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

NCT01012323 | Completed Phase 3 Results DMC

• 2 other identifiers: NGAM-012009-011434-10
• Study Type: interventional
• Target: 51
• Locations: 1 country
• Sites: 7

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.

Conditions

Primary Immunodeficiency Diseases

Keywords

PID

Outcome Measures

Primary Outcomes (1)

• Number of Serious Bacterial Infections Per Person-year of Treatment

  The number of serious bacterial infections per person-year of treatment was calculated by the following formula: Total number of serious bacterial infections / patient-years on NewGam treatment. Serious bacterial infections were defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess.

  Baseline to end of the study (up to 12 months)

Secondary Outcomes (18)

• IgG Trough Level Concentration

  Baseline to end of the study (up to 12 months)

• Trough Level Concentration of Antibodies Against Haemophilus Influenzae

  Baseline to end of the study (up to 12 months)

• Trough Level Concentration of Antibodies Against Measles

  Baseline to end of the study (up to 12 months)

• Trough Level Concentration of Antibodies Against Streptococcus Pneumoniae

  Baseline to end of the study (up to 12 months)

• Trough Level Concentration of Antibodies Against Cytomegalovirus

  Baseline to end of the study (up to 12 months)

Study Arms (1)

NewGam

EXPERIMENTAL

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

Biological: NewGam

Interventions

NewGam 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.

Also known as: Human normal immunoglobulin

NewGam

Eligibility Criteria

• Age: 2 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Octapharma: lead
• Premier Research: collaborator

Study Sites (7)

Sudir Gupta, MD

Irvine, California, United States

Location

Isaac Melamed, MD

Centennial, Colorado, United States

Location

James Moy, MD

Chicago, Illinois, United States

Location

William Smits, MD

Fort Wayne, Indiana, United States

Location

Dr. Alan Knutsen

St Louis, Missouri, 63104, United States

Location

Ai Lan Kobayashi, MD

Papillion, Nebraska, United States

Location

Hans Ochs, MD

Seattle, Washington, United States

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Interventions

gamma-Globulins

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Results Point of Contact

• Title: Michael Eppolito, Director, Clinical Operations Immunology and ICU Medicine
• Organization: Octapharma USA

michael.eppolito@octapharma.com

201 604-1155

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 11, 2009
• First Posted: November 13, 2009
• Study Start: January 1, 2010
• Primary Completion: June 1, 2012
• Study Completion: June 1, 2012
• Last Updated: March 16, 2017
• Results First Posted: March 16, 2017

Record last verified: 2017-02

Locations

US (7)