious Bacterial Infections Per Person-year of Treatment
timeFrame: Baseline to end of the study (up to 12 months)
Secondary outcomes:
measure: IgG Trough Level Concentration
timeFrame: Baseline to end of the study (up to 12 months)
measure: Trough Level Concentration of Antibodies Against Haemophilus Influenzae
timeFrame: Baseline to end of the study (up to 12 months)
measure: Trough Level Concentration of Antibodies Against Measles
timeFrame: Baseline to end of the study (up to 12 months)
measure: Trough Level Concentration of Antibodies Against Streptococcus Pneumoniae
timeFrame: Baseline to end of the study (up to 12 months)
measure: Trough Level Concentration of Antibodies Against Cytomegalovirus
timeFrame: Baseline to end of the study (up to 12 months)
measure: Trough Level Concentration of Antibodies Against Tetanus
timeFrame: Baseline to end of the study (up to 12 months)
measure: Trough Level Concentration of Antibodies Against Varicella-zoster Virus
timeFrame: Baseline to end of the study (up to 12 months)
measure: Total Number of Infections
timeFrame: Baseline to end of the study (up to 12 months)
measure: Number of Non-serious Infections
timeFrame: Baseline to end of the study (up to 12 months)
measure: Time to Resolution of Serious and Other Infections
timeFrame: Baseline to end of the study (up to 12 months)
measure: Percentage of Participants Treated With Antibiotics
timeFrame: Baseline to end of the study (up to 12 months)
measure: Number of Antibiotic Treatment Episodes Per Person-year of Treatment
timeFrame: Baseline to end of the study (up to 12 months)
measure: Number of Antibiotic Treatment Days Per Person-year of Treatment
timeFrame: Baseline to end of the study (up to 12 months)
measure: Number of Participants Hospitalized Due to an Infection
timeFrame: Baseline to end of the study (up to 12 months)
measure: Percentage of Participants With at Least 1 Episode of Fever
timeFrame: Baseline to end of the study (up to 12 months)
measure: Percentage of Participants That Missed School or Work Due to an Infection
timeFrame: Baseline to end of the study (up to 12 months)
measure: Changes in the Physical and Psychosocial Child Health Questionnaire-Parent Form Scores From Baseline to the End of the Study
timeFrame: Baseline to end of the study (up to 12 months)
measure: Changes in the Physical and Mental Short Form-36 Health Survey Scores From Baseline to the End of the Study
timeFrame: Baseline to end of the study (up to 12 months)
Other outcomes:
Locations:
city: Irvine
country: United States
facility: Sudir Gupta, MD
state: California
city: Centennial
country: United States
facility: Isaac Melamed, MD
state: Colorado
city: Chicago
country: United States
facility: James Moy, MD
state: Illinois
city: Fort Wayne
country: United States
facility: William Smits, MD
state: Indiana
city: St. Louis
country: United States
facility: Dr. Alan Knutsen
state: Missouri
city: Papillion
country: United States
facility: Ai Lan Kobayashi, MD
state: Nebraska
city: Seattle
country: United States
facility: Hans Ochs, MD
state: Washington
Study officials:
References: