Trial Outcomes & Findings for Safety and Efficacy Trial of BHV3000 (Rimegepant) 75 mg for the Acute Treatment of Migraine (NCT NCT04574362)

Last Updated: 2023-12-14

Results Overview

Pain was measured on a 4-point Likert scale, with following scores: 0= none, 1= mild, 2= moderate, 3= severe. Participants with score of 0 (no pain) were considered to have freedom from pain. Exact 95 percent (%) confidence interval (CI) was based on Clopper-Pearson method.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

1648 participants

Primary outcome timeframe

2 hours post-dose

Results posted on

2023-12-14

Participant Flow

Overall, 1648 participants were enrolled and screened. A total of 217 participants were screen failures. Only 1431 participants were randomized. Out of 1431 participants, only 1342 participants were treated.

Participants were dispensed with 1 dose of study drug at randomization (baseline). Participants were required to administer pre-dispensed study drug only when the participants developed a migraine headache of moderate or severe intensity and completed all required migraine assessment questions in the electronic diary (eDiary), including their current most bothersome migraine symptom within 45 days of randomization.

Participant milestones

Participant milestones
Measure	Rimegepant 75 Milligram (mg) Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to 7 days post-dose.	Placebo Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Overall Study STARTED	716	715
Overall Study Treated	668	674
Overall Study COMPLETED	709	704
Overall Study NOT COMPLETED	7	11

Reasons for withdrawal

Reasons for withdrawal
Measure	Rimegepant 75 Milligram (mg) Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to 7 days post-dose.	Placebo Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Overall Study Other	0	5
Overall Study COVID-19	0	1
Overall Study Adverse Event	1	1
Overall Study Withdrawal by Subject	2	4
Overall Study Participant anxious to go to their hometown and withdrew from study	1	0
Overall Study Participant refused to come to the hospital	1	0
Overall Study Participant refused the visit	1	0
Overall Study Participant quit early because of business trip	1	0

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Rimegepant 75 mg n=668 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.	Total n=1342 Participants Total of all reporting groups
Age, Continuous	37.8 Years STANDARD_DEVIATION 10.13 • n=668 Participants	37.7 Years STANDARD_DEVIATION 10.70 • n=674 Participants	37.8 Years STANDARD_DEVIATION 10.42 • n=1342 Participants
Sex: Female, Male Female	526 Participants n=668 Participants	563 Participants n=674 Participants	1089 Participants n=1342 Participants
Sex: Female, Male Male	142 Participants n=668 Participants	111 Participants n=674 Participants	253 Participants n=1342 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.

PRIMARY outcome

Timeframe: 2 hours post-dose

Population: Modified intent to treat (mITT) participants included all randomized participants who took study drug, had a migraine of moderate or severe intensity at the time of treatment, and provided at least 1 post-treatment efficacy data point.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=666 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants Who Had Freedom From Pain at 2 Hours Post-dose	19.8 Percentage of participants Interval 16.9 to 23.1	10.7 Percentage of participants Interval 8.5 to 13.3

PRIMARY outcome

Timeframe: 2 hours post-dose

MBS included nausea, phonophobia or photophobia. MBS were measured using a binary scale as 0= absent, 1= present. Participants who had score of 0 (MBS absent) were considered to have freedom from MBS. Exact 95% CI was based on Clopper-Pearson method.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=666 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants Who Had Freedom From Most Bothersome Symptoms (MBS) at 2 Hours Post-dose	50.5 Percentage of participants Interval 46.6 to 54.3	35.8 Percentage of participants Interval 32.1 to 39.5

SECONDARY outcome

Timeframe: 2 hours post-dose

Pain was measured on a 4-point Likert scale, with following scores: 0= none, 1= mild, 2= moderate, 3= severe. Participants who reported a pain level of moderate or severe at baseline and then reported a pain level of none or mild at 2 hours post-dose, were considered to have pain relief. Exact 95% CI was based on Clopper-Pearson method.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=666 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants With Pain Relief at 2 Hours Post-dose	66.5 Percentage of participants Interval 62.8 to 70.1	48.5 Percentage of participants Interval 44.7 to 52.4

