Clinical Trial: Phase III Efficacy, Safety, and Tolerability Study of HYQVIA/HyQvia and GAMMAGARD LIQUID/KIOVIG in CIDP

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Immune Globulin Infusion 10% (Human) With Recombinant Human Hyaluronidase (HYQVIA/HyQvia) and Immune Globulin Infusion (Human),

Brief Summary: This is a Phase III, prospective, multicenter study with two study parts (epochs) to evaluate the efficacy, safety, and tolerability of HYQVIA/HyQvia (IGI, 10% with recombinant human hyaluronidase (rHuPH20) administered subcutaneously) for maintenance therapy to prevent relapse (Epoch 1) and GAMMAGARD LIQUID/KIOVIG (immunoglobulin G intravenous (IGIV) 10% administered intravenously) for the treatment of CIDP (Epoch 2). This study will enroll adult subjects with a confirmed diagnosis of CIDP and who have remained on a stable dosing regimen (monthly equivalent dose of 0.4 to 2.4 g/kg body weight (BW) with a dosing interval of 2 to 6 weeks) of IGIV therapy for at least 3 months prior to screening. In this double-blind, placebo-controlled phase of the study (Epoch 1), eligible subjects will be randomized in a 1:1 ratio to receive either HYQVIA/HyQvia or 0.25% albumin placebo solution with rHuPH20 every two, three, or four weeks in a double-blind fashion for a period of 6 months or until relapse. Subjects who relapse during Epoch 1 will enter Epoch 2 to receive IGIV treatment for a period of 6 months.

Detailed Summary:
Sponsor: Baxalta US Inc.

Current Primary Outcome:

  • Epoch 1: Relapse rate (proportion of participants who experience a worsening of functional disability) [ Time Frame: Screening; Pre-SC Baseline; AND participants on: 1) Q2W: week 15 & 27; End of Epoch 1 (EOE1); Unscheduled relapse visit assessment (UV); & if early termination occurs (ET). 2) Q3W: week 14 & 26; EOE1; UV; and ET. 3) Q4W: week 16 & 28; EOE1; UV; and ET. ]
    Worsening of functional disability defined as an increase of ≥1 point relative to the pre- subcutaneous (SC) treatment baseline score in 2 consecutive adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale.
  • Epoch 2: Responder rate (proportion of participants with clinically meaningful improvement in functional ability) [ Time Frame: Epoch 2: Pre-IV Baseline and every 3 weeks through end of Epoch 2 (6 months) ]
    Clinically meaningful improvement in functional ability defined as a decrease of ≥1 point in the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score at the completion of the intravenous (IV) treatment period [6 months] or at the last study visit of the IV treatment period, relative to the pre-IV treatment baseline score


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Epoch 1: Time to relapse [ Time Frame: Throughout Epoch 1, up to 6 months ]
    Defined as time from the date of the first SC administration of HYQVIA/HyQvia or placebo with rHuPH20 to the date of relapse
  • Epoch 1: Change from pre-subcutaneous (SC) treatment baseline in Rasch-built Overall Disability Scale (R-ODS) score [ Time Frame: Pre-subcutaneous (SC) treatment baseline, then weekly through Epoch 1 (up to 6 months); Unscheduled relapse visit assessment; & if early termination occurs. ]
    The Rasch-Built Overall Disability Scale (R-ODS) is a patient self-reported, linearly-weighted overall disability scale that was specifically designed to capture activity and social participation limitations in patients with immune-mediated peripheral neuropathies including chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The R-ODS is comprised of 24 items for which subjects are asked to rate their functioning (ie, no difficulty, some difficulty, or could not do) related to a variety of everyday tasks at the moment of completion.
  • Epoch 1: Number of participants experiencing any treatment-emergent serious and/or non-serious adverse events (SAEs and/or AEs), regardless of causality [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of participants experiencing causally related serious and/or non-serious adverse events (SAEs and/or AEs) [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of participants with serious and/or non-serious adverse reactions (ARs) plus suspected ARs [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of infusions associated with treatment-emergent serious and/or non-serious adverse events (SAEs and/or AEs), regardless of causality [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of infusions associated with causally related serious and/or non-serious adverse events (SAEs and/or AEs) [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of infusions temporally associated with adverse events (AEs) [ Time Frame: During an infusion or within 72 hours after completion of an infusion ]
    AEs occurring during an infusion or within 72 hours after completion of an infusion
  • Epoch 1: Number of infusions associated with serious and/or non-serious adverse reactions (ARs) plus suspected ARs [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of infusions associated with treatment-emergent systemic adverse events (AEs) [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of infusions associated with treatment-emergent local infusion site reactions [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of infusions for which the infusion rate was reduced and/or the infusion was interrupted or stopped due to intolerability and/or adverse events (AEs) [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of systemic and local adverse events (AEs), regardless of causality, expressed as number of events per infusion [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of systemic and local adverse events (AEs), regardless of causality, expressed as number of events per participant [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of systemic and local adverse events (AEs), regardless of causality, expressed as number of events per participant-year [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of causally related systemic and local adverse events (AEs), expressed as number of events per infusion [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of causally related systemic and local adverse events (AEs), expressed as number of events per participant [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of causally related systemic and local adverse events (AEs), expressed as number of events per participant-year [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of systemic and local adverse reactions (ARs) plus suspected ARs, expressed as number of events per infusion [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of systemic and local adverse reactions (ARs) plus suspected ARs, expressed as number of events per participant [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Rates of systemic and local adverse reactions (ARs) plus suspected ARs, expressed as number of events per participant-year [ Time Frame: Throughout Epoch 1, up to 6 months ]
  • Epoch 1: Number of participants who develop binding and/or neutralizing antibodies to Recombinant human hyaluronidase (rHuPH20) [ Time Frame: Througho

    Original Secondary Outcome:

    • Epoch 1: Time to relapse [ Time Frame: Throughout Epoch 1, up to 6 months ]
      Defined as time from the date of the first SC administration of HYQVIA/HyQvia or placebo with rHuPH20 to the date of relapse
    • Epoch 1: Change from pre-subcutaneous (SC) treatment baseline in Rasch-built Overall Disability Scale (R-ODS) score [ Time Frame: Pre-subcutaneous (SC) treatment baseline, then weekly through Epoch 1 (up to 6 months); Unscheduled relapse visit assessment; & if early termination occurs. ]
      The Rasch-Built Overall Disability Scale (R-ODS) is a patient self-reported, linearly-weighted overall disability scale that was specifically designed to capture activity and social participation limitations in patients with immune-mediated peripheral neuropathies including chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The R-ODS is comprised of 24 items for which subjects are asked to rate their functioning (ie, no difficulty, some difficulty, or could not do) related to a variety of everyday tasks at the moment of completion.
    • Epoch 1: Number of participants experiencing any treatment-emergent serious and/or non-serious adverse events (SAEs and/or AEs), regardless of causality [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of participants experiencing causally related serious and/or non-serious adverse events (SAEs and/or AEs) [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of participants with serious and/or non-serious adverse reactions (ARs) plus suspected ARs [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of infusions associated with treatment-emergent serious and/or non-serious adverse events (SAEs and/or AEs), regardless of causality [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of infusions associated with causally related serious and/or non-serious adverse events (SAEs and/or AEs) [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of infusions temporally associated with adverse events (AEs) [ Time Frame: During an infusion or within 72 hours after completion of an infusion ]
      AEs occurring during an infusion or within 72 hours after completion of an infusion
    • Epoch 1: Number of infusions associated with serious and/or non-serious adverse reactions (ARs) plus suspected ARs [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of infusions associated with treatment-emergent systemic adverse events (AEs) [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of infusions associated with treatment-emergent local infusion site reactions [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of infusions for which the infusion rate was reduced and/or the infusion was interrupted or stopped due to intolerability and/or adverse events (AEs) [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of systemic and local adverse events (AEs), regardless of causality, expressed as number of events per infusion [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of systemic and local adverse events (AEs), regardless of causality, expressed as number of events per participant [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of systemic and local adverse events (AEs), regardless of causality, expressed as number of events per participant-year [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of causally related systemic and local adverse events (AEs), expressed as number of events per infusion [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of causally related systemic and local adverse events (AEs), expressed as number of events per participant [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of causally related systemic and local adverse events (AEs), expressed as number of events per participant-year [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of systemic and local adverse reactions (ARs) plus suspected ARs, expressed as number of events per infusion [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of systemic and local adverse reactions (ARs) plus suspected ARs, expressed as number of events per participant [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Rates of systemic and local adverse reactions (ARs) plus suspected ARs, expressed as number of events per participant-year [ Time Frame: Throughout Epoch 1, up to 6 months ]
    • Epoch 1: Number of participants who develop binding and/or neutralizing antibodies to Recombinant human hyaluronidase (rHuPH20) [ Time Frame: Througho

      Information By: Baxalta US Inc.

      Dates:
      Date Received: September 11, 2015
      Date Started: December 2015
      Date Completion: January 2019
      Last Updated: August 23, 2016
      Last Verified: August 2016