Clinical Trial: Study of the Efficacy and Safety of Immune Globulin Intravenous (Human) Flebogamma® 5% DIF in Patients With Post-polio Syndrome

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

Official Title: A Multicenter, Prospective, Randomized, Placebo-controlled, Double-blind, Parallel‑Group Clinical Trial to Assess the Efficacy and Safety of Immune Globulin Intravenous (Human) Flebogamma®

Brief Summary:

This is a multicenter, prospective, randomized, placebo-controlled, double-blind, parallel group clinical trial with adaptive dose selection in subjects with post polio syndrome (PPS).

The main purpose of this study is to select a dose of Flebogamma 5% DIF and confirm the efficacy of the selected Flebogamma 5% DIF dose by assessing physical performance, as measured by 2 Minutes Walk Distance (2MWD) test.


Detailed Summary:

This is a phase II/III multicentre, prospective, randomized, placebo-controlled, double-blind, parallel‑group clinical trial with an adaptive design (flexible group sequential design with adaptive dose selection) in subjects with PPS.

This study will consist of two stages. The first stage (Stage 1) is for dose selection, and the second stage (Stage 2) is to establish the superiority (efficacy confirmation) of Flebogamma® 5% DIF and for overall safety analysis. At Stage 1, three treatment arms, Flebogamma® 5% DIF 2 g/kg of body weight (IVIG 2 g/kg arm), or the equivalent volume of Normal Saline Solution (40 ml/kg of body weight), or Flebogamma® 5% DIF 1 g/kg of body weight plus the equivalent volume of Normal Saline Solution (20 ml/kg of body weight) (IVIG 1 g/kg arm) will be administered every 4 weeks over two consecutive days during a 52-week treatment period. At Stage 2, two treatment arms, the selected dose of Flebogamma® 5% DIF from Stage 1 and Normal Saline Solution (40 ml/kg of body weight), will be administered every 4 weeks over two consecutive days during a 52-week treatment period. During Stage 2, the selected dose of Flebogamma® 5% DIF and Normal Saline Solution will be administered in the same manner as in Stage 1, including administering the total dose for both treatment arms at a volume equivalent to that for the IVIG 2 g/kg arm, regardless of the selected dose.

Primary efficacy endpoint will be:

  • Physical performance (2MWD) from baseline to the end of the treatment period (at End of Treatment Visit -Week 52).

Secondary efficacy endpoints will be:

  • Pain (VAS of pa
    Sponsor: Instituto Grifols, S.A.

    Current Primary Outcome: Change from baseline in 2MWD [ Time Frame: Baseline, Week 52 ]

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Change from baseline in Visual Analogue Scale (VAS) of pain [ Time Frame: Baseline, Week 52 ]
    • Change from baseline in Medical Outcomes Study 36-Item Short Form Health Survey (SF-36) Physical Component Summary (PCS) [ Time Frame: Baseline, Week 52 ]
    • Change from baseline in Six Minutes Walk Distance (6MWD) [ Time Frame: Baseline, Week 52 ]


    Original Secondary Outcome: Same as current

    Information By: Grifols Biologicals Inc.

    Dates:
    Date Received: June 25, 2014
    Date Started: July 2014
    Date Completion: December 2018
    Last Updated: April 5, 2016
    Last Verified: April 2016