Migraine DisordersPhase 4NCT04050293
Therapy for Migraine Prevention in Children 6-11 Years of Age
A Phase 4 study testing SPN-538 in people with Migraine Disorders and Headache Disorders, run by Supernus Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
The aim of this study is to evaluate the effect of SPN-538 for the prophylaxis of migraine in pediatric patients 6 to 11 years old. Read more about this study
- Who is it for?
- Ages 6 to 11 years
- Conditions the study is for
- Migraine Disorders, Headache Disorders
- What is being tested?
- SPN-538
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Jul 2020to 1 Mar 2023
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Otherwise, healthy male or female (6 to 11 years of age at the time of screening) with a history of migraine with or without aura for at least 6 months prior to screening, 3-12 migraine episodes and no more than 14 headache days (migraine and non-migraine) per month during the 3 months prior to Screening and during the 28-day Prospective Baseline Period, a PedMIDAS Disability score of ≥20 and ≤139 and weight at least 20 kg and no more than 60 kg.
Who cannot join
- Subjects with chronic migraine (>14 headache days per month), cluster headaches, or migraine aura without headache and who are unable to distinguish migraines from other headache types.
- Subjects with more than 14 headache days during the 28-day Prospective Baseline Period
- Have taken any disallowed migraine preventive medication including TPM within 14 days prior to the start of the Prospective Baseline Period; or used onabotulinumtoxinA (Botox®) within 3 months prior to Screening.
- Failure to respond to topiramate prophylaxis therapy (2 to 3 mg/kg/day) for a minimum of 3 months, or those who have previously discontinued TPM due to AEs within 6 months prior to Screening.
- Failure to more than 2 adequate clinical trials of an established prophylactic antimigraine regimen within 6 months prior to Screening.
- Current use of antipsychotics, antimanics, barbiturates, benzodiazepines amitriptyline, lithium, valproic acid, tricyclic antidepressants, AEDs, calcium channel blockers, sedatives, SSRIs, NSRIs, CGRP receptor antagonists, CBD oil, herbal preparations/supplements such as feverfew or St John's wort and/or any medications that could impair or decrease thinking and concentration.
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- Overuse of analgesic or migraine-specific agents for acute treatment of migraine (>10 treatment days/month of ergot-containing medications or triptans; or >15 treatment days/month with simple analgesics (including non steroidal anti-inflammatory drugs [NSAIDs]), or use of narcotics.
- Use of non-pharmacologic complementary and alternative prophylactic approaches for migraine prevention, such as neuromodulation, acupuncture, behavioral interventions, spinal manipulation, occipital nerve block and neurofeedback.
- Diagnosis of psychiatric disorder (e.g., psychosis, bipolar disorder, major depression, generalized anxiety disorders), or documented developmental delays or impairments (e.g., autism, cerebral palsy, or mental retardation).
- Subjects with seizures or a history of seizure-like events.
- Known history of visual field defects, neurological disorder or structural disorder of the brain from birth; head trauma or previous CNS surgery.
- Evidence of active suicidal ideation and/or suicidal behaviors 6 months before screening, pregnancy, active liver disease or abnormal kidney function.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Parallel Group Study to Evaluate the Efficacy and Safety of SPN-538 as a Therapy for the Prevention of Migraine in Subjects Ages 6-11 Years
The aim of this study is to evaluate the effect of SPN-538 for the prophylaxis of migraine in pediatric patients 6 to 11 years old.
Read the detailed description
The study is to assess the efficacy and safety of SPN-538 in reducing the monthly migraine headache frequency in pediatric patients with migraine.
This is a multicenter, randomized, double-blind, placebo-controlled, 2-arm, parallel group Phase 4 study in children 6 to 11 years of age to evaluate the efficacy and safety of SPN-538 for the prevention of migraine. SPN-538 (or matching placebo) will be administered as a single oral dose once a day (QD), starting at 25 mg/day and increasing every 2 weeks in 25 mg increments to a target dose of 2 to 3 mg/kg/day or the maximum tolerated dose (MTD), whichever is less. The total study duration is a maximum of 34 weeks including a Screening Period, Prospective Baseline Period of up to 8 weeks, a Treatment Phase of 20 weeks (8 weeks titration followed by 12 weeks of maintenance dosing), a Dose Tapering Period of 1 to 3 weeks, and a Safety Follow-up Period of 1 to 3 weeks
- Conditions
- Migraine Disorders, Headache Disorders
- Being tested
- SPN-538, Placebo
- Medical subject headings
- Migraine Disorders, Headache Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPN-538Drug
Participants will receive SPN-538
PlaceboDrug
Participants will receive Placebo
| Study group | Role | What they receive |
|---|---|---|
| SPN-538 (Topiramate XR capsule) | Experimental | Participants will be treated with SPN-538 Receives: Drug: SPN-538 |
| Placebo | Placebo comparator | Participants will be treated with Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Monthly Migraine Days (MMDs) in Children 6 to 11 Years Old With Migraine.
The primary efficacy endpoint was the change in rate of the MMDs relative to the Prospective Baseline Period (PBP). The 28-day rate of MMDs was calculated as the ratio of the # days of migraine during the last 4 weeks of PBP and the # days with non-missing headache record in the electronic diary during the last 4 weeks of the 20-week double-blind Treatment Phase, x 28. The primary outcome measure was recorded on headache electronic diary uploaded on a Patient Reported Outcome (ePRO) application. The electronic diary will serve as the primary tool to collect daily headaches information.
