Trial Outcomes & Findings for Study of Efficacy and Safety of Erenumab in Adult Chronic Migraine Patients (NCT NCT03867201)

Last Updated: 2025-10-16

Results Overview

A migraine day was defined as any calendar day in which the subject experienced a qualified migraine headache (onset, continuation, or recurrence of the migraine headache). A qualified migraine headache was defined as a migraine with or without aura, lasting for ≥4 continuous hours, and meeting at least one of the following criteria: 1. ≥2 of the following pain features: * Unilateral * Throbbing * Moderate to severe * Exacerbated with exercise/physical activity 2. ≥1 of the following associated symptoms: * Nausea and/or vomiting * Photophobia and phonophobia

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

557 participants

Primary outcome timeframe

baseline (4 weeks period prior to start of study drug), week 9 to 12

Results posted on

2025-10-16

Participant Flow

A screening period of 2 weeks was used to assess initial eligibility, followed by a 4-week baseline period to assess diary compliance and headache frequency.

Participant milestones

Participant milestones
Measure	Erenumab 70 mg Administered by pre-filled syringe every 4 weeks starting at Day 1	Placebo Placebo administered by pre-filled syringe at Day 1 and Weeks 4 and 8. Erenumab 70 mg administered by pre-filled syringe every 4 weeks starting at Week 12
Double-Blind Period Until Week 12 STARTED	279	278
Double-Blind Period Until Week 12 COMPLETED	273	272
Double-Blind Period Until Week 12 NOT COMPLETED	6	6
Open-Label Period From Week 12 STARTED	224	232
Open-Label Period From Week 12 COMPLETED	105	121
Open-Label Period From Week 12 NOT COMPLETED	119	111

Reasons for withdrawal

Reasons for withdrawal
Measure	Erenumab 70 mg Administered by pre-filled syringe every 4 weeks starting at Day 1	Placebo Placebo administered by pre-filled syringe at Day 1 and Weeks 4 and 8. Erenumab 70 mg administered by pre-filled syringe every 4 weeks starting at Week 12
Double-Blind Period Until Week 12 Lack of Efficacy	1	0
Double-Blind Period Until Week 12 Withdrawal by Subject	1	2
Double-Blind Period Until Week 12 Protocol Violation	1	2
Double-Blind Period Until Week 12 Pregnancy	1	1
Double-Blind Period Until Week 12 Adverse Event	2	1
Open-Label Period From Week 12 Adverse Event	5	11
Open-Label Period From Week 12 Lost to Follow-up	1	0
Open-Label Period From Week 12 Physician Decision	6	3
Open-Label Period From Week 12 Pregnancy	2	2
Open-Label Period From Week 12 Protocol Violation	2	0
Open-Label Period From Week 12 Withdrawal by Subject	90	85
Open-Label Period From Week 12 No longer clinically benefiting	12	10
Open-Label Period From Week 12 New Therapy for Study Indication	1	0

Baseline Characteristics

Study of Efficacy and Safety of Erenumab in Adult Chronic Migraine Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Erenumab 70 mg n=279 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8	Placebo n=278 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8	Total n=557 Participants Total of all reporting groups
Age, Continuous	41.4 years STANDARD_DEVIATION 10.9 • n=5 Participants	41.9 years STANDARD_DEVIATION 10.9 • n=7 Participants	41.7 years STANDARD_DEVIATION 10.9 • n=5 Participants
Sex: Female, Male Female	217 Participants n=5 Participants	237 Participants n=7 Participants	454 Participants n=5 Participants
Sex: Female, Male Male	62 Participants n=5 Participants	41 Participants n=7 Participants	103 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	277 Participants n=5 Participants	275 Participants n=7 Participants	552 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	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: baseline (4 weeks period prior to start of study drug), week 9 to 12

Population: Full Analysis Set. All randomized participants with evaluable measurements at the respective timepoint.

Outcome measures

Outcome measures
Measure	Erenumab 70 mg n=270 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8	Placebo n=274 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8
Change From Baseline in Monthly Migraine Days During the Last 4 Weeks of the 12-week Treatment Period	-8.19 days Standard Error 0.46	-6.62 days Standard Error 0.45

SECONDARY outcome

Timeframe: baseline (4 weeks period prior to start of study drug), week 9 to 12

Population: Full Analysis Set. All randomized participants with evaluable measurements at the respective timepoint.

The modified MIDAS is a 5-item self-administered questionnaire that sums the number of productive days lost over the past month in two settings: the workplace and the home. The MIDAS also assesses disability in family, social, and leisure activities. The MIDAS score is the sum of missed days due to a headache from paid work, housework, and non-work (family, social, leisure) activities; and days at paid work or housework where productivity was reduced by at least half.

Outcome measures

Outcome measures
Measure	Erenumab 70 mg n=263 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8	Placebo n=268 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8
Change From Baseline in Migraine-related Disability and Productivity as Measured by the mMIDAS During the Last 4 Weeks of the 12-week Treatment Period	-14.67 days Standard Error 1.20	-12.93 days Standard Error 1.19

SECONDARY outcome

Timeframe: baseline (4 weeks period prior to start of study drug), week 9 to 12

Population: Full Analysis Set. All randomized participants.

