Clinical Trial: A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

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

Official Title: A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Dose-range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients With

Brief Summary: This study will test the safety, tolerability, and pharmacokinetics of multiple escalating doses of ISIS-DMPKRx administered subcutaneously to adult patients with DM1.

Detailed Summary: This is a Phase 1/2a multicenter, blinded, placebo-controlled study of ISIS-DMPK Rx in adult patients with DM1.
Sponsor: Ionis Pharmaceuticals, Inc.

Current Primary Outcome:

  • Safety (The number of participants with adverse events) [ Time Frame: Patricipants will be followed for the duration of the study; an expected 24 - 32 weeks ]
    The number of participants with adverse events
  • Tolerability (The number of participants with adverse events) [ Time Frame: Patricipants will be followed for the duration of the study; an expected 24 -32 weeks ]
    The number of participants with adverse events


Original Primary Outcome:

  • Safety (The number of participants with adverse events) [ Time Frame: Patricipants will be followed for the duration of the study; an expected 24 weeks ]
    The number of participants with adverse events
  • Tolerability (The number of participants with adverse events) [ Time Frame: Patricipants will be followed for the duration of the study; an expected 24 weeks ]
    The number of participants with adverse events


Current Secondary Outcome:

  • Plasma Pharmacokinetics (Cmax, Tmax) [ Time Frame: Plasma at 0.5, 1, 1.5, 2, 4, 6, 8, 12 and 24 hours after dosing. ]
    • the maximal observed plasma drug concentration (Cmax)
    • the time to reach Cmax (Tmax)
    • the area under the plasma concentration time curve from the time of the subcutaneous dose to the last collected sample (24 hours after dosing)
  • Urine Pharmacokinetics (Amount of drug excreted in the urine) [ Time Frame: 0-24 hours post-dosing ]
    Amount of drug excreted in the urine


Original Secondary Outcome:

  • Plasma Pharmacokinetics (Cmax, Tmax) [ Time Frame: Plasma at 0.5, 1, 1.5, 2, 4, 6, 8, 12 and 24 hours after dosing. ]
    • the maximal observed plasma drug concentration (Cmax)
    • the time to reach Cmax (Tmax)
    • the area under the plasma concentration time curve from the time of the subcutanous dose to the last collected sample (24 hours after dosing)
  • Urine Pharmacokinetics (Amount of drug excreated in the urine) [ Time Frame: 0-24 hours post-dosing ]
    Amount of drug excreated in the urine


Information By: Ionis Pharmaceuticals, Inc.

Dates:
Date Received: December 4, 2014
Date Started: December 2014
Date Completion:
Last Updated: November 2, 2016
Last Verified: September 2016