SECONDARY outcome

Timeframe: 2 hours post-dose

Population: mITT participants included all randomized participants who took study drug, had a migraine of moderate or severe intensity at the time of treatment, and provided at least 1 post-treatment efficacy data point. Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Participants rated the level of disability they perceived as a result of their migraine in performing normal actions using following level of severity: normal function, mild impairment, severe impairment, or required bedrest. This outcome measure was analyzed only among those participants who reported any impairment at baseline. Percentage of participants with a response of "normal function" at the 2 hours post-dose were reported in this outcome measure.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=545 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=551 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants Who Functioned Normally at 2 Hours Post-dose	40.7 Percentage of participants Interval 36.6 to 45.0	23.8 Percentage of participants Interval 20.3 to 27.6

SECONDARY outcome

Timeframe: 24 hours post-dose

Percentage of participants who used rescue medications within 24 hours of administration of study drug were reported in this outcome measure. Exact 95% CI was based on Clopper-Pearson method.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=666 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants Who Used Rescue Medication Within 24 Hours Post-dose	8.4 Percentage of participants Interval 6.4 to 10.8	20.0 Percentage of participants Interval 17.1 to 23.3

SECONDARY outcome

Timeframe: 2 to 24 hours post-dose

Pain was measured on a 4-point Likert scale, with following scores: 0= none, 1= mild, 2= moderate, 3= severe. Participants with score of 0 (with no pain) through 2 to 24 hours post-dose were considered to have sustained pain freedom. Exact 95% CI was based on Clopper-Pearson method.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=666 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants Who Sustained Pain Freedom From 2 to 24 Hours Post-dose	15.6 Percentage of participants Interval 12.9 to 18.6	7.9 Percentage of participants Interval 5.9 to 10.2

SECONDARY outcome

Timeframe: 2 to 48 hours post-dose

Pain was measured on a 4-point Likert scale, with following scores: 0= none, 1= mild, 2= moderate, 3= severe. Participants with score of 0 (with no pain) through 2 to 48 hours post-dose were considered to have sustained pain freedom. Exact 95% CI was based on Clopper-Pearson method.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=666 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants Who Sustained Pain Freedom From 2 to 48 Hours Post-dose	14.9 Percentage of participants Interval 12.2 to 17.8	7.1 Percentage of participants Interval 5.3 to 9.3

SECONDARY outcome

Timeframe: 15, 30, 45, 60 and 90 minutes post-dose

Pain was measured on a 4-point Likert scale, with following scores: 0= none, 1= mild, 2= moderate, 3= severe. Percentage of participants who reported a pain level of moderate or severe just before taking study treatment and then reported a pain level of none at the specified timepoints. Exact 95% CI was based on Clopper-Pearson method.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=666 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants Who Had Freedom From Pain at 15, 30, 45, 60 and 90 Minutes Post-dose 15 minutes post-dose	0.8 Percentage of participants Interval 0.2 to 1.7	1.5 Percentage of participants Interval 0.7 to 2.7
Percentage of Participants Who Had Freedom From Pain at 15, 30, 45, 60 and 90 Minutes Post-dose 30 minutes post-dose	1.1 Percentage of participants Interval 0.4 to 2.2	1.0 Percentage of participants Interval 0.4 to 2.1
Percentage of Participants Who Had Freedom From Pain at 15, 30, 45, 60 and 90 Minutes Post-dose 45 minutes post-dose	3.3 Percentage of participants Interval 2.1 to 5.0	2.2 Percentage of participants Interval 1.3 to 3.6
Percentage of Participants Who Had Freedom From Pain at 15, 30, 45, 60 and 90 Minutes Post-dose 60 minutes post-dose	6.8 Percentage of participants Interval 5.0 to 8.9	4.5 Percentage of participants Interval 3.0 to 6.3
Percentage of Participants Who Had Freedom From Pain at 15, 30, 45, 60 and 90 Minutes Post-dose 90 minutes post-dose	12.3 Percentage of participants Interval 9.9 to 15.1	7.1 Percentage of participants Interval 5.3 to 9.3

SECONDARY outcome

Timeframe: 15, 30, 45, 60 and 90 minutes post-dose

MBS included nausea, phonophobia or photophobia. MBS were measured using a binary scale as 0= absent, 1= present. Participants who had score of 0 (MBS absent) were considered to have freedom from MBS at the specified timepoints. Exact 95% CI was based on Clopper-Pearson method.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=666 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants Who Had Freedom From MBS at 15, 30, 45, 60 and 90 Minutes Post-dose 15 minutes post-dose	10.2 Percentage of participants Interval 8.0 to 12.8	11.1 Percentage of participants Interval 8.9 to 13.7
Percentage of Participants Who Had Freedom From MBS at 15, 30, 45, 60 and 90 Minutes Post-dose 30 minutes post-dose	16.8 Percentage of participants Interval 14.1 to 19.9	14.8 Percentage of participants Interval 12.2 to 17.7
Percentage of Participants Who Had Freedom From MBS at 15, 30, 45, 60 and 90 Minutes Post-dose 45 minutes post-dose	24.2 Percentage of participants Interval 21.0 to 27.6	19.9 Percentage of participants Interval 16.9 to 23.1
Percentage of Participants Who Had Freedom From MBS at 15, 30, 45, 60 and 90 Minutes Post-dose 60 minutes post-dose	31.1 Percentage of participants Interval 27.6 to 34.7	24.8 Percentage of participants Interval 21.6 to 28.2
Percentage of Participants Who Had Freedom From MBS at 15, 30, 45, 60 and 90 Minutes Post-dose 90 minutes post-dose	41.4 Percentage of participants Interval 37.7 to 45.3	31.8 Percentage of participants Interval 28.2 to 35.4

SECONDARY outcome

Timeframe: 2 Hours to 48 Hours Post-dose

Pain was measured on a 4-point Likert scale, with following scores: 0= none, 1= mild, 2= moderate, 3= severe. This outcome measure was analyzed only in those participants who were pain free at 2 hours post-dose. Percentage of participants who were pain free at 2 hours post-dose and then had a migraine of any pain severity (score 2 or 3 on the 4-point scale) within 48 hours after administration of study drug were considered to have pain relapse. Exact 95% CI was based on Clopper-Pearson method.

Outcome measures

Outcome measures
Measure	Rimegepant 75 mg n=132 Participants Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=72 Participants Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Percentage of Participants With Pain Relapse	25.0 Percentage of participants Interval 17.9 to 33.3	33.3 Percentage of participants Interval 22.7 to 45.4

Adverse Events

Rimegepant 75 mg

Serious events: 1 serious events

Other events: 92 other events

Deaths: 0 deaths

Placebo

Serious events: 2 serious events

Other events: 94 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Rimegepant 75 mg n=668 participants at risk Participants were randomized to receive 1 orally disintegrating tablet of rimegepant 75 mg. Participants were followed up to for 7 days post-dose.	Placebo n=674 participants at risk Participants were randomized to receive placebo matched to rimegepant. Participants were followed up to 7 days post-dose.
Gastrointestinal disorders Haemoperitoneum	0.00% 0/668 • Up to 7 days post-dose Same event may appear as adverse event and serious adverse event, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Treated population evaluated.	0.15% 1/674 • Up to 7 days post-dose Same event may appear as adverse event and serious adverse event, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Treated population evaluated.
Infections and infestations Infection	0.15% 1/668 • Up to 7 days post-dose Same event may appear as adverse event and serious adverse event, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Treated population evaluated.	0.00% 0/674 • Up to 7 days post-dose Same event may appear as adverse event and serious adverse event, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Treated population evaluated.
Injury, poisoning and procedural complications Decreased embryo viability	0.00% 0/668 • Up to 7 days post-dose Same event may appear as adverse event and serious adverse event, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Treated population evaluated.	0.15% 1/674 • Up to 7 days post-dose Same event may appear as adverse event and serious adverse event, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Treated population evaluated.
Pregnancy, puerperium and perinatal conditions Ectopic pregnancy	0.00% 0/668 • Up to 7 days post-dose Same event may appear as adverse event and serious adverse event, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Treated population evaluated.	0.15% 1/674 • Up to 7 days post-dose Same event may appear as adverse event and serious adverse event, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Treated population evaluated.

Other adverse events

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer Inc.

Phone: 1-800-718-1021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Publication restrictions are in place

Restriction type: OTHER