Outcome measures

Outcome measures
Measure	Erenumab 70 mg n=279 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8	Placebo n=278 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8
Number of Participants With at Least 50% Reduction From Baseline in Monthly Migraine Days During the Last 4 Weeks of the 12-week Treatment Period	131 Participants	102 Participants

SECONDARY outcome

Timeframe: baseline (4 weeks period prior to start of study drug), week 9 to 12

Population: Full Analysis Set. All randomized participants with evaluable measurements at the respective timepoint.

An acute headache medication day was defined as a day when medication was taken to treat acute headache.

Outcome measures

Outcome measures
Measure	Erenumab 70 mg n=270 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8	Placebo n=274 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8
Change From Baseline in Monthly Acute Headache Medication Days During the Last 4 Weeks of the 12-week Treatment Period	-5.34 days Standard Error 0.39	-4.66 days Standard Error 0.39

SECONDARY outcome

Timeframe: DBTP: 12 weeks for participants entering the OLTP. 20 weeks for participants NOT entering the OLTP. OLTP: From week 12 until up to approximately 4 years.

Population: Safety Set (for DBTP). Number of participants entering the OLTP (for OLTP).

Number of subjects with adverse events was assessed separately in the double-blind treatment period (DBTP) and the open-label treatment period (OLTP).

Outcome measures

Outcome measures
Measure	Erenumab 70 mg n=279 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8	Placebo n=278 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8
Number of Subjects With Adverse Events as a Measure of Safety Open-Label Treatment Period: Number of subjects with at least one AE	152 Participants	176 Participants
Number of Subjects With Adverse Events as a Measure of Safety Double-Blind Treatment Period: Number of subjects with at least one AE	127 Participants	132 Participants

SECONDARY outcome

Timeframe: baseline, 20 weeks

Population: FAS including participants with a valid assessment

anti-AMG 334 antibodies assessed for binding and neutralizing)

Outcome measures

Outcome measures
Measure	Erenumab 70 mg n=266 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8	Placebo n=264 Participants Administered by pre-filled syringe at Day 1 and Weeks 4 and 8
Number of Subjects With Anti-AMG 334 Antibodies Binding antibody positive at baseline	0 Participants	0 Participants
Number of Subjects With Anti-AMG 334 Antibodies Neutralizing antibody positive at baseline	0 Participants	0 Participants
Number of Subjects With Anti-AMG 334 Antibodies Binding antibody positive at any time from baseline to week 20	9 Participants	0 Participants
Number of Subjects With Anti-AMG 334 Antibodies Neutralizing antibody positive at any time from baseline to week 20	1 Participants	0 Participants

Adverse Events

Erenumab 70mg DBTP

Serious events: 7 serious events

Other events: 58 other events

Deaths: 0 deaths

Placebo DBTP

Serious events: 7 serious events

Other events: 59 other events

Deaths: 0 deaths

Erenumab 70mg OLTP (Erenumab 70mg in DBTP)

Serious events: 21 serious events

Other events: 92 other events

Deaths: 0 deaths

Erenumab 70mg OLTP (Placebo in DBTP)

Serious events: 24 serious events

Other events: 120 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	Erenumab 70mg DBTP n=279 participants at risk double-blind treatment period (DBTP). Administered by pre-filled syringe at Day 1 and Weeks 4 and 8.	Placebo DBTP n=278 participants at risk double-blind treatment period (DBTP). Administered by pre-filled syringe at Day 1 and Weeks 4 and 8.	Erenumab 70mg OLTP (Erenumab 70mg in DBTP) n=224 participants at risk open-label treatment period (OLTP). Administered by pre-filled syringe every 4 weeks starting at week 12.	Erenumab 70mg OLTP (Placebo in DBTP) n=232 participants at risk open-label treatment period (OLTP). Administered by pre-filled syringe every 4 weeks starting at week 12.
Gastrointestinal disorders Constipation	8.6% 24/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	4.0% 11/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	6.2% 14/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	12.5% 29/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.
Infections and infestations COVID-19	0.00% 0/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	0.36% 1/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	17.4% 39/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	20.3% 47/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.
Infections and infestations Nasopharyngitis	3.6% 10/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	1.8% 5/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	2.7% 6/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	5.6% 13/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.
Infections and infestations Upper respiratory tract infection	5.7% 16/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	6.8% 19/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	13.8% 31/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	13.4% 31/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.
Musculoskeletal and connective tissue disorders Back pain	1.4% 4/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	0.72% 2/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	2.7% 6/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	5.2% 12/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.
Nervous system disorders Dizziness	1.8% 5/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	4.3% 12/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	4.5% 10/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	7.3% 17/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.
Nervous system disorders Headache	0.36% 1/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	2.2% 6/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	5.8% 13/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	5.2% 12/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.
Nervous system disorders Migraine	1.4% 4/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	2.2% 6/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	1.3% 3/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	6.5% 15/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.
Psychiatric disorders Insomnia	1.4% 4/279 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	1.8% 5/278 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	4.0% 9/224 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.	5.6% 13/232 • Double-Blind Treatment Period: 12 weeks for participants entering the open-label treatment period. 20 weeks for participants NOT entering the open-label treatment period. Open-Label Treatment Period: From week 12 until up to approximately 4 years.

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: + 1 862 778 8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER