Eptinezumab for Pediatric Migraine

(PROSPECT-1 Trial)

Recruiting at 96 trial locations

Overseen By Email contact via H. Lundbeck A/S

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: H. Lundbeck A/S

Disqualifiers: Chronic headaches, Severe head trauma, others

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 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

315 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if eptinezumab (also known as Vyepti, a monoclonal antibody) can reduce the number of migraine days in children and teens. Participants will receive either varying doses of eptinezumab or a placebo (a harmless, inactive substance) through an IV infusion. The trial suits children and teens who have experienced migraines for at least six months and have at least four migraine days per month. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking advancements in migraine treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that eptinezumab has been tested in over 2,000 people for migraine prevention. In these studies, most participants tolerated eptinezumab well without serious side effects. Common side effects included a stuffy nose and sore throat.

Eptinezumab is approved for adults to prevent migraines, indicating its safety for them. However, researchers are still studying its safety and effectiveness for children. Early tests in young people suggest the treatment is manageable, but more data is needed to fully understand its safety for kids.

For those considering joining the trial, this information may help provide a clearer understanding of eptinezumab's safety. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard migraine treatments for children, which typically include oral medications like ibuprofen or triptans, eptinezumab offers a novel approach by targeting the migraine process itself. Eptinezumab is a monoclonal antibody that specifically blocks the calcitonin gene-related peptide (CGRP), a protein that plays a key role in causing migraines. What sets eptinezumab apart is its delivery method: it is administered as a single intravenous infusion, which could potentially offer faster and more sustained relief than traditional oral medications. Researchers are excited about eptinezumab because it targets the root cause of migraines and could provide long-lasting relief with just one dose, offering a promising new option for young migraine sufferers.

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

Research has shown that eptinezumab can significantly reduce migraine frequency. In one study, 40% of patients who received 300 mg of eptinezumab experienced no migraines for at least a month. Another study found that eptinezumab consistently met its goals in lowering the number of migraine days each month. In this trial, participants will receive either 300 mg or 100 mg of eptinezumab, both of which have proven effective in reducing migraine frequency. Eptinezumab has already been shown to prevent migraines in adults, and early trials with young people appear promising. These findings suggest that eptinezumab could be an effective option for reducing migraines in children. ^{2 6 7 8 9}

Who Is on the Research Team?

Email contact via H. Lundbeck A/S

Principal Investigator

[e-mail]

Are You a Good Fit for This Trial?

This trial is for kids with episodic migraines, diagnosed per international standards, who've had migraines for at least 6 months. They must have tracked their headaches in an eDiary during the screening period. It's not for those with severe head trauma, other major neurological disorders, unusual migraine types like hemiplegic migraine or if they have uncontrolled psychiatric conditions.

Inclusion Criteria

I have been diagnosed with migraines for at least 6 months.

During the 28-day screening period, the participant (and their parent/caregiver, when applicable) must adequately complete the headache eDiary (≥23 of the 28 days) following the day of the Screening Visit

I've had 14 or fewer headache days, with at least 4 being migraines, in the last 28 days.

Exclusion Criteria

My mental health condition has been stable and treated for at least 6 months.

History of moderate or severe head trauma or other neurological disorder or systemic medical disease that is, in the investigator's opinion, likely to affect the functions of the central nervous system

I have been diagnosed with a specific type of severe or unusual headache.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous infusion of eptinezumab or placebo

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Eptinezumab

Trial Overview The study aims to find out if Eptinezumab can reduce the number of days children experience episodic migraines compared to a placebo (a treatment with no active drug). Participants will be randomly assigned to receive either Eptinezumab or the placebo.

How Is the Trial Designed?

Group I: Eptinezumab 300 mg Experimental Treatment 1 Intervention

Participants will receive a single intravenous (IV) infusion of eptinezumab 300 mg (weight adjusted).

Group II: Eptinezumab 100 mg Experimental Treatment 1 Intervention

Participants will receive a single IV infusion of eptinezumab 100 mg (weight adjusted).

Group III: Placebo Placebo Group 1 Intervention

Participants will receive a single IV infusion of matching placebo to eptinezumab.

Eptinezumab is already approved in United States for the following indications:

Approved in United States as Vyepti for:

• Preventive treatment of migraine in adults

Who Is Running the Clinical Trial?

H. Lundbeck A/S

Lead Sponsor

Trials

332

Recruited

78,300+

Charl van Zyl

H. Lundbeck A/S

Chief Executive Officer since 2023

Degree in Medical Biochemistry from the University of Cape Town, South Africa

Johan Luthman

H. Lundbeck A/S

Chief Medical Officer since 2019

MD from the University of Gothenburg, Sweden

Published Research Related to This Trial

Eptinezumab (Vyepti) is an effective preventive treatment for migraines, showing benefits from day 1 and maintaining efficacy for up to a year at doses of 100 mg and 300 mg, with no drug interactions reported.

The safety profile of eptinezumab is favorable, with low frequency of treatment-related adverse events, primarily mild conditions like nasopharyngitis and upper respiratory infections, and it is safe for patients with comorbidities such as obesity and type 1 diabetes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Review of Eptinezumab Use in Migraine. Datta, A., Maryala, S., John, R.[2023]

Eptinezumab-jjmr is a humanized monoclonal antibody targeting CGRP, showing significant efficacy in preventing migraines, which are a prevalent and debilitating condition affecting many adults.

Traditional migraine treatments often have poor adherence and limited effectiveness, highlighting the need for new therapies like Eptinezumab, which aims to fill these gaps in migraine management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Eptinezumab-jjmr, a humanized monoclonal specific to Calcitonin Gene Related Peptide, for the preventive treatment of migraine in adults. Berger, AA., Keefe, J., Stark, CW., et al.[2022]

Eptinezumab significantly reduced the mean monthly migraine days in adults with two-to-four previous preventive treatment failures, showing a reduction of -2.7 days for the 100 mg dose and -3.2 days for the 300 mg dose compared to placebo, indicating its efficacy as a preventive treatment.

The safety profile of eptinezumab was acceptable, with treatment-emergent adverse events occurring in 42% of the 100 mg group and 41% of the 300 mg group, similar to the 40% in the placebo group, suggesting it is well-tolerated among patients with a history of treatment failures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of eptinezumab for migraine prevention in patients with two-to-four previous preventive treatment failures (DELIVER): a multi-arm, randomised, double-blind, placebo-controlled, phase 3b trial. Ashina, M., Lanteri-Minet, M., Pozo-Rosich, P., et al.[2022]

Eptinezumab, a newly approved treatment for migraines, significantly reduces monthly migraine days by about 4 days for episodic migraine and 8 days for chronic migraine compared to placebo, with a low number needed to treat of 6.

The treatment has a quick onset of action, showing effects as soon as one day after infusion, and is generally well-tolerated, with nausea and fatigue being the most common side effects and a low incidence of serious adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Eptinezumab: A calcitonin gene-related peptide monoclonal antibody infusion for migraine prevention. Morgan, KW., Joyner, KR.[2021]

Eptinezumab, administered as a 300 mg intravenous treatment every 12 weeks, showed a favorable safety profile in a 2-year study involving 128 adults with chronic migraine, with only a 6.3% discontinuation rate due to adverse events.

Patients reported early and sustained improvements in migraine-related symptoms and overall quality of life, with benefits observed as early as 4 weeks and maintained through the end of the study at week 104.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and tolerability of eptinezumab in patients with chronic migraine: a 2-year, open-label, phase 3 trial. Kudrow, D., Cady, RK., Allan, B., et al.[2021]

In a small study of 11 adolescents with chronic refractory headaches, eptinezumab (100 mg) showed promising results, with some patients experiencing complete cessation of daily headaches and reductions in headache intensity after the first infusion.

Eptinezumab was well tolerated among participants, and subsequent higher doses (300 mg) led to further improvements in headache frequency and intensity, suggesting its potential as a safe treatment option for this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Eptinezumab for adolescents with chronic refractory headache: A retrospective chart review. Zorrilla, N., Gelfand, AA., Irwin, SL.[2023]

Eptinezumab, an anti-CGRP monoclonal antibody, demonstrated significant long-term efficacy in reducing migraine-related disability, with mean MIDAS headache days decreasing from 47.4 at baseline to 16.3 after 104 weeks of treatment.

Patients experienced sustained reductions in both headache frequency and pain severity, with notable improvements in absenteeism and presenteeism, indicating that eptinezumab effectively enhances the quality of life for individuals with chronic migraine.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term reductions in disease impact in patients with chronic migraine following preventive treatment with eptinezumab. Blumenfeld, A., Ettrup, A., Hirman, J., et al.[2022]

Eptinezumab is a humanized monoclonal antibody that effectively blocks the action of calcitonin gene-related peptide (CGRP), a key player in migraine development, making it a targeted treatment for migraine prevention.

Approved in February 2020 for adults, eptinezumab is administered intravenously and represents a significant advancement in migraine therapy, highlighting its efficacy in preventing migraine attacks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Eptinezumab: First Approval. Dhillon, S.[2022]

Citations

1. vyeptihcp.com vyeptihcp.com/efficacy-and-patient-outcomes

Efficacy & Patient Outcomes | VYEPTI® (eptinezumab-jjmr) During a 6-month period chronic migraine pivotal trial, 40% of patients treated with VYEPTI 300 mg were 100% migraine free for a month or more vs 22% with ...

2. prnewswire.com prnewswire.com/news-releases/new-data-demonstrates-robust-efficacy-of-vyepti-eptinezumab-in-otherwise-difficult-to-treat-patients-with-severe-migraine-302487652.html

New data demonstrates robust efficacy of Vyepti® ... In the RESOLUTION trial (n = 608), eptinezumab met the both the primary and secondary endpoints, showing significantly greater monthly migraine ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05897320

NCT05897320 | A Study of Eptinezumab in Pediatric ... The main goal of this trial is to learn whether eptinezumab helps reduce the number of days with episodic migraine in pediatric participants.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10444733/

Effectiveness and Safety of Eptinezumab in Episodic and ... Across participants, administration of 100 mg of eptinezumab appeared to result in at least moderate reductions in migraine frequency and ...

5. headachejournal.onlinelibrary.wiley.com headachejournal.onlinelibrary.wiley.com/doi/10.1111/head.14959

Pharmacokinetics and safety of eptinezumab in children and ... Eptinezumab is proven to prevent migraine in adults, and in this open-label trial, we gave eptinezumab to young people with migraine to see if ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2020/761119s000lbl.pdf

VYEPTI (eptinezumab-jjmr) - accessdata.fda.gov The safety of VYEPTI was evaluated in 2076 patients with migraine who received at least one dose of VYEPTI, representing 1615 patient-years of exposure; of ...

7. nature.com nature.com/articles/s41598-025-09490-1

Comprehensive safety analysis of adverse events ... Migraine significantly impacts quality of life, with eptinezumab emerging as a promising calcitonin gene-related peptide-targeting therapy.

8. ema.europa.eu ema.europa.eu/en/documents/product-information/vyepti-epar-product-information_en.pdf

Vyepti, eptinezumab - EMA Paediatric population The safety and efficacy of VYEPTI in children aged 6 to 18 years has not yet been established. Currently no data are available. There is ...

9. vyeptihcp.com vyeptihcp.com/safety-and-tolerability

VYEPTI Safety Profile & Tolerability Learn about VYEPTI safety results from both pivotal trials, the long-term, open-label safety and tolerability study, and common adverse reactions.