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Photosensitive EpilepsyPhase 3NCT03790137

Treatment of Sunflower Syndrome With ZX008 (Fenfluramine Hydrochloride) in Children and Young Adults (Ages 4-25).

A Phase 3 study testing Fenfluramine Hydrochloride in people with Photosensitive Epilepsy, run by Elizabeth Anne Thiele at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

Sunflower Syndrome (also referred to as Self-induced Photosensitive Epilepsy) is a rare epileptic disorder characterized by a distinctive seizure that manifests itself in a highly stereotyped physical behavior. Read more about this study

Who is it for?
Ages 4 to 25 years
Conditions the study is for
Photosensitive Epilepsy
What is being tested?
Fenfluramine Hydrochloride
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteUnited States
Study dates
31 May 2019to 19 Jul 2024
Phase
Phase 3
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 4 to 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Subject is male or non-pregnant, non-lactating female, age 4 to 25 years, inclusive as of the day of the screening visit. Female subjects of childbearing potential must have a negative serum pregnancy test at screening. Subjects of childbearing or child-fathering potential must be willing to use medically acceptable forms of birth control, which includes abstinence, while in this study and for 90 days after the last dose of study drug.
  • Subjects must have a diagnosis of Sunflower Syndrome, where seizures are not completely controlled by their current treatment plan.
  • Subjects must experience seizures including absence seizures and/or generalized tonic-clonic seizures which involve seeking out a light source, staring at the light source, and waving one hand in front of their eye(s). Subject's must experience an average of 6 hand waving associated with absence seizures and/or generalized tonic-clonic seizures per week.
  • Evidence of EEG in the medical history that shows generalized spike and wave discharges between seizures and a strong photoparoxysmal response during photic stimulation. Acceptable evidence includes a copy of the EEG trace, EEG report, or physician note that appropriately describes the EEG findings.
  • All medications or interventions for epilepsy must be stable for at least 4 weeks prior to screening and are expected to remain stable until Month 3.
  • Subject and/or parent/guardian has been informed of the nature of the study and informed consent has been obtained from the subject and/or legally responsible parent/guardian.
  • Subject has provided assent in accordance with Institutional Review Board (IRB)/Ethics Committee requirements, if capable.
  • Subjects parent/caregiver is willing and able to be compliant with diary completion, visit schedule, and study drug accountability.

Who cannot join

  • Subject has a known hypersensitivity to fenfluramine hydrochloride or any other ingredients in the investigational drug,
  • Subject's etiology of seizures is a degenerative neurological disease.
  • Subject is pregnant.
  • Subject is not willing to comply with a method of birth control acceptable to the PI during the study and for 90 days following completion of the study.
  • Subject is breastfeeding.
  • Subject has a history of drug or alcohol abuse.
Show all 24Show fewer
  • Subject has pulmonary arterial hypertension.
  • Subject has current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke, or clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, patent ductus arteriosis, and patent foramen ovale with reversal of shunt. (note: bicuspid valve is not considered exclusionary, but may be associated with the following diseases, which are exclusionary: coarctation of the aorta, Turner syndrome, supravalvular aortic stenosis, subvalvular aortic stenosis, patent ductus arteriosus, Sinus of Valsalva aneurysm, ventricular septal defect, Shone's complex, ascending aortic aneurysm, Loeys-Dietz syndrome, ACTA2 mutation familial thoracic aortic aneurysm syndrome, and MAT2A mutation familial thoracic aortic aneurysm syndrome).
  • Subject has current or recent history of anorexia nervosa, bulimia, or depression within the prior year that required medical treatment or psychological treatment for a duration greater than 1 month.
  • Subject has a current or past history of glaucoma.
  • Subject has had an anoxic episode requiring resuscitation within 6 months of the screening visit.
  • Subject has moderate or severe hepatic impairment. Asymptomatic subjects with mild hepatic impairment (elevated liver enzymes < 3x upper limit of normal (ULN) and/or elevated bilirubin <2x ULN) may be entered into the study after review and approval by the Medical Monitor in conjunction with the sponsor, in consideration of comorbidities and concomitant medications.
  • Subject has severe renal impairment (estimated glomerular filtration rate <30mL/min/1.73m2)
  • Subject is receiving concomitant therapy with: centrally-acting anorectic agents; monoamine-oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; other centrally-acting noradrenergic agonists, including atomoxetine; or cyproheptadine. (Note: Short-term medication requirements for prohibited medications will be handled on a per case basis by the medical monitor.)
  • Subject has positive result (as defined in the laboratory manual) on urine tetrahydrocannabinol (THC) Panel or whole blood cannabidiol (CBD) at the screening visit.
  • Subject has been taking felbamate for less than 1 year prior to screening and/or does not have stable liver function and hematology laboratory tests, and/or the dose has not been stable for at least 60 days prior to the screening visit.
  • Subject is known to be human immunodeficiency virus (HIV) positive.
  • Subject is known to have active viral hepatitis (B or C).
  • Subject is currently receiving an investigational medicinal product.
  • Subject has participated in another clinical trial within the past 30 days (calculated from that study's last scheduled visit). Participation in non-treatment trials will be reviewed by the medical monitor.
  • Subject is at imminent risk of self-harm or harm to others, in the investigator's opinion.
  • Subject is unwilling or unable to comply with scheduled visits, drug administration plan, laboratory tests, other study procedures, and study restrictions.
  • Subject is institutionalized in a general nursing home (i.e., in a facility that does not provide skilled epilepsy care).-Subject does not have a reliable caregiver who can provide seizure diary information throughout the study.
  • Subject has a clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to the Screening Visit, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

