CadasilPhase 2NCT04334408
Safety and Efficacy of Fremanezumab for Migraine in Adult CADASIL
A Phase 2 study testing Fremanezumab in people with Cadasil and Migraine, run by Mayo Clinic.
Trial summary
Researchers are trying to find out more about the side effects of fremanezumab when treating patients with Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL) for migraine headaches. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- What is being tested?
- Fremanezumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Dec 2021to Dec 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 to 70 years old
- English speaking
- Diagnosis of CADASIL confirmed by presence of a pathogenic NOTCH3 gene mutation
- Diagnosis of migraine for at least 12 months and diagnosis of chronic migraine for at least 28 days according to the International Classification of Headache Disorders
- MIDAS score of >10 points
- Ability to provide written informed consent
Who cannot join
- History of ischemic stroke within 4 weeks of screening assessment
- Screening blood pressure >150 mm Hg
- Use of onabotulinum toxin A 4 months prior to trial or other injectable/stimulatory/magnetic method of headache control
- Use of opiates or barbiturates 4 days prior to trial
- Patients with competing intracerebral pathology (e.g. history of intracerebral hemorrhage, multiple sclerosis)
- NYHA Class III or IV congestive heart failure
Show all 11Show fewer
- History of myocardial infarction
- History of coronary bypass surgery or coronary stenting
- Pregnancy or breastfeeding
- Contraindication to undergoing brain MRI per standard clinical practice guidelines
- Vulnerable populations, including incarcerated inmates, dementia, and inability to provide consent
About this study
Official title: A Phase II Double-Blinded Placebo Controlled Individual Subject Stepped Wedge Clinical Trial Evaluating the Safety and Efficacy of Fremanezumab for Migraine in Adult CADASIL
Researchers are trying to find out more about the side effects of fremanezumab when treating patients with Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL) for migraine headaches.
Read the detailed description
Subjects will receive 4 to 20 weeks of placebo injections followed by 12 weeks of monthly administered fremanezumab by subcutaneous injection (225 mg split four weeks apart for 12 weeks). Following completion of the 12 weeks of therapy, the participant will return to placebo for the washout period. Both the patient and the investigator will be blinded to the timing of transition to and from placebo and the transition to and from fremanezumab.
- Being tested
- Fremanezumab, Placebo
- Medical subject headings
- CADASIL, Migraine Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FremanezumabDrug
Also called Ajovy
Subjects will receive a single dose total 225 mg (1 injection of 225 mg per 1.5 mL injection) followed by a single dose of 225 mg (1 1.5mL injection) at four week intervals. Both the subject and the investigator will be blinded to the timing of transition to and from placebo and the transition to and from fremanezumab.
PlaceboDrug
Placebo injections during the run in and washout serves as the control condition. Both the subject and the investigator will be blinded to the timing of transition to and from placebo and the transition to and from fremanezumab.
| Study group | Role | What they receive |
|---|---|---|
| Subjects with CADASIL treatment intervention | Active comparator | Subjects that have been diagnosed with both CADASIL and moderately to severely disabling migraine headaches will be treated with Fremanezumab injections. Receives: Drug: Fremanezumab; Drug: Placebo |
| Subjects with CADASIL placebo intervention | Placebo comparator | Subjects that have been diagnosed with both CADASIL and moderately to severely disabling migraine headaches will be treated with placebo injections. Receives: Drug: Fremanezumab; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in migraine-related disability
Measured using the The Migraine Disability Assessment (MIDAS) questionnaire which is a brief, self-administered questionnaire designed to quantify headache-related disability over a 3 month period using number of days
Measured: Baseline, 2 week intervals up to 48 weeks
Change in headache intensity
Measured using Headache Impact Test (HIT)-6 scores with the answers being never, rarely, sometimes, very often or always and equating to 6, 8, 10, 11, and 13 points respectively. Each answer is summated to equal the final score.
Measured: Baseline, 2 week intervals up to 48 weeks
Adverse events
Number of adverse events reported
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2020
- Study startDec 2021 (estimated)
- Primary completionDec 2024 (estimated)
- Study completionDec 2024 (estimated)
Why it stopped: Lack of funding to support the trial.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CadasilStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, James F. Meschia, Mayo Clinic
- Investigators
- James F Meschia, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04334408
- Sponsor's protocol ID
- 18-010786
- Status last verified
- Feb 2022
- First submitted
- 2 Apr 2020
- Last updated
- 28 Feb 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Double-Blinded Placebo Controlled Individual Subject Stepped Wedge Clinical Trial Evaluating the Safety and Efficacy of Fremanezumab for Migraine in Adult CADASIL
- ClinicalTrials.gov ID
- NCT04334408
- Acronym / sponsor ID
- 18-010786
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of funding to support the trial.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Dec 2021 (estimated)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 6 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Researchers are trying to find out more about the side effects of fremanezumab when treating patients with Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL) for migraine headaches.