Measured: Baseline, Titration Period 1 (Month 1), Titration Period 2 (Month 2), Maintenance Period 1 (Month 3), Maintenance Period 2 (Month 3), Maintenance Period 2 (Month 4) and Maintenance Period 3 (Month 5)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2019
- Study start14 Jul 2020
- Primary completion1 Mar 2023
- Study completion1 Mar 2023
Why it stopped: Difficulty in recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Migraine DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double-blind, randomized, placebo-controlled, 2-arm, parallel group study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Supernus Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gianpiera Ceresoli-Borroni, PhD, Study director, Employee of Supernus
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04050293
- Sponsor's protocol ID
- 538P401
- Status last verified
- Dec 2025
- First submitted
- 2 Aug 2019
- Last updated
- 22 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Parallel Group Study to Evaluate the Efficacy and Safety of SPN-538 as a Therapy for the Prevention of Migraine in Subjects Ages 6-11 Years
- ClinicalTrials.gov ID
- NCT04050293
- Acronym / sponsor ID
- 538P401
- Conditions
- Migraine Disorders, Headache Disorders
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Difficulty in recruitment
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Supernus Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Jul 2020 (actual)
- Primary completion
- 1 Mar 2023 (actual)
- Study completion
- 1 Mar 2023 (actual)
- First posted
- 8 Aug 2019 (actual)
- Results
- Posted 21 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to evaluate the effect of SPN-538 for the prophylaxis of migraine in pediatric patients 6 to 11 years old.
Detailed description
The study is to assess the efficacy and safety of SPN-538 in reducing the monthly migraine headache frequency in pediatric patients with migraine.
This is a multicenter, randomized, double-blind, placebo-controlled, 2-arm, parallel group Phase 4 study in children 6 to 11 years of age to evaluate the efficacy and safety of SPN-538 for the prevention of migraine. SPN-538 (or matching placebo) will be administered as a single oral dose once a day (QD), starting at 25 mg/day and increasing every 2 weeks in 25 mg increments to a target dose of 2 to 3 mg/kg/day or the maximum tolerated dose (MTD), whichever is less. The total study duration is a maximum of 34 weeks including a Screening Period, Prospective Baseline Period of up to 8 weeks, a Treatment Phase of 20 weeks (8 weeks titration followed by 12 weeks of maintenance dosing), a Dose Tapering Period of 1 to 3 weeks, and a Safety Follow-up Period of 1 to 3 weeks
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Double-blind, randomized, placebo-controlled, 2-arm, parallel group study
| Arm | Type | Receives |
|---|---|---|
| SPN-538 (Topiramate XR capsule) | Experimental | Drug: SPN-538Participants will be treated with SPN-538 |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will be treated with Placebo |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Otherwise, healthy male or female (6 to 11 years of age at the time of screening) with a history of migraine with or without aura for at least 6 months prior to screening, 3-12 migraine episodes and no more than 14 headache days (migraine and non-migraine) per month during the 3 months prior to Screening and during the 28-day Prospective Baseline Period, a PedMIDAS Disability score of ≥20 and ≤139 and weight at least 20 kg and no more than 60 kg.
Key exclusion criteria (12 listed)
- Subjects with chronic migraine (>14 headache days per month), cluster headaches, or migraine aura without headache and who are unable to distinguish migraines from other headache types.
- Subjects with more than 14 headache days during the 28-day Prospective Baseline Period
- Have taken any disallowed migraine preventive medication including TPM within 14 days prior to the start of the Prospective Baseline Period; or used onabotulinumtoxinA (Botox®) within 3 months prior to Screening.
- Failure to respond to topiramate prophylaxis therapy (2 to 3 mg/kg/day) for a minimum of 3 months, or those who have previously discontinued TPM due to AEs within 6 months prior to Screening.
- Failure to more than 2 adequate clinical trials of an established prophylactic antimigraine regimen within 6 months prior to Screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SPN-538Participants will receive SPN-538 | Drug | SPN-538 (Topiramate XR capsule) |
| PlaceboParticipants will receive Placebo | Drug | Placebo |
Primary outcome
Change From Baseline in Monthly Migraine Days (MMDs) in Children 6 to 11 Years Old With Migraine.
Time frame: Baseline, Titration Period 1 (Month 1), Titration Period 2 (Month 2), Maintenance Period 1 (Month 3), Maintenance Period 2 (Month 3), Maintenance Period 2 (Month 4) and Maintenance Period 3 (Month 5)
The primary efficacy endpoint was the change in rate of the MMDs relative to the Prospective Baseline Period (PBP). The 28-day rate of MMDs was calculated as the ratio of the # days of migraine during the last 4 weeks of PBP and the # days with non-missing headache record in the electronic diary during the last 4 weeks of the 20-week double-blind Treatment Phase, x 28. The primary outcome measure was recorded on headache electronic diary uploaded on a Patient Reported Outcome (ePRO) application. The electronic diary will serve as the primary tool to collect daily headaches information.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gianpiera Ceresoli-Borroni, PhD study director
Employee of Supernus
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing SPN-538 in people with Migraine Disorders and Headache Disorders, run by Supernus Pharmaceuticals, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Nov 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Monthly Migraine Days (MMDs) in Children 6 to 11 Years Old With Migraine. (time frame: Baseline, Titration Period 1 (Month 1), Titration Period 2 (Month 2), Maintenance Period 1 (Month 3), Maintenance Period 2 (Month 3), Maintenance Period 2 (Month 4) and Maintenance Period 3 (Month 5)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jan 2026.