Other trials in United States

About this study

Official title: An Open-Label Treatment of ZX008 (Fenfluramine Hydrochloride) in Pediatric and Adult Epilepsy Patients (Ages 4-25) With Sunflower Syndrome.

Sunflower Syndrome (also referred to as Self-induced Photosensitive Epilepsy) is a rare epileptic disorder characterized by a distinctive seizure that manifests itself in a highly stereotyped physical behavior. Seizure types associated with Sunflower Syndrome include absence seizures and generalized tonic-clonic seizures. Individuals with Sunflower Syndrome obsessively seek out a light source, stare at the light source, and wave one hand in front of their eye(s). Electroencephalogram (EEG) features include generalized spike and wave discharges interictally, and typically strong photoparoxysmal response during photic stimulation.

Currently, Sunflower syndrome is poorly characterized in medical literature and is often misunderstood at the clinical level. The name self-induced photosensitive epilepsy may be a misnomer as research concerning the neurochemical and neuropsychological pathways cannot conclusively determine that it is self-induced (conscious behavior) as the name implies. Although some reports have concluded that the hand waiving induces the seizure, these findings are not consistent throughout scientific literature. In fact, an EEG report found that the seizures can begin simultaneously with the hand waving. This suggests that the hand waving may in fact be part of the seizure, not the cause.

Read more

There are no treatments specifically approved for the treatment of Sunflower Syndrome in the United States. Broad spectrum anticonvulsant medications, including sodium valproate, lamotrigine, levetiracetam, and clobazam, have not shown full efficacy in seizure prevention in patients with Sunflower Syndrome. Accordingly, there remains a significant unmet need for an approved treatment for children and adults with Sunflower Syndrome.

Because this epilepsy typically does not respond to anticonvulsant medications, and because Aicardi described the successful treatment with fenfluramine of at least one child with this syndrome, the investigators of this study will investigate if fenfluramine is an effective, safe and well tolerated treatment for Sunflower Syndrome.

The primary objective of this study is to determine the efficacy of ZX008 on seizure frequency in children and young adults with Sunflower Syndrome. The goal of treatment is to provide a 30 percent or greater reduction of generalized tonic-clonic seizures and/or hand waving associated with absence seizures.

Secondary objectives of the study include evaluation of the effect of ZX008 (fenfluramine hydrochloride) on EEG patterns and quality of life. Patients with Sunflower Syndrome often experience low self-esteem, bullying due to the unusual motor movements associated with their seizures, school performance issues, anxiety, and depression.

The study population will include pediatric and young adult patients seen by Elizabeth A. Thiele, M.D., Ph.D. at MGH's Pediatric Epilepsy Clinic who were identified as candidates. The Principal Investigator (PI) will follow up to 20 patients with Sunflower Syndrome who will be taking ZX008.

