A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases

NCT ID: NCT01012323

Last Updated: 2017-03-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 51 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-01-31
• Study Completion Date: 2012-06-30

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.

Conditions

Primary Immunodeficiency Diseases

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

NewGam

Participants received NewGam 200-800 mg/kg intravenously every 3 weeks (17 infusions) or 4 weeks (13 infusions) for 1 year.

Group Type: EXPERIMENTAL

NewGam

Intervention Type: BIOLOGICAL

The dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as minimum trough levels of serum immunoglobulin G (IgG) was above 5 g/L. If serum IgG trough levels dropped to 5 g/L or less, the dose was to be adjusted at the investigator's discretion. NewGam was supplied as a solution for infusion.

Interventions

NewGam

The dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as minimum trough levels of serum immunoglobulin G (IgG) was above 5 g/L. If serum IgG trough levels dropped to 5 g/L or less, the dose was to be adjusted at the investigator's discretion. NewGam was supplied as a solution for infusion.

Intervention Type: BIOLOGICAL

Other Intervention Names

Human normal immunoglobulin

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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Premier Research Group plc

UNKNOWN

Sponsor Role: collaborator

Octapharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Sudir Gupta, MD

Irvine, California, United States

Isaac Melamed, MD

Centennial, Colorado, United States

James Moy, MD

Chicago, Illinois, United States

William Smits, MD

Fort Wayne, Indiana, United States

Dr. Alan Knutsen

St Louis, Missouri, United States

Ai Lan Kobayashi, MD

Papillion, Nebraska, United States

Hans Ochs, MD

Seattle, Washington, United States

Countries

United States

Other Identifiers

2009-011434-10

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

NGAM-01

Identifier Type: -

Identifier Source: org_study_id