Eptinezumab for Pediatric Migraine

(PROSPECT-1 Trial)

Recruiting at 68 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

Trial Summary

What is the purpose of this trial?

The main goal of this trial is to learn whether eptinezumab helps reduce the number of days with episodic migraine in pediatric participants.

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.

What data supports the effectiveness of the drug Eptinezumab for pediatric migraine?

Eptinezumab has been shown to reduce the number of migraine days in adults, with significant reductions in both episodic and chronic migraines. Although specific data for pediatric use is not provided, its effectiveness in adults suggests potential benefits for children as well. ^{1 2 3 4 5}

Is eptinezumab safe for use in humans?

Eptinezumab, also known as Vyepti, is generally considered safe for preventing migraines in adults, with common mild side effects like nasal congestion and upper respiratory infections. It has been well tolerated in studies, even in patients with other health conditions like obesity and diabetes, and long-term safety studies show it is well tolerated over time. ^{1 3 4 6 7}

How is the drug Eptinezumab different from other migraine treatments?

Eptinezumab is unique because it is given as a quarterly infusion, which means it is administered directly into the bloodstream every three months. This method allows for a quick onset of action, which can be especially helpful for people with severe or hard-to-treat migraines. ^{1 2 4 5 8}

Research Team

Email contact via H. Lundbeck A/S

Principal Investigator

[e-mail]

Eligibility Criteria

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

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 have been diagnosed with migraines for at least 6 months.

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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, 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]

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 (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 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, 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]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Eptinezumab: First Approval. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Eptinezumab-jjmr, a humanized monoclonal specific to Calcitonin Gene Related Peptide, for the preventive treatment of migraine in adults. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Eptinezumab for adolescents with chronic refractory headache: A retrospective chart review. [2023]

4. England 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. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Eptinezumab: A calcitonin gene-related peptide monoclonal antibody infusion for migraine prevention. [2021]

6. United States pubmed.ncbi.nlm.nih.gov

A Review of Eptinezumab Use in Migraine. [2023]

7. England 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. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Long-term reductions in disease impact in patients with chronic migraine following preventive treatment with eptinezumab. [2022]