Trial Outcomes & Findings for Erenumab for Idiosyncratic Facial Pain (NCT NCT04249427)

NCT ID: NCT04249427

Last Updated: 2024-09-24

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 29 participants
• Primary outcome timeframe: Baseline, 1,3, and 6 months
• Results posted on: 2024-09-24

Participant Flow

Four participants withdrew prior to administration of injection.

Participant milestones

Measure	Erenumab 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Overall Study STARTED	13	12
Overall Study COMPLETED	9	8
Overall Study NOT COMPLETED	4	4

Reasons for withdrawal

Measure	Erenumab 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Overall Study Adverse Event	3	1
Overall Study Lack of Efficacy	0	1
Overall Study Withdrawal by Subject	1	2

Baseline Characteristics

Erenumab for Idiosyncratic Facial Pain

Baseline characteristics by cohort

Measure	Erenumab n=13 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=12 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection	Total n=25 Participants Total of all reporting groups
Age, Continuous	51.4 years STANDARD_DEVIATION 11.5 • n=5 Participants	43.2 years STANDARD_DEVIATION 12.5 • n=7 Participants	47.4 years STANDARD_DEVIATION 12.4 • n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	10 Participants n=7 Participants	21 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	12 Participants n=5 Participants	12 Participants n=7 Participants	24 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants	9 Participants n=7 Participants	19 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Region of Enrollment United States	13 Participants n=5 Participants	12 Participants n=7 Participants	25 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 1,3, and 6 months

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.

Outcome measures

Measure	Erenumab n=13 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=12 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Mean Number of Days Per Month With Significant Mid-facial Pain or Pressure	-6.0 days per month Standard Deviation 6.9	-5.8 days per month Standard Deviation 8.0

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Population: Participants with data collected at both timepoints.

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.

Outcome measures

Measure	Erenumab n=9 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=9 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in SNOT-22 (Sino-nasal Outcome Test)	-10.1 score on a scale Standard Deviation 13.8	-23.3 score on a scale Standard Deviation 25.2

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Population: Participants with data collected at both timepoints.

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.

Outcome measures

Measure	Erenumab n=9 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=9 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Physical Function as Measured by Migraine Functional Impact Questionnaire (MFIQ)	-3.3 score on a scale Standard Deviation 4.5	-5.6 score on a scale Standard Deviation 6.5

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Population: Participants with data collected at both timepoints.

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.

Outcome measures

Measure	Erenumab n=9 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=9 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Usual Activities as Measured by Migraine Functional Impact Questionnaire (MFIQ)	-2.1 score on a scale Standard Deviation 4.4	-10.9 score on a scale Standard Deviation 9.6

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Population: Participants with data collected at both timepoints.

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.

Outcome measures

Measure	Erenumab n=9 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=9 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Social Function as Measured by Migraine Function Impact Questionnaire (MFIQ)	-1.0 score on a scale Standard Deviation 3.6	5.7 score on a scale Standard Deviation 13.0

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Population: Participants with data collected at both timepoints.

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.

Outcome measures

Measure	Erenumab n=9 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=9 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Emotional Function as Measured by Migraine Function Impact Questionnaire (MFIQ)	0.4 score on a scale Standard Deviation 5.9	-6.6 score on a scale Standard Deviation 5.7

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Population: Participants with data collected at both timepoints.

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.

Outcome measures

Measure	Erenumab n=9 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=9 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Overall Impact (Global) as Measured by Migraine Function Impact Questionnaire (MFIQ)	0.11 score on a scale Standard Deviation 0.78	-1.33 score on a scale Standard Deviation 1.00

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Measured by daily diary completion via mobile app. Reported as the change from baseline to six months.

Outcome measures

Measure	Erenumab n=13 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=12 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Mean Number of Days Per Month With Significant Nasal Congestion	-3.2 days per month Standard Deviation 6.0	-2.8 days per month Standard Deviation 4.7

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Measured by daily diary completion via mobile app. Reported as the change from baseline to six months.

Outcome measures

Measure	Erenumab n=13 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=12 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Mean Number of Days Per Month With Significant Rhinorrhea	-1.4 days per month Standard Deviation 5.7	-1.8 days per month Standard Deviation 4.8

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

Measured by daily diary completion via mobile app. Reported as the change from baseline to six months.

Outcome measures

Measure	Erenumab n=13 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=12 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change in Mean Number of Days of Rescue Medication Used Per Month	-5.2 days per month Standard Deviation 8.4	-2.1 days per month Standard Deviation 3.8

SECONDARY outcome

Timeframe: Baseline, 1, 3 and 6 months

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.

Outcome measures

Measure	Erenumab n=13 Participants 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=12 Participants Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Change From Baseline in Mean Daily Pain Score	-0.9 score on a scale Standard Deviation 1.1	-1.2 score on a scale Standard Deviation 1.6

Adverse Events

Erenumab

Serious events: 2 serious events

Other events: 7 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Measure	Erenumab n=13 participants at risk 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=12 participants at risk Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Vascular disorders Hypertension	15.4% 2/13 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.	0.00% 0/12 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.

Other adverse events

Measure	Erenumab n=13 participants at risk 140mg Erenumab administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Erenumab Prefilled Syringe: 140mg Erenumab, pre-filled syringe given by subcutaneous injection	Placebo n=12 participants at risk Placebo administered by subcutaneous injection (in the abdomen, thigh, or upper arm), once monthly for six months. Placebo: Placebo, pre-filled syringe given by subcutaneous injection
Musculoskeletal and connective tissue disorders Arm soreness/pain	7.7% 1/13 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.	0.00% 0/12 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.
Vascular disorders Hypertension	46.2% 6/13 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.	33.3% 4/12 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.
Renal and urinary disorders Urinary tract infection	7.7% 1/13 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.	0.00% 0/12 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.
Gastrointestinal disorders Constipation	15.4% 2/13 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.	8.3% 1/12 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.
General disorders Septoplasty	0.00% 0/13 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.	8.3% 1/12 • 6 months AEs and SAEs were systematically investigated at each study visit and self-reported and recorded using REDCap. Study team was notified via REDCap if subjects self-reported hypertension.

Additional Information

David Jang, MD

Duke University

Phone: 919-613-6407

Email: david.jang@duke.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place