Erenumab for Idiosyncratic Facial Pain

A Randomized, Double-Blinded, Single Site Study to Evaluate the Efficacy of Erenumab for Treatment of Idiosyncratic Facial Pain Mimicking Rhinosinusitis

This is a phase IV randomized, double-blinded, placebo-controlled study to evaluate the efficacy of Erenumab in subjects with midfacial pain or pressure, without clinical or radiographic evidence of sinusitis.

Study Overview

• Status: Completed
• Conditions: Facial Pain; Rhinosinusitis
• Intervention / Treatment: Drug: Erenumab Prefilled Syringe; Other: Placebo

Detailed Description

Eligible participants are randomized to receive either Erenumab or placebo by subcutaneous injection once monthly for 6 months. Study duration is eight months which includes a 30 day screening/lead-in period and 6 monthly treatment visits followed by a follow-up visit one month following the last dose of study drug administration. Participants will be expected to score on a scale of 1 to 10 their symptoms of facial pain/pressure, nasal congestion, and rhinorrhea as well well as any rescue medicines taken for pain each day via a mobile app.

• Study Type: Interventional
• Enrollment (Actual): 29
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center and affiliated practices

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Adults 18 years of age and older presenting to Duke Head & Neck Surgery and Communications Sciences clinic for evaluation of rhinosinusitis and/or facial pain or pressure.
2. Symptoms are present at least 10 days a month for the last 3 months as reported by subject.
3. Symptoms must include midfacial pain or uncomfortable pressure (may be unilateral or bilateral), which is defined as pain in the regions overlying the maxillary, ethmoid, frontal sinuses either together or individually.
4. Nasal endoscopy in the last three months shows no signs of inflammation (i.e. thick drainage, polyps, watery edema in the middle meatus or spheno-ethmoid recess (mild edema permitted).
5. Sinus CT scan or MRI within 12 months of enrollment during a symptomatic period shows no more than scattered minimal mucosal edema or mucous retention cyst with patent infundibula bilaterally. For subjects with a CT scan more than 12 months old or just an MRI, a CT will be repeated for study purposes. For patients with CT scans less than 12 months old, a CT will be repeated for study purposes if the subject has changes in symptoms suggestive of sinusitis.
6. Ability to read/write English.
7. Has a smart phone, ipod or iPad touch for completion of the EMA on a daily basis.

Exclusion Criteria:

1. Hypersensitivity to erenumab or to any of the drug components (acetate, polysorbate, and sucrose).
2. Previous exposure to erenumab or any other CGRP inhibitor in the six months prior to treatment.
3. Allergy to latex.
4. Inability to differentiate facial pain from other headaches.
5. Non-English speaking or unable to provide written informed consent.

6. On a preventative migraine medication (see below) during the 30 day lead-in period:

   • Category 1: Divalproex sodium, sodium valproate
   • Category 2: Topiramate
   • Category 3: Beta blockers (for example: atenolol, bisoprolol, metoprolol, nadolol, ebivolol, pindolol, propranolol, timolol)
   • Category 4: Tricyclic antidepressants (for example: amitriptyline, nortriptyline, protriptyline)
   • Category 5: Serotonin-norepinephrine reuptake inhibitors (for example: venlafaxine, desvenlafaxine, duloxetine, milnacipran)
   • Category 6: Flunarizine, verapamil
   • Category 7: Lisinopril, candesartan
7. Received botulinum toxin (Botox) to the head and neck for migraines in the last four months.
8. More than one major open surgery of the nose or sinuses for sinonasal cancer.
9. History of uncontrolled or unstable blood pressure.
10. History of liver failure.
11. History of metastatic malignancy in the last five years or actively undergoing treatment for cancer.
12. Active seizure disorder or other significant neurological conditions other than migraine.
13. Myocardial infarction (MI), stroke, transient ischemic attack (TIA), unstable angina, or coronary artery bypass surgery or other revascularization procedure within 12 months prior to screening.
14. History or evidence of any other unstable or clinically significant medical condition that in the opinion of the sponsor-investigator/ Principal Investigator, would pose a risk to subject safety or interfere with the study evaluation, procedures or completion.
15. Evidence of drug or alcohol abuse or dependence within 12 months prior to screening, based on medical records or patient self-report.
16. Pregnant or breastfeeding, or expecting to conceive during the study, including through 16 weeks after the last dose of investigational product or placebo

17. Female subject of childbearing potential who is unwilling to use an acceptable method of effective contraception during treatment with investigational product or placebo through 16 weeks after the last dose of investigational product. Female subjects not of childbearing potential are defined as any female who is post-menopausal by history, defined as:

    • Age ≥ 55 years with cessation of menses for 12 or more months, OR
    • Age < 55 years but no spontaneous menses for at least 2 years, OR
    • Underwent bilateral oophorectomy, bilateral salpingectomy, or hysterectomy
18. Unlikely to be able to complete all protocol required study visits or procedures.
19. Currently receiving treatment in another investigational device or drug study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Erenumab; 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months.	Drug: Erenumab Prefilled Syringe; 140mg Erenumab, pre-filled syringe given by subcutaneous injection
Placebo Comparator: Placebo; Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months.	Other: Placebo; Placebo, pre-filled syringe given by subcutaneous injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Mean Number of Days Per Month With Significant Mid-facial Pain or Pressure	Significant mid-facial pain or pressure defined as greater than 4/10 on scale of 1 to 10, measured by daily diary completion. Reported as the change from baseline to six months.	Baseline, 1,3, and 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in SNOT-22 (Sino-nasal Outcome Test)	The SNOT-22 consists of 22 items, each rated from 0 (no problem at all) to 5 (worst possible symptom). SNOT-22 total scores range from 0 to 110, with higher SNOT-22 total scores indicating worse symptoms. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change in Physical Function as Measured by Migraine Functional Impact Questionnaire (MFIQ)	The MFIQ is a 26-item self-report questionnaire, developed to measure the subjective impact of migraine on physical functioning (everyday activities and physical impairment), as well as social and emotional functioning in the past seven days. The MFIQ Physical Function subscale is scored on a scale of 0-100, with higher scores indicating a greater impact. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change in Usual Activities as Measured by Migraine Functional Impact Questionnaire (MFIQ)	The MFIQ is a 26-item self-report questionnaire, developed to measure the subjective impact of migraine on physical functioning (everyday activities and physical impairment), as well as social and emotional functioning in the past seven days. The MFIQ usual activities subscale is scored on a scale of 0-100, with higher scores indicating a greater impact. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change in Social Function as Measured by Migraine Function Impact Questionnaire (MFIQ)	The MFIQ is a 26-item self-report questionnaire, developed to measure the subjective impact of migraine on physical functioning (everyday activities and physical impairment), as well as social and emotional functioning in the past seven days. The MFIQ Social Function subscale is scored on a scale of 0-100, with higher scores indicating a greater impact. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change in Emotional Function as Measured by Migraine Function Impact Questionnaire (MFIQ)	The MFIQ is a 26-item self-report questionnaire, developed to measure the subjective impact of migraine on physical functioning (everyday activities and physical impairment), as well as social and emotional functioning in the past seven days. The MFIQ Emotional Function subscale is scored on a scale of 0-100, with higher scores indicating a greater impact. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change in Overall Impact (Global) as Measured by Migraine Function Impact Questionnaire (MFIQ)	The MFIQ is a 26-item self-report questionnaire, developed to measure the subjective impact of migraine on physical functioning (everyday activities and physical impairment), as well as social and emotional functioning in the past seven days. The MFIQ Overall Impact is scored on a scale of 0-100, with higher scores indicating a greater impact. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change in Mean Number of Days Per Month With Significant Nasal Congestion	Measured by daily diary completion via mobile app. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change in Mean Number of Days Per Month With Significant Rhinorrhea	Measured by daily diary completion via mobile app. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change in Mean Number of Days of Rescue Medication Used Per Month	Measured by daily diary completion via mobile app. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months
Change From Baseline in Mean Daily Pain Score	Measured by daily diary completion via mobile app on a scale of 1-10, where 1=no pain and 10=intense pain. Reported as the change from baseline to six months.	Baseline, 1, 3 and 6 months

Collaborators and Investigators

• Sponsor: David Jang, M.D.
• Collaborators: Amgen
• Investigators: Principal Investigator: David Jang, MD, Duke Health

Study record dates

Study Major Dates

• Study Start (Actual): February 10, 2021
• Primary Completion (Actual): July 11, 2023
• Study Completion (Actual): July 11, 2023

Study Registration Dates

• First Submitted: January 29, 2020
• First Submitted That Met QC Criteria: January 29, 2020
• First Posted (Actual): January 31, 2020

Study Record Updates

• Last Update Posted (Actual): September 24, 2024
• Last Update Submitted That Met QC Criteria: August 30, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Infections; Respiratory Tract Infections; Respiratory Tract Diseases; Pain; Neurologic Manifestations; Disease Attributes; Otorhinolaryngologic Diseases; Paranasal Sinus Diseases; Nose Diseases; Rhinitis; Sinusitis; Facial Pain; Facies; Rhinosinusitis; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; Calcitonin Gene-Related Peptide Receptor Antagonists; Erenumab
• Other Study ID Numbers: Pro00104284

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No