Efficacy and Safety of IONIS-PKKRx for Preventive Treatment of Chronic Migraine
Condition: Chronic Migraine
MeSH terms: Migraine Disorders
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to evaluate the safety, tolerability, and changes in the number
      of migraine and headache days with repeated subcutaneous administration of IONIS-PKKRx (ISIS
      546254) or placebo in subjects with chronic migraine.
    

Detailed description: 
      This is a double-blind, placebo-controlled, randomized, multi-center study in subjects with
      chronic migraine. The study will consist of 7 office visits, 6 Sample collection visits and 3
      phone call assessments. Subjects agreeing to participate in the study and meeting the entry
      criteria assessed at the screening visit, will begin a 28 day baseline period to confirm
      their diagnosis, and establish a baseline frequency of migraine and headache days. During the
      baseline period, subjects will continue treating their migraines in their usual manner. They
      will monitor headache activity, migraine related symptoms, and medication usage with an
      electronic daily headache diary.

      Subjects who, after completing the baseline, continue to meet entrance criteria will be
      eligible to enter into the 4 month treatment phase. They will be randomized according to the
      Clinvest generated randomization schedule. A total of 30 randomized subjects will enter the
      treatment phase receiving IONIS-PKKRx (ISIS 546254; SC) or placebo in a 1:1 design. Study
      drug or placebo will be administered weekly for 16 weeks. A short phone call to assess any
      treatment related adverse events will take place 1 and 2 days after randomization. Daily
      electronic diary assessments will collect headache frequency and severity, associated
      migraine symptoms, acute medication usage, and the emergence of unusual symptoms and adverse
      events. Subjects will return to the site at weeks 4, 8, and 12 for investigational product
      (IP) accountability/dispensing, medication and medication updates, biomarker/lab sample
      collection, and assessment of adverse events. An end of treatment visit will take place 16
      weeks after randomization.

      Subjects will have a follow-up safety visit one month after their last dosage of IP ) for
      assessment of any adverse events (AE) and satisfaction and a final safety phone call 2 months
      following their last office visit (3 months after last dose of IP) for assessment of any
      adverse events (AE). Subjects will continue to complete headache diaries through Visit 7.
      Subjects will also have hematology samples collected every other week starting after Visit 2
      through Day 154.

      Safety and tolerability will be monitored by the Investigators. Patients who discontinue
      study treatment prematurely should complete any follow-up visits associated with the most
      recent dose and should move into and complete the Follow-up Phase.

      Subjects will undergo sampling for (pharmacokinetics) PK, coagulation, chemistry, hematology,
      and optional future biomedical research, as specified in the schedule of procedures.
    

Intervention name(s): IONIS-PKKRx (ISIS 546254); Placebo
Intervention type(s): Drug; Other
Intervention description(s): Those randomized to IONIS-PKKRx (ISIS 546254) will receive subcutaneous injections containing 1.00 mL (200mg) of IONIS- PKKRx (ISIS 546254) weekly for weeks 1-16.; Those randomized to placebo will receive subcutaneous injections 1.00 mL (200mg) weekly for weeks 1-16.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary purpose: Treatment
Keywords: migraine