Trial Outcomes & Findings for Safety and Efficacy in Adult Subjects With Acute Migraines (NCT NCT03237845)

NCT ID: NCT03237845

Last Updated: 2023-02-16

Results Overview

Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the electronic diary (eDiary). Pain freedom was defined as pain level of none.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

1499 participants

Primary outcome timeframe

2 Hours post-dose

Results posted on

2023-02-16

Participant Flow

This study was conducted at 50 sites in United States of which 49 sites enrolled at least 1 participant.

A total of 1499 participants were enrolled, of which 1186 participants were randomized to rimegepant 75 milligram (mg) or placebo. A total of 313 participants failed screening mainly due to failure to meet eligibility criteria. The randomization was stratified in a 1:1 ratio based on use of prophylactic migraine medications (yes or no).

Participant milestones

Participant milestones
Measure
Rimegepant 75 mg
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Overall Study
STARTED
594
592
Overall Study
Treated
543
543
Overall Study
COMPLETED
538
542
Overall Study
NOT COMPLETED
56
50

Reasons for withdrawal

Reasons for withdrawal
Measure
Rimegepant 75 mg
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Overall Study
Adverse Event
0
1
Overall Study
Lost to Follow-up
13
10
Overall Study
Not Experienced Moderate/Severe Migraine
26
31
Overall Study
Non-Compliance With Study Treatment
0
1
Overall Study
Physician Decision
1
2
Overall Study
Pregnancy
1
0
Overall Study
Protocol Deviation
0
1
Overall Study
Technical Problems
8
1
Overall Study
Withdrawal by Subject
3
3
Overall Study
Other Reasons
4
0

Baseline Characteristics

Safety and Efficacy in Adult Subjects With Acute Migraines

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Total
n=1072 Participants
Total of all reporting groups
Age, Continuous
40.195 Years
STANDARD_DEVIATION 11.8693 • n=99 Participants
40.910 Years
STANDARD_DEVIATION 12.1168 • n=107 Participants
40.552 Years
STANDARD_DEVIATION 11.9932 • n=206 Participants
Sex: Female, Male
Female
479 Participants
n=99 Participants
472 Participants
n=107 Participants
951 Participants
n=206 Participants
Sex: Female, Male
Male
58 Participants
n=99 Participants
63 Participants
n=107 Participants
121 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
77 Participants
n=99 Participants
83 Participants
n=107 Participants
160 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
460 Participants
n=99 Participants
452 Participants
n=107 Participants
912 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
American Indian or Alaska Native
4 Participants
n=99 Participants
5 Participants
n=107 Participants
9 Participants
n=206 Participants
Race (NIH/OMB)
Asian
8 Participants
n=99 Participants
8 Participants
n=107 Participants
16 Participants
n=206 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
6 Participants
n=99 Participants
0 Participants
n=107 Participants
6 Participants
n=206 Participants
Race (NIH/OMB)
Black or African American
111 Participants
n=99 Participants
118 Participants
n=107 Participants
229 Participants
n=206 Participants
Race (NIH/OMB)
White
394 Participants
n=99 Participants
399 Participants
n=107 Participants
793 Participants
n=206 Participants
Race (NIH/OMB)
More than one race
14 Participants
n=99 Participants
5 Participants
n=107 Participants
19 Participants
n=206 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Primary Migraine Type
Migraine without Aura
355 Participants
n=99 Participants
366 Participants
n=107 Participants
721 Participants
n=206 Participants
Primary Migraine Type
Migraine with Aura
182 Participants
n=99 Participants
169 Participants
n=107 Participants
351 Participants
n=206 Participants
Randomization Strata, Prophylactic Migraine Medication Use
Yes
89 Participants
n=99 Participants
89 Participants
n=107 Participants
178 Participants
n=206 Participants
Randomization Strata, Prophylactic Migraine Medication Use
No
448 Participants
n=99 Participants
446 Participants
n=107 Participants
894 Participants
n=206 Participants

PRIMARY outcome

Timeframe: 2 Hours post-dose

Population: The analysis was performed on modified intent to treat (mITT) participants.

Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the electronic diary (eDiary). Pain freedom was defined as pain level of none.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Freedom From Pain at 2 Hours Post-dose
19.6 Percentage of participants
Interval 16.2 to 22.9
12.0 Percentage of participants
Interval 9.2 to 14.7

PRIMARY outcome

Timeframe: 2 Hours

Population: The analysis was performed on mITT participants.

