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

Primary Immunodeficiency Diseases Clinical Trial

— Newgam  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01012323
• Other study ID #: NGAM01
• Status: Completed
• Phase: Phase 3
• First received: November 11, 2009
• Last updated: August 24, 2012
• Start date: November 2009
• Est. completion date: August 2012

Study information

• Verified date: August 2012
• Source: Octapharma
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 51
• Est. completion date: August 2012
• Est. primary completion date: June 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years to 75 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Primary Immunodeficiency Diseases

Intervention

Biological:
• NewGam
Intravenous Q 3 weeks, up to .08 Ml/kg
• NewGam
Q 4 weeks, Intravenous, up to .08 ml/kg

Locations

Country	Name	City	State
United States	Dr. Alan Knutsen	St. Louis	Missouri

Sponsors (2)

Lead Sponsor	Collaborator
Octapharma	Premier Research Group plc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To assess the efficacy of NewGam in preventing serious bacterial infections compared to historical control data.		Every study visit (every 3 (Q 3) or every 4 (Q 4) weeks)	No
Secondary	To evaluate the safety of NewGam.		Every study visit (Q 3 or Q 4 weeks)	Yes
Secondary	To determine the pharmacokinetic (PK) profile of NewGam.		After week 24 for Q 3 week patients and after week 32 for Q 4 weeks patients	No
Secondary	To assess the effect of NewGam on quality of life (QoL) measures.		5 times during the 52 week trial	No