Pharmacokinetics (PK) and Safety of Subgam®VF in Primary Immunodeficiency Diseases (SCIG03)

This study has suspended participant recruitment.
(Study suspended pending advice from the FDA)
Information provided by (Responsible Party):
Bio Products Laboratory Identifier:
First received: June 14, 2013
Last updated: January 29, 2014
Last verified: January 2014

The main objective of the study is to determine the pharmacokinetics profile of Subgam-VF. The secondary objectives are to assess the safety of Subgam-VF and refine the dose adjustment coefficient for Subgam-VF needed for subjects switching from prior intravenous immunoglobulin (IGIV) therapy.

Condition Intervention Phase
Primary Immune Deficiency Disorders
Common Variable Immunodeficiency
X-linked Agammaglobulinaemia
Hyper-Immunoglobulin M (IgM0Syndrome
Biological: Subgam
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: A Phase III, Multicenter, Open-Label Study to Evaluate the Pharmacokinetics and Safety of Subgam®VF in Primary Immunodeficiency Diseases

Resource links provided by NLM:

Further study details as provided by Bio Products Laboratory:

Primary Outcome Measures:
  • Determine the PK profile of Subgam-VF and compare the Area under the curve to a given treatment period (AUC [0-t)]) with historical data all standardized to 1 week at steady state from Gammaplex 5% IGIV Primary Immune studies (GMX01 and GMX04) [ Time Frame: 26 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Assess the safety of Subgam-VF including the incidence of adverse events (AEs) and site infusion reactions in subjects with primary immunodeficiency [ Time Frame: 26 weeks ] [ Designated as safety issue: Yes ]
    Adverse events: Number and percent of infusions associated with 1 or more AEs during the study and specifically AEs that begin during the infusion or within 72 hours after completion of the infusion.

  • Refine the dose adjustment coefficient for Subgam-VF [ Time Frame: Week 26 ] [ Designated as safety issue: No ]

Estimated Enrollment: 35
Study Start Date: January 2015
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Subgam-VF Biological: Subgam
Subgam-VF dose will be given as 1.45 of the established IGIV dose (expressed in mg/kg/week) for 26 weeks (26 infusions) beginning one week after the last IGIV infusion. Dose of Subgam-VF will then be adjusted based on the ratio of the Immunoglobulin G (IgG) average concentration achieved with Subgam-VF compared to IGIV.
Other Name: Subgam-VF


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

Inclusion criteria:

  1. Aged between 2 and 75 years.
  2. Diagnosed with primary immunodeficiency disease e.g. common variable immunodeficiency, X-linked and autosomal forms of agammaglobulinaemia, hyper-IgM syndrome.
  3. Currently receiving a licensed IGIV at a dose that has not changed by ± 50% of the mean dose for at least three months before study entry (Visit 1) and

    1. Dose is between 300 and 800 mg/kg/infusion.
    2. The infusion interval is between 21 and 28 days inclusive for IGIV.
    3. Has maintained a trough level of ≥ 5 g/L (500 mg/dL) for the last three months before Visit 1.
  4. Female subjects who are (or become) sexually active must practice contraception by using a method of proven reliability for the duration of the study.
  5. Females of child-bearing potential must have a negative result on an human chorionic gonadotropin (HCG)-based pregnancy test.
  6. Willing to comply with all aspects of the protocol for the duration of the study.
  7. Signed an informed consent form. In the case of subjects under the legal age the parent/guardian will sign an informed consent form and where appropriate the subject will sign an assent form.

Exclusion Criteria:

  1. Is currently using subcutaneous IgG (SCIG).
  2. Has a history of any severe anaphylactic reaction to blood or any blood-derived product.
  3. Has selective IgA deficiency, history of reaction to products containing IgA, or has a history of antibodies to immunoglobulin A (IgA).
  4. Has impaired cellular or innate immunity (i.e. only subjects with impaired humoral immunity to be included)
  5. Has evidence of an active infection at the time of enrolment.
  6. Has previously completed or withdrawn from this study.
  7. Is currently receiving, or has received, any investigational agent within the prior three months.
  8. Is pregnant (confirmed by a positive result on an HCG-based pregnancy test) or is nursing.
  9. Is positive for any of the following at screening:

    • Serological test for Human Immunodeficiency Virus (HIV) 1&2, hepatitis C virus (HCV), or Hepatitis B Surface Antigen (HBsAg)
    • Nucleic acid Amplification Test [Polymerase Chain Reaction - PCR] (NAT) for HCV
    • NAT for HIV
  10. Has levels at screening greater than 2.5 times the upper limit of normal as defined at the central laboratory of any of the following:

    • Alanine transaminase (ALT)
    • Aspartate transaminase (AST)
  11. Has severe renal impairment (defined as serum creatinine greater than two times the upper limit of normal or blood urea nitrogen (BUN) greater than 2.5 times the upper limit of normal for the range of the laboratory doing the analysis); the subject is on dialysis; or has a history of acute renal failure.
  12. Is known to abuse alcohol, opiates, psychotropic agents, or other chemicals or drugs, or has done so within the past 12 months.
  13. Has a history of deep vein thrombosis (DVT), or thrombotic complications of IGIV therapy.
  14. Suffers from any acute or chronic medical condition, (e.g. renal disease or predisposing conditions for renal disease, coronary artery disease, or protein losing state, proteinuria) that the investigator feels may interfere with the conduct of the study.
  15. Has an acquired medical condition, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (ANC < 1000 x 109/L).
  16. Is receiving the following medication:

    • Steroids (long-term daily, > 0.15 mg of prednisone equivalent/kg/day). Requirement for short or intermittent courses would not exclude a subject.
    • Immunosuppressive drugs
    • Immunomodulatory drugs
  17. Has non-controlled arterial hypertension (systolic blood pressure > 160 mmHg and/or diastolic blood pressure > 100 mmHg).
  18. Has anemia (hemoglobin < 10 g/dL) at screening.
  19. Has severe dermatitis that would preclude sites for safe product administration.
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Please refer to this study by its identifier: NCT01884311

Sponsors and Collaborators
Bio Products Laboratory
Study Director: Tim J. Aldwinckle, MD Bio Products Laboratory Limited
  More Information

No publications provided

Responsible Party: Bio Products Laboratory Identifier: NCT01884311     History of Changes
Other Study ID Numbers: SCIG03
Study First Received: June 14, 2013
Last Updated: January 29, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Bio Products Laboratory:
Primary Immune Deficiency Disorders
Common Variable Immunodeficiency
X-linked agammaglobulinaemia
Hyper-IgM Syndrome

Additional relevant MeSH terms:
Common Variable Immunodeficiency
Genetic Diseases, X-Linked
Immunologic Deficiency Syndromes
Blood Protein Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Immune System Diseases
Lymphatic Diseases
Lymphoproliferative Disorders processed this record on May 20, 2015