Clinical Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Patients With Primary Immunodeficiency Diseases - Full Text View

Purpose

The purpose of this study is to determine the efficacy of NewGam in preventing serious bacterial infections, and on quality of life. The safety and pharmacokinetic profile of NewGam will also be evaluated.

Condition	Intervention	Phase
Primary Immunodeficiency Diseases	Biological: NewGam	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Clinical Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam)in Patients With Primary Immunodeficiency Diseases

Resource links provided by NLM:

Genetics Home Reference related topics: complement factor I deficiency

MedlinePlus related topics: Bacterial Infections

U.S. FDA Resources

Further study details as provided by Octapharma:

Primary Outcome Measures:

To assess the efficacy of NewGam in preventing serious bacterial infections compared to historical control data. [ Time Frame: Every study visit (every 3 (Q 3) or every 4 (Q 4) weeks) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To evaluate the safety of NewGam. [ Time Frame: Every study visit (Q 3 or Q 4 weeks) ] [ Designated as safety issue: Yes ]
To determine the pharmacokinetic (PK) profile of NewGam. [ Time Frame: After week 24 for Q 3 week patients and after week 32 for Q 4 weeks patients ] [ Designated as safety issue: No ]
To assess the effect of NewGam on quality of life (QoL) measures. [ Time Frame: 5 times during the 52 week trial ] [ Designated as safety issue: No ]

Enrollment:	51
Study Start Date:	November 2009
Study Completion Date:	August 2012
Primary Completion Date:	June 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: Newgam Q 3 weeks	Biological: NewGam Intravenous Q 3 weeks, up to .08 Ml/kg Biological: NewGam Q 4 weeks, Intravenous, up to .08 ml/kg
Active Comparator: NewGam Q 4 weeks	Biological: NewGam Intravenous Q 3 weeks, up to .08 Ml/kg Biological: NewGam Q 4 weeks, Intravenous, up to .08 ml/kg

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. IVIG has proved to be useful in a variety of clinical conditions other than for replacement of immunoglobulins, in which IVIG exhibits an immunomodulatory effect. Children and adults with PID have an increased risk of getting recurrent bacterial and viral infections that typically attack the respiratory tract (sinusitis, bronchitis, pneumonia) but can also affect the gastrointestinal tract (gastroenteritis). They 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

Ages Eligible for Study:	2 Years to 75 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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 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 (e.g. 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, i.e. 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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01012323

Locations

United States, Missouri
Dr. Alan Knutsen
St. Louis, Missouri, United States, 63104

Sponsors and Collaborators

Octapharma

Premier Research

More Information

No publications provided

Responsible Party:	Octapharma
ClinicalTrials.gov Identifier:	NCT01012323 History of Changes
Other Study ID Numbers:	NGAM01
Study First Received:	November 11, 2009
Last Updated:	August 24, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Octapharma:

PID

Additional relevant MeSH terms:

Immunologic Deficiency Syndromes
Immune System Diseases
Immunoglobulins
Antibodies

Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 22, 2013

Clinical Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Patients With Primary Immunodeficiency Diseases (Newgam)