Being tested
Fenfluramine Hydrochloride
Keywords
Sunflower Syndrome, Epilepsy
Medical subject headings
Epilepsy, Reflex, Epilepsy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Fenfluramine HydrochlorideDrug

    Also called ZX008

    Fenfluramine Hydrochloride will be supplied to the treatment group as an oral solution in a concentration of 2.5 mg/mL. Subjects will receive their daily dose of Fenfluramine Hydrochloride in two doses (one in the morning and one in the evening). After a four week baseline, subjects that meet enrollment criteria will enter a titration period. The starting dose will be 0.2 mg/kg/day for the first 14 days. The dose will be increased every 2 weeks as tolerated by 0.2 mg/kg/day, to a maximum dose of 0.7 mg/kg/day, or a total maximum dose of 26 mg/day. The subject will remain on a dose of 0.7 mg/kg/day, 26 mg/day, or maximum tolerated daily dose through the end of the core study period.

Study groups in NCT03790137
Study groupRoleWhat they receive
Treatment groupExperimentalThe treatment group will include approximately 20 pediatric and young adult patients (ages 4-25 years) seen by Elizabeth A. Thiele, M.D., Ph.D. at MGH's Pediatric Epilepsy Clinic. Subjects will be treated on an outpatient basis and will not require hospital admission. The treatment group will receive the investigational new drug, Fenfluramine Hydrochloride for approximately 4 months. Patients that benefit from this treatment will remain on medication through an extension phase of the study.
Receives: Drug: Fenfluramine Hydrochloride

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. % Change in Frequency of Handwaving Episodes

    The number of handwaving episodes (seizures) per day were recorded on a seizure diary by either the subject or the parent of the subject. One handwaving episode was defined as the period starting from when the hand first came up toward the face, until there was a brief pause in waving. Subjects and their caregivers were instructed to record the total number of handwaving episodes each day during a one month baseline, and during the 3 month treatment period.

    The baseline seizure frequency was calculated as an average of the reported handwaving episodes per day during the baseline period. This number was compared to the average episodes per day for the final month of the study (month 3 of treatment with fenfluramine) for each patient. The median percent change in frequency of handwaving episodes across all patients is reported below.

    Measured: Month 3

  2. Change in Frequency of Generalized Tonic-clonic Seizures

    Daily seizure logs will be maintained by either the subject or the parent of the subject and used to calculate seizure frequency. Frequency of Generalized Tonic-clonic seizures was recorded as an average number of seizures/day. The average number of tonic-clonic seizures per day during the baseline period was compared to the average number of tonic-clonic seizures per day during the final month (month 3) for each patient that had experienced at least one tonic-clonic seizure in their lifetime.

    Measured: Month 3

Secondary outcomes (11)

  1. Change in Spike Frequency on EEG

    The investigators will read and interpret pre-drug (baseline) and post-drug EEGs (month 3 of core study) for the first 9 patients. The average number of spikes/hour across these patients will be reported for both pre- and post-treatment EEGs.

    Measured: Month 3

  2. Number of Patients That Experienced a Photoparoxysmal Response on EEG

    The investigators will read and interpret pre- (baseline) and post-treatment (month 3) EEGs. The number of patients that experienced a photoparoxysmal response during the EEGs will be reported.

    Measured: Month 3

  3. Changes in Cognitive Functioning Determined by the Weschler Abbreviated Scale of Intelligence (WASI-II Subtests)

    The Weschler Abbreviated Scale of Intelligence (WASI-II subtests) will be administered to the first 10 patients at baseline and 84 days after the start of the first dose of study drug. Results of these cognitive tests will be compared between baseline and 84 days post initial dose.

    The WASI-II measures cognitive functioning through 2 subtests, vocabulary and matrix reasoning. The raw scores on these subtests are combined to give a composite full-scale IQ (FSIQ) that is scaled to an average of 100 and standard deviation of 15.

    Maximum raw scores for the subtests are as follows (higher values are considered better and minimum score on each is 0).

    Vocabulary:

    ages 7-11: 47 ages 12-14: 53 ages 15-25: 59

    Matrix Reasoning:

    ages 6-8: 24 ages 9-25: 30

    Measured: Month 3 (~84 days after the first dose of fenfluramine.)

  4. Changes in Cognitive Functioning Determined by the Weschler Intelligence Scale for Children (WISC-V) -Processing Speed Subtests.

