Clinical Trial: Safety Study of an Immunomodulating Microparticle to Treat Progressive Multiple Sclerosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase 2, Open-label, Dose-escalation Study Evaluating the Safety, Tolerability, and Pharmacodynamics of Intravenously Administered MIS416 in Patients With Chronic Progressive M

Brief Summary: The purpose of the study is to determine the safety and tolerability, dose-limiting toxicities, maximum tolerated dose, and recommended therapeutic dose of intravenously administered MIS416 weekly in patients with chronic progressive multiple sclerosis.

Detailed Summary:

This is a single center, open-label, non-randomized, dose-escalation study, to be conducted in two phases:

  • a dose-escalation (DE) phase, to evaluate the safety, tolerability, MTD, and PD of MIS416 administered IV once weekly for 4 doses; and
  • a dose-confirmation (DC) phase, which will be a cohort expansion at or below the MTD (i.e., the RTD) of MIS416, dosed once weekly for up to 12 doses.

Subjects will be treated with a weekly IV dose of MIS416 in 28-day cycles: 1 cycle in the DE phase, and up to 3 cycles in the DC phase. Subjects will be evaluated and dosed weekly each cycle in each phase. Subjects will return for a follow-up visit 7 days after completion of the last dose of study drug.

The primary objectives of this study are:

  1. To determine the safety and tolerability, dose-limiting toxicities (DLTs), maximum tolerated dose (MTD), and recommended therapeutic dose (RTD) of intravenously (IV) administered MIS416 weekly in patients with chronic progressive multiple sclerosis (CPMS); and
  2. To assess the pharmacodynamic (PD) effects of MIS416, including effects on serum cytokine levels and peripheral blood mononuclear cell (PBMC) composition, cytokine/chemokine expression and function.

The secondary objectives of this study are:

  1. To document any changes in MS clinical status occurring during the 12-week MIS416 dosing period in the dose-confirmation phase, as determined by the Multiple Sclerosis Functional Composite (MSFC), Fatigue Severity Scale (FSS), Shor
    Sponsor: Innate Immunotherapeutics

    Current Primary Outcome: Safety profile, including maximum tolerated dose [ Time Frame: 1 month in DE phase, 3 months in DC phase ]

    Dose-limiting toxicities, adverse events, safety MRI assessments


    Original Primary Outcome: Safety profile, including maximum tolerated dose [ Time Frame: 1 month in DE phase, 6 months in DC phase ]

    Dose-limiting toxicities, adverse events, safety MRI assessments


    Current Secondary Outcome:

    • Pharmacodynamic assessments [ Time Frame: 1 month in DE phase, 3 months in DC phase ]
      Serum and cellular immunological assays
    • MRI assessments [ Time Frame: 1 month in DE phase, 3 months in DC phase ]
      Safety MRIs
    • Clinical status [ Time Frame: 3 months in DC phase ]
      Neurological examination, Expanded Disability Status Scale (EDSS), Multiple Sclerosis Functional Composite (MSFC), Fatigue Severity Scale (FSS), Multiple Sclerosis Quality of Life (MSQLI).


    Original Secondary Outcome:

    • Pharmacodynamic assessments [ Time Frame: 1 month in DE phase, 6 months in DC phase ]
      Serum and cellular immunological assays
    • MRI assessments [ Time Frame: 1 month in DE phase, 6 months in DC phase ]
      Safety MRIs
    • Clinical status [ Time Frame: 6 months in DC phase ]
      Neurological examination, Expanded Disability Status Scale (EDSS), Multiple Sclerosis Functional Composite (MSFC)


    Information By: Innate Immunotherapeutics

    Dates:
    Date Received: August 30, 2010
    Date Started: August 2010
    Date Completion:
    Last Updated: December 5, 2012
    Last Verified: December 2012