Detailed description
Subjects will receive 4 to 20 weeks of placebo injections followed by 12 weeks of monthly administered fremanezumab by subcutaneous injection (225 mg split four weeks apart for 12 weeks). Following completion of the 12 weeks of therapy, the participant will return to placebo for the washout period. Both the patient and the investigator will be blinded to the timing of transition to and from placebo and the transition to and from fremanezumab.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Subjects with CADASIL treatment intervention | Active comparator | Drug: Fremanezumab, Drug: PlaceboSubjects that have been diagnosed with both CADASIL and moderately to severely disabling migraine headaches will be treated with Fremanezumab injections. |
| Subjects with CADASIL placebo intervention | Placebo comparator | Drug: Fremanezumab, Drug: PlaceboSubjects that have been diagnosed with both CADASIL and moderately to severely disabling migraine headaches will be treated with placebo injections. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age 18 to 70 years old
- English speaking
- Diagnosis of CADASIL confirmed by presence of a pathogenic NOTCH3 gene mutation
- Diagnosis of migraine for at least 12 months and diagnosis of chronic migraine for at least 28 days according to the International Classification of Headache Disorders
- MIDAS score of >10 points
Key exclusion criteria (11 listed)
- History of ischemic stroke within 4 weeks of screening assessment
- Screening blood pressure >150 mm Hg
- Use of onabotulinum toxin A 4 months prior to trial or other injectable/stimulatory/magnetic method of headache control
- Use of opiates or barbiturates 4 days prior to trial
- Patients with competing intracerebral pathology (e.g. history of intracerebral hemorrhage, multiple sclerosis)
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 |
|---|---|---|
| FremanezumabAlso: AjovySubjects will receive a single dose total 225 mg (1 injection of 225 mg per 1.5 mL injection) followed by a single dose of 225 mg (1 1.5mL injection) at four week intervals. Both the subject and the investigator will be blinded to the timing of transition to and from placebo and the transition to and from fremanezumab. | Drug | Subjects with CADASIL placebo intervention, Subjects with CADASIL treatment intervention |
| PlaceboPlacebo injections during the run in and washout serves as the control condition. Both the subject and the investigator will be blinded to the timing of transition to and from placebo and the transition to and from fremanezumab. | Drug | Subjects with CADASIL placebo intervention, Subjects with CADASIL treatment intervention |
Primary outcome
Change in migraine-related disability
Time frame: Baseline, 2 week intervals up to 48 weeks
Measured using the The Migraine Disability Assessment (MIDAS) questionnaire which is a brief, self-administered questionnaire designed to quantify headache-related disability over a 3 month period using number of days
Change in headache intensity
Time frame: Baseline, 2 week intervals up to 48 weeks
Measured using Headache Impact Test (HIT)-6 scores with the answers being never, rarely, sometimes, very often or always and equating to 6, 8, 10, 11, and 13 points respectively. Each answer is summated to equal the final score.
Adverse events
Time frame: 48 weeks
Number of adverse events reported
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Feb 2022.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Cadasil
RecruitingObservational study
Registry for CADASIL
Cerebral Autosomal Dominant Ateriopathy With Subcortical Infarcts and Leukoencephalopathy
RecruitingObservational study
Natural History Study of CADASIL
Cardiovascular Disease, Arterial Stiffness
RecruitingObservational study
The Erlanger Stroke Registry (ESPro) is the Only Ongoing Prospective Population-based Stroke Registry in Germany, Started in 1994. ESPro Includes All Hospitalized and Non-hospitalized Patients With Stroke in the Community of Erlangen (120.000 Inhabitants), With a Long-life Follow-up.
Intracerebral Haemorrhage (ICH), Cerebral Infarction
Active, not recruitingObservational study
Florida Cerebrovascular Disease Biorepository and Genomics Center
Cerebrovascular Disease, Ischemic Stroke
RecruitingObservational study
Effect of the Mediterranean Diet in Patients Affected by CADASIL and Cerebral Amyloid Angiopathy.
Cerebral Amyloid Angiopathy, CAA - Cerebral Amyloid Angiopathy
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.
James F Meschia principal investigator
Mayo Clinic
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fremanezumab in people with Cadasil and Migraine, run by Mayo Clinic.
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 6 inclusion and 11 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: withdrawn, as of the sponsor's last update on 28 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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 in migraine-related disability (time frame: Baseline, 2 week intervals up to 48 weeks). The study lists 3 primary outcomes in all. 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 28 Feb 2022.