Efficacy and Safety of IONIS-PKKRx for Preventive Treatment of Chronic Migraine

NCT03108469 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 15-001IS
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Chronic Migraine

Keywords

migraine

Outcome Measures

Primary Outcomes (1)

• Migraine Days

  Compare the efficacy of ISIS 546254 in the preventive treatment of chronic migraine, measured by the number of monthly migraine days comparing baseline to the final month of the 4-month treatment period for subjects treated with ISIS 546254 vs. placebo.

  Comparing baseline to treatment period, up to 16 weeks

Secondary Outcomes (7)

• Headache Severity

  Comparing baseline to treatment period, up to 16 weeks

• Headache Days

  Comparing baseline to treatment period, up to 16 weeks

• Number of Subjects Reporting a ≥ 50% Reduction in the Number of Migraine Headaches

  Comparing baseline to treatment period, up to 16 weeks

• Count of Migraine Headache Days Requiring Use of Migraine Medication

  Comparing baseline to treatment period, up to 16 weeks

• Migraine Specific Quality of Life (MSQ) Questionnaire Score

  Comparing baseline to treatment period, up to 16 weeks

Study Arms (2)

IONIS-PKKRx (ISIS 546254)

ACTIVE COMPARATOR

Those randomized to IONIS-PKKRx (ISIS 546254) will receive subcutaneous injections containing 1.00 mL (200mg) weekly for weeks 1-16.

Drug: IONIS-PKKRx (ISIS 546254)

Placebo

PLACEBO COMPARATOR

Those randomized to placebo will receive subcutaneous injections containing 1.00 mL (200mg) weekly for weeks 1-16.

Other: Placebo

Interventions

IONIS-PKKRx (ISIS 546254) DRUG

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.

IONIS-PKKRx (ISIS 546254)

Placebo OTHER

Those randomized to placebo will receive subcutaneous injections 1.00 mL (200mg) weekly for weeks 1-16.

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Potential subjects must meet the following criteria at the screening visit to enter this study:
• male or female, in otherwise good health, 18 to 65 years of age.
• history of chronic migraine meeting the diagnostic criteria listed in the International Classification of Headache Disorders (ICHD-III beta version, 2013), as follows:
• a. History of frequent headaches suggestive of chronic migraine (15 or greater days of qualifying headaches per month) for at least three months prior to screening b. Verification of headache frequency through prospectively collected baseline information during the 28-day run-in phase demonstrating headaches on at least 15 days, with at least 8 days per month fulfilling any ONE of the following; i. Qualify as being a migraine attack ii. Relieved by migraine specific acute medications
• onset of migraine before age 50.
• stable pattern of migraine pattern for at least 3 months prior to screening.
• not currently taking a migraine preventive OR has been taking a stable dose of a preventive for at least 30 days prior to screening and agrees to not start, stop, or change medication and/or dosage during the study period.
• i. Subjects on migraine preventative should have stable headache pattern ii. Injections of onabotulinumtoxinA are allowable if subject has completed at least 2 injection cycles and agrees to maintain a regular injection cycle for the duration of the study

You may not qualify if:

• Potential subjects meeting any of the following criteria will be excluded from entering this study:
• unable to understand the study requirements, the informed consent, or complete headache records as required per protocol.
• pregnant, actively trying to become pregnant, or breast-feeding.
• history of medication overuse (MO) of opioids, or butalbital, as defined by ICHD-3 beta criteria and/or MO during baseline period.
• history of substance abuse and/or dependence, in the opinion of the Investigator.
• unstable neurological condition or a significantly abnormal neurological examination with focal signs or signs of increased intracranial pressure.

Sponsors & Collaborators

• Smith, Timothy R., M.D.: lead
• Clinvest Research, LLC: collaborator
• Ionis Pharmaceuticals, Inc.: collaborator

Study Sites (2)

StudyMetrix

City of Saint Peters, Missouri, 63303, United States

Location

Clinvest Research, LLC

Springfield, Missouri, 65807, United States

Location

MeSH Terms

Conditions

Migraine Disorders

Condition Hierarchy (Ancestors)

Headache Disorders, Primary; Headache Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Results Point of Contact

• Title: Jim Sly
• Organization: Clinvest Research

jsly@clinvest.com

4178413673

Study Officials

• Timothy R Smith, MD

  Clinvest Research, LLC

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: At randomization (Visit 2), neither the subject nor the investigator will be aware to which treatment group the subject has been assigned. Investigational product will be drawn and administered in a double blind format. If needed, for safety and proper treatment of the subject, the investigator can unblind the subject's treatment assignment to determine which treatment has been assigned and institute appropriate follow-up care. When possible, Sponsor and Clinvest should be notified prior to unblinding study drug.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDIV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: March 27, 2017
• First Posted: April 11, 2017
• Study Start: August 1, 2017
• Primary Completion: April 26, 2019
• Study Completion: April 26, 2019
• Last Updated: February 12, 2020
• Results First Posted: February 5, 2020

Record last verified: 2020-02

Locations

US (2)