Subcutaneous Ig NextGen 16% in PID Patients

A Multi-centre, Open-label Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Subcutaneous Infusions of Ig NextGen 16% in Patients With Primary Immunodeficiency (PID).

This study aims to assess the safety, tolerability, efficacy and pharmacokinetics of Ig NextGen 16% in people with antibody deficiency currently being treated with IntragamP. Ig NextGen 16% is a liquid immunoglobulin (antibody) preparation manufactured using predominately chromatographic techniques. Eligible patients will switch from monthly intravenous IntragamP therapy to weekly subcutaneous Ig NextGen 16% treatment. Initial hospital training will be required for subcutaneous administration and then the patient will perform the infusion in their own home, returning once a month for a supervised infusion. Patients will be monitored on the study for up to 10 months to assess blood IgG levels and rate of serious bacterial infections.

Study Overview

• Status: Completed
• Conditions: Primary Immunodeficiency (PID)
• Intervention / Treatment: Drug: IgNextGen 16%

• Study Type: Interventional
• Enrollment (Actual): 35
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Australian Capital Territory
    • Garran, Australian Capital Territory, Australia, 2605
      • The Canberra Hospital
  • New South Wales
    • New Lambton Heights, New South Wales, Australia, 2305
      • John Hunter Hospital
    • Randwick, New South Wales, Australia, 2031
      • Sydney Children's Hospital
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Royal Adelaide Hospital
    • North Adelaide, South Australia, Australia, 5006
      • Women's & Children's Hospital
  • Victoria
    • Frankston, Victoria, Australia
      • Frankston Hospital
    • Melbourne, Victoria, Australia, 3052
      • Royal Children's Hospital
  • Western Australia
    • Perth, Western Australia, Australia
      • Princess Margaret Hospital for Children
• New Zealand
  • Auckland, New Zealand
    • Starship Children's Hospital
  • Auckland, New Zealand
    • Auckland Hospital
  • Christchurch, New Zealand
    • Christchurch Hospital
  • Wellington, New Zealand
    • Wellington Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Inclusion Criteria:

  • Males or females 3 years of age or greater and at least 13 kg at enrolment.
  • PID patients receiving Ig replacement therapy, with a diagnosis of X-linked agammaglobulinemia (XLA) or Common Variable immunodeficiency (CVID) with severe hypogammaglobulinemia.
  • Patients who have received a consistent dose of Intragam®P at 3-, 4-, 5- or 6-weekly intervals, within the range of 0.2 - 0.6 g/kg body weight, for at least six months prior to the Screening visit.
  • Patients must have maintained IgG trough serum level of ≥ 5 g/L during the six months prior to Visit 0, with at least two trough levels to have been documented during this period.
  • Patients and/or their legally acceptable representative/guardian must give written informed consent to participate in the study and must understand the nature of the study and must be willing to comply with all protocol requirements

Exclusion Criteria:

• • Patients newly diagnosed with PID within six months of the Screening visit.

  • Patients with known or suspected severe hypersensitivity or previous evidence of severe side effects to immunoglobulin therapy or other blood products
  • Patients with known selective IgA deficiency or antibodies to IgA
  • Patients receiving immunosuppressive treatment other than topical and/or inhaled steroids and low dose oral steroids.
  • Females who are pregnant, breast feeding or planning a pregnancy during the course of the study. Females who are of child bearing potential must have a negative pregnancy test at screening.
  • Patients with protein-losing enteropathies, and kidney diseases with substantial proteinuria
  • Patients with malignancies of lymphoid cells such as chronic lymphocytic leukaemia, Non-Hodgkin's lymphoma and immunodeficiency with thymoma.
  • Patients who have within 30 days priors to the study screening visit, participated in a clinical study or used an investigational compound (eg: a new chemical entity not registered for clinical use).

  • Patients with any of the following abnormal lab results:

    • Serum creatinine >1.5 x Upper limit of Normal (ULN).
    • Serum ALT & AST > 2.5 x ULN.
    • Albumin < 25 g/L
  • Patients who are suffering from an acute or chronic medical condition, other than PID, which may, in the opinion of the Investigator, affect the conduct of the trial.
  • Patients who are not willing or are unable to comply with protocol.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ig NextGen 16%	Drug: IgNextGen 16%; IgNextGen 16% administered subcutaneously on a weekly basis from visit 1 to 12

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Efficacy	Continually from Visits 7 to 12 & monthly IgG troughs

Secondary Outcome Measures

Outcome Measure	Time Frame
Safety, Tolerability, Quality of Life, Pharmacokinetics	Visits 0, 6, 9, and12

Collaborators and Investigators

• Sponsor: CSL Limited
• Investigators: Principal Investigator: Marianne Empson, Dr, Auckland City Hospital

Publications and helpful links

General Publications

• Empson MB, Tang ML, Pearce LK, Rozen L, Gold MS, Katelaris CH, Langton D, Smart J, Smith WB, Steele RH, Ziegler JB, Maher D. Efficacy, safety and pharmacokinetics of a novel subcutaneous immunoglobulin, Evogam(R), in primary immunodeficiency. J Clin Immunol. 2012 Oct;32(5):897-906. doi: 10.1007/s10875-011-9641-4. Epub 2012 Apr 13.

Study record dates

Study Major Dates

• Study Start: April 1, 2007
• Primary Completion (Actual): October 1, 2009
• Study Completion (Actual): October 1, 2009

Study Registration Dates

• First Submitted: October 20, 2006
• First Submitted That Met QC Criteria: October 20, 2006
• First Posted (Estimate): October 23, 2006

Study Record Updates

• Last Update Posted (Estimate): June 7, 2012
• Last Update Submitted That Met QC Criteria: June 5, 2012
• Last Verified: June 1, 2012

More Information

Terms related to this study

• Keywords: Quality of Life; SCIG; PID; Ig; Serious Bacterial Infections
• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases
• Other Study ID Numbers: CSLCT-SCIG-05-23