    The Weschler Intelligence Scale for Children (WISC-V) -Processing Speed subtests will be administered at baseline and 84 days after the start of the first dose of study drug (Month 3; visit 5). Results of these cognitive tests will be compared between baseline and 84 days post initial dose.

    Raw scores for the coding and symbol search subtests will be used to evaluate processing speed. The maximum raw scores are as follows (the minimum score is 0 and higher scores are considered better).

    Coding:

    ages 6-7: 65 ages 8-16: 119

    Symbol search:

    ages 6-7: 45 ages 8-16: 60

    Measured: Month 3 (approximately 84 days after the first dose of fenfluramine)

  5. Changes in Quality of Life Determined by the Quality of Life in Childhood Epilepsy Questionnaire(QOLCE-16)

    The Quality of Life in Childhood Epilepsy Questionnaire will be administered at baseline, and 84 days after the first dose (Month 3; visit 5). This questionnaire assesses mood, social relationships, and behaviors. Results will be compared.

    The QOLCE assesses quality of life across 4 domains of functioning: cognitive, emotional, social and physical. Scores range from 0-100. Composite scores are calculated as a mean across all measures.

    Measured: Month 3 (approximately 84 days after first dose of fenfluramine)

  6. Changes in Executive Functioning Determined by The Behavioral Rating Inventory of Executive Function (BRIEF) Questionnaire.

    The Behavioral Rating Inventory of Executive Function (BRIEF) questionnaire will be administered at baseline, and 84 days after the first dose of fenfluramine (Month 3; Visit 5). This questionnaire assesses executive function across 8 clinical scales:

    1. Inhibit
    2. Shift
    3. Emotional Control
    4. Initiate
    5. Working Memory
    6. Plan/Organize
    7. Organization of Materials
    8. Monitor

    Item level scores are rated on a 3 point Likert Scale from 1-3:

    1. Never
    2. Sometimes
    3. Often

    The Global Executive Composite (GEC) score is the total score of all 8 clinical subscales. The item scores are summed for each scale and raw scores are converted to Tscores. The mean of the GEC score will be reported.

    T scores from 60 to 64 are considered mildly elevated. T scores from 65 to 69 are considered potentially clinically elevated. T scores at or above 70 are considered clinically elevated.

    Measured: Month 3 (approximately 84 days after the first dose of fenfluramine)

  7. Changes in Self-Concept Determined by The Beck Self Report Inventory.

    The Beck Self Report Inventory will be administered at baseline, and 84 days after the first dose (Month 3; Visit 5). This questionnaire assesses self-concept, depression, anxiety, anger and disruptive behavior through a 100-item self-report.

    The raw scores for subscales are calculated in the following manner:

    • Add scores from Items 1-20 for the total Beck Self Concept Inventory for Youth (BSCI-Y) score.
    • Add scores from Items 21-40 for the total Beck Anxiety Inventory for Youth (BAI-Y) score.
    • Add scores from Items 41-60 for the total Beck Depression Inventory for Youth (BDI-Y) score.
    • Add scores from Items 61-80 for the total Beck Anger Inventory for Youth (BANI-Y) score.
    • Add scores from Items 81-100 for the total Beck Disruptive Inventory for Youth (BDBI-Y) score.

    Raw scores will be translated into T-scores.

    T-scores for BYI are as follows:

    • 55 or less = average
    • 55-59 = mildly elevated
    • 60-69 = moderately elevated
    • 70+ = extremely elevated

    Measured: Month 3 (approximately 84 days after first dose of fenfluramine)

  8. Changes in Anxiety Determined by the Beck Self Report Inventory

    The Beck Self Report Inventory will be administered at baseline, and 84 days after the first dose (Month 3; Visit 5). This questionnaire assesses self-concept, depression, anxiety, anger and disruptive behavior through a 100-item self-report.

    The raw scores for subscales are calculated in the following manner:

    • Add scores from Items 1-20 for the total Beck Self Concept Inventory for Youth (BSCI-Y) score.
    • Add scores from Items 21-40 for the total Beck Anxiety Inventory for Youth (BAI-Y) score.
    • Add scores from Items 41-60 for the total Beck Depression Inventory for Youth (BDI-Y) score.
    • Add scores from Items 61-80 for the total Beck Anger Inventory