MBS was reported as nausea, photophobia, or phonophobia at migraine onset using the eDiary. Symptom status (absent, present) was assessed post-dose using the eDiary separately for nausea, photophobia, and phonophobia. Freedom from MBS was defined as MBS reported at onset that was absent post-dose.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Freedom From Most Bothersome Symptom (MBS) at 2 Hours Post-dose
37.6 Percentage of Participants
Interval 33.5 to 41.7
25.2 Percentage of Participants
Interval 21.6 to 28.9

SECONDARY outcome

Timeframe: 2 hours post-dose

Population: The analysis was performed on mITT participants with photophobia present at migraine onset.

Photophobia (sensitivity to light) status was measured as absent or present in the eDiary. Freedom from photophobia was defined as photophobia absent.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=489 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=477 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Freedom From Photophobia at 2 Hours Post-dose
37.4 Percentage of Participants
Interval 33.1 to 41.7
22.3 Percentage of Participants
Interval 18.6 to 26.0

SECONDARY outcome

Timeframe: 2 hours post-dose

Population: The analysis was performed on mITT participants with phonophobia present at migraine onset.

Phonophobia (sensitivity to sound) status was measured as absent or present in the eDiary. Freedom from phonophobia was defined as phonophobia absent.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=362 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=374 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Freedom From Phonophobia at 2 Hours Post-dose
36.7 Percentage of Participants
Interval 31.7 to 41.7
26.8 Percentage of Participants
Interval 22.3 to 31.3

SECONDARY outcome

Timeframe: 2 hours post-dose

Population: The analysis was performed on mITT participants..

Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Pain relief was defined as pain level of none or mild.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Pain Relief at 2 Hours Post-dose
58.1 Percentage of Participants
Interval 53.9 to 62.3
42.8 Percentage of Participants
Interval 38.6 to 47.0

SECONDARY outcome

Timeframe: 2 hours post-dose

Population: The analysis was performed on mITT participants with nausea present at migraine onset.

Nausea status was measured as absent or present in the eDiary. Freedom from nausea was defined as nausea absent.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=355 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=336 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Freedom From Nausea at 2 Hours Post-dose
48.1 Percentage of Participants
Interval 42.9 to 53.3
43.3 Percentage of Participants
Interval 38.0 to 48.6

SECONDARY outcome

Timeframe: 24 hours post-dose

Population: The analysis was performed on mITT participants.

Participants who did not experience relief of their migraine headache at the end of 2 hours after dosing with study medication (and after the 2-hour assessments had been completed on the eDiary) were permitted to use the following rescue medications: aspirin, ibuprofen, acetaminophen up to 1000 mg/day (this includes Excedrin Migraine), naproxen (or any other type of nonsteroidal anti-inflammatory drug), antiemetics (e.g., metoclopramide or promethazine), or baclofen. The participant's use of rescue medication was recorded by the participant in a paper diary.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Rescue Medication Use Within 24 Hours Post-dose
21.0 Percentage of Participants
Interval 17.6 to 24.5
37.0 Percentage of Participants
Interval 32.9 to 41.1

SECONDARY outcome

Timeframe: From 2 hours up to 24 hours post-dose

Population: The analysis was performed on mITT participants

Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain freedom was defined as pain level of none at 2 hours up to 24 hours post-dose with no rescue medication use through 24 hours post-dose.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Sustained Pain Freedom From 2 to 24 Hours Post-dose
12.3 Percentage of Participants
Interval 9.5 to 15.1
7.1 Percentage of Participants
Interval 4.9 to 9.3

SECONDARY outcome

Timeframe: From 2 hours up to 24 hours post-dose

Population: The analysis was performed on mITT participants.

Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain relief was defined as pain level of none or mild at 2 hours up to 24 hours post-dose with no rescue medication use through 24 hours post-dose.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Sustained Pain Relief From 2 to 24 Hours Post-dose
42.6 Percentage of Participants
Interval 38.5 to 46.8
26.5 Percentage of Participants
Interval 22.8 to 30.3

SECONDARY outcome

Timeframe: From 2 hours up to 48 hours post-dose

Population: The analysis was performed on mITT participants.

Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain freedom was defined as pain level of none at 2 hours up to 48 hours post-dose with no rescue medication use through 48 hours post-dose.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Sustained Pain Freedom From 2 to 48 Hours Post-dose
9.9 Percentage of Participants
Interval 7.3 to 12.4
6.0 Percentage of Participants
Interval 4.0 to 8.0

SECONDARY outcome

Timeframe: From 2 hours up to 48 hours post-dose

Population: The analysis was performed on mITT participants.

Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Sustained pain relief was defined as pain level of none or mild at 2 hours up to 48 hours post-dose with no rescue medication use through 48 hours post-dose.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Sustained Pain Relief From 2 to 48 Hours Post-dose
36.3 Percentage of Participants
Interval 32.2 to 40.4
22.6 Percentage of Participants
Interval 19.1 to 26.2

SECONDARY outcome

Timeframe: From 2 hours up to 48 hours post-dose

Population: The analysis population was performed on mITT participants with pain freedom at 2 hours post-dose.

Pain levels were assessed on a 4-point scale (none, mild, moderate, severe) using the eDiary. Pain relapse was defined as pain level of mild, moderate, or severe after 2 hours up to 48 hours for the participants who were pain-free at 2 hours post-dose.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=105 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=64 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Pain Relapse From 2 to 48 Hours Post-dose
49.6 Percentage of Participants
Interval 40.1 to 59.1
50.0 Percentage of Participants
Interval 37.8 to 62.1

SECONDARY outcome

Timeframe: 2 hours post-dose

Population: The analysis was performed on mITT participants.

Functional disability level was assessed in the eDiary on a 4-point scale: normal function, mild impairment, severe impairment, required bed rest. Freedom from functional disability was defined as normal function.

Outcome measures

Outcome measures
Measure
Rimegepant 75 mg
n=537 Participants
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=535 Participants
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Percentage of Participants With Freedom From Functional Disability at 2 Hours Post-dose
32.6 Percentage of Participants
Interval 28.6 to 36.5
23.4 Percentage of Participants
Interval 19.8 to 26.9

Adverse Events

Rimegepant 75 mg

Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths

Placebo

Serious events: 2 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Rimegepant 75 mg
n=543 participants at risk
Participants were administered a single oral dose of 75 mg of rimegepant tablet on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
Placebo
n=543 participants at risk
Participants were administered a single oral dose of matching placebo tablet for rimegepant (75 mg) on occurrence of migraine that reached moderate or severe intensity up to 45 days after randomization.
General disorders
Chest Pain
0.00%
0/543 • Serious adverse events (SAEs) were collected from informed consent up to the end of the study, and adverse events (AEs) were collected from randomization up to end of the study (up to 52 days).
The safety population, all enrolled participants who received at least 1 dose of rimegepant or placebo, was used to determine the number of participants at risk for SAEs and other AEs.
0.18%
1/543 • Number of events 1 • Serious adverse events (SAEs) were collected from informed consent up to the end of the study, and adverse events (AEs) were collected from randomization up to end of the study (up to 52 days).
The safety population, all enrolled participants who received at least 1 dose of rimegepant or placebo, was used to determine the number of participants at risk for SAEs and other AEs.
Infections and infestations
Urinary Tract Infection
0.00%
0/543 • Serious adverse events (SAEs) were collected from informed consent up to the end of the study, and adverse events (AEs) were collected from randomization up to end of the study (up to 52 days).
The safety population, all enrolled participants who received at least 1 dose of rimegepant or placebo, was used to determine the number of participants at risk for SAEs and other AEs.
0.18%
1/543 • Number of events 1 • Serious adverse events (SAEs) were collected from informed consent up to the end of the study, and adverse events (AEs) were collected from randomization up to end of the study (up to 52 days).
The safety population, all enrolled participants who received at least 1 dose of rimegepant or placebo, was used to determine the number of participants at risk for SAEs and other AEs.
Musculoskeletal and connective tissue disorders
Back Pain
0.18%
1/543 • Number of events 1 • Serious adverse events (SAEs) were collected from informed consent up to the end of the study, and adverse events (AEs) were collected from randomization up to end of the study (up to 52 days).
The safety population, all enrolled participants who received at least 1 dose of rimegepant or placebo, was used to determine the number of participants at risk for SAEs and other AEs.
0.00%
0/543 • Serious adverse events (SAEs) were collected from informed consent up to the end of the study, and adverse events (AEs) were collected from randomization up to end of the study (up to 52 days).
The safety population, all enrolled participants who received at least 1 dose of rimegepant or placebo, was used to determine the number of participants at risk for SAEs and other AEs.

Other adverse events

Adverse event data not reported

Additional Information

Chief Medical Officer

Biohaven Pharmaceuticals, Inc.

Phone: 203-404-0410

Results disclosure agreements

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

Restriction type: LTE60