Rimegepant for Pediatric Migraine

Recruiting at 234 trial locations

Overseen By Pfizer Pfizer CT.gov Call Center

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Biohaven Pharmaceuticals, Inc.

Must be taking: Prophylactic migraine

Must not be taking: Drugs of abuse

Disqualifiers: Cluster headache, Hemiplegic migraine, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 58 weeks

600 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the long-term safety of rimegepant for treating migraines in children and teenagers. It focuses on those who have experienced migraines for at least six months and regularly suffer moderate to severe attacks. Participants must distinguish between migraines and other headaches and have been stable on any preventive migraine medication for at least 12 weeks. The trial aims to assess how well rimegepant reduces migraine pain and frequency over time. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new migraine treatment.

Will I have to stop taking my current medications?

The trial allows participants to continue taking their current migraine prevention medication if the dose has been stable for at least 12 weeks before the study starts. The protocol does not specify about other medications, so it's best to discuss with the trial team.

Is there any evidence suggesting that rimegepant is likely to be safe for children and adolescents?

Research has shown that rimegepant is generally safe for treating migraines. One study found that participants who took 75 mg of rimegepant for up to a year mostly did not experience serious side effects, indicating a good safety record.

However, taking rimegepant more than 18 times a month has not been proven safe and may lead to more headaches.

Overall, rimegepant is considered safe for many people when used as directed. Prospective trial participants should discuss any concerns with their doctor. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for migraine?

Rimegepant is unique because it offers a new way to tackle pediatric migraines by working as a calcitonin gene-related peptide (CGRP) receptor antagonist. Unlike typical treatments like NSAIDs and triptans, which often focus on pain relief after a migraine starts, rimegepant targets the migraine pathway directly to prevent and treat attacks. Additionally, its orally disintegrating tablet (ODT) form provides a convenient and fast-acting option for children who might have difficulty swallowing pills. Researchers are excited about its potential to provide quicker relief and fewer side effects compared to traditional migraine medications.

What evidence suggests that rimegepant might be an effective treatment for pediatric migraine?

Research has shown that rimegepant, the investigational treatment in this trial, effectively treats migraines. In studies, about 50% of participants taking rimegepant experienced significant pain relief within 2 hours. This relief was often as good as or better than that from other migraine treatments. Additionally, around 42.2% of users experienced pain relief lasting up to 48 hours. Rimegepant targets a protein involved in migraine attacks, helping to ease symptoms. These findings suggest that rimegepant may be a promising option for managing migraines. ^{1 5 6 7 8}

Who Is on the Research Team?

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Are You a Good Fit for This Trial?

This trial is for children and teens aged 6 to under 18 with a history of migraines, weighing at least 40 kg. They should have experienced 1-8 moderate or severe migraine attacks per month in the last two months and be able to tell migraines apart from other headaches. Stable use of preventive migraine medication is allowed.

Inclusion Criteria

I am between 6 and 17 years old.

I needed treatment for a migraine at least once during the observation period.

You can tell the difference between migraines and other kinds of headaches.

Exclusion Criteria

You are currently using or have a history of using drugs, and your drug test at the screening shows positive results.

I have not had untreated mental health issues for the past 6 months.

I have a history of cluster or hemiplegic migraine headaches.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive rimegepant for the acute treatment of migraine

58 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Rimegepant / BHV3000

Trial Overview The study tests the long-term safety of Rimegepant (BHV3000) for treating acute, moderate or severe migraines in young participants. It aims to understand how well they tolerate this medication over an extended period.

How Is the Trial Designed?

Group I: Active Experimental Treatment 1 Intervention

rimegepant 75 mg, 50 mg or 35 mg ODT

Rimegepant / BHV3000 is already approved in United States, European Union for the following indications:

Approved in United States as Nurtec ODT for:

• Acute treatment of migraine

Approved in European Union as Vydura for:

• Acute treatment of migraine

Who Is Running the Clinical Trial?

Biohaven Pharmaceuticals, Inc.

Lead Sponsor

Trials

49

Recruited

30,100+

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Published Research Related to This Trial

Rimegepant is an effective treatment for migraines, showing significant pain relief and reduction in bothersome symptoms compared to placebo in phase III trials, and it can also reduce the number of monthly migraine days when used preventively.

The medication is generally well tolerated, with no signs of liver or heart-related side effects, making it a safe option for adults managing migraines.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rimegepant: A Review in the Acute Treatment and Preventive Treatment of Migraine. Blair, HA.[2023]

Rimegepant, an oral CGRP receptor antagonist, has shown significant efficacy in treating migraines, providing pain relief and symptom improvement for up to 48 hours after a single dose, based on completed phase II and III trials.

The medication was well tolerated with only mild to moderate adverse events reported, leading to its FDA approval for acute migraine treatment on February 27, 2020.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rimegepant for the treatment of migraine. Negro, A., Martelletti, P.[2021]

Rimegepant, an orally disintegrating tablet approved by the FDA, has been shown in three phase 3 trials to significantly improve pain relief and reduce bothersome migraine symptoms within 2 hours of administration.

This medication offers a new mechanism of action for treating migraines, making it a suitable option for patients who do not respond well to traditional treatments like triptans or who experience adverse effects from them.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rimegepant Orally Disintegrating Tablet for Acute Migraine Treatment: A Review. DeFalco, AP., Lazim, R., Cope, NE.[2021]

Rimegepant, taken every other day, significantly reduced the mean number of migraine days per month compared to placebo, with a difference of -0.8 days, indicating its efficacy as a preventive treatment for migraines.

The safety profile of rimegepant was comparable to that of placebo, with similar rates of adverse events and no serious safety concerns reported during the study involving 747 participants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Oral rimegepant for preventive treatment of migraine: a phase 2/3, randomised, double-blind, placebo-controlled trial. Croop, R., Lipton, RB., Kudrow, D., et al.[2021]

In a study involving 13 patients with migraines, rimegepant was found to be well tolerated when used as an acute treatment alongside CGRP monoclonal antibodies (mAbs) for preventive treatment, with no serious adverse events reported.

Out of the patients, 38% experienced mild to moderate adverse events, primarily nasopharyngitis, indicating that while some side effects occurred, they were generally not severe and did not lead to treatment discontinuation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety of Rimegepant, an Oral CGRP Receptor Antagonist, Plus CGRP Monoclonal Antibodies for Migraine. Berman, G., Croop, R., Kudrow, D., et al.[2021]

In a phase 3 trial involving 1466 participants, the orally disintegrating tablet formulation of rimegepant (75 mg) was significantly more effective than placebo in providing freedom from pain (21% vs 11%) and freedom from the most bothersome symptom (35% vs 27%) at 2 hours post-dose.

The safety profile of rimegepant was comparable to placebo, with no serious adverse events reported, and the most common side effects were mild, such as nausea and urinary tract infections, indicating good tolerability.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy, safety, and tolerability of rimegepant orally disintegrating tablet for the acute treatment of migraine: a randomised, phase 3, double-blind, placebo-controlled trial. Croop, R., Goadsby, PJ., Stock, DA., et al.[2020]

Rimegepant significantly improves outcomes for migraine patients, including freedom from pain and pain relief at 2 hours, with odds ratios indicating strong efficacy compared to placebo.

The treatment shows sustained benefits at 24 hours and does not appear to increase the risk of adverse events, suggesting it is a safe option for migraine management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Rimegepant for Migraine Patients: A Meta-analysis of Randomized Controlled Studies. Yang, C., Zhang, Y.[2023]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10299922/

Rimegepant: A Review in the Acute Treatment and ... Rimegepant was also significantly more effective than placebo irrespective of migraine attack frequency (≤ 4 or > 4 attacks per month) [32], ...

2. practicalneurology.com practicalneurology.com/news/study-compares-nurtec-odt-vs-14-other-treatments-for-acute-migraine/2475275/

Study Compares Nurtec ODT vs 14 Other Treatments for Acute ... 50.0% of participants treated with Nurtec ODT achieved ≥50% pain freedom within 2 hours, which was higher than or similar to other medications ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03732638

NCT03732638 | Efficacy and Safety Trial of Rimegepant for ... The purpose of this is study is to compare the efficacy of BHV-3000 (rimegepant) to placebo as a preventive treatment for migraine, as measured by the ...

4. nurtec.com nurtec.com/

Treatment of Migraine | Nurtec® ODT (rimegepant) | Safety Info Pain relief was sustained in 42.2% of people taking Nurtec ODT at 48 hours (vs 25.2% with placebo). Pain freedom: 21.2% of people taking Nurtec ODT experienced ...

5. jamanetwork.com jamanetwork.com/journals/jama/fullarticle/2770007

Rimegepant (Nurtec ODT) for Acute Treatment of Migraine Limited data suggest that rimegepant may be effective for acute treatment of migraine in patients treated with a CGRP antagonist for migraine ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2025/212728Orig1s025Correctedlbl.pdf

Nurtec ODT - accessdata.fda.gov The safety of using more than 18 doses in a 30-day period has not been established.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11127653/

Safety of Rimegepant in Patients Using Preventive ... Conclusion. Acute treatment of migraine with rimegepant 75 mg for up to 52 weeks was well tolerated and had a favorable safety profile in ...

8. mayoclinic.org mayoclinic.org/drugs-supplements/rimegepant-oral-route/description/drg-20484237

Rimegepant (oral route) - Side effects & dosage Using rimegepant alone or in combination with other migraine medicines for 15 or more days per month may lead to worsening of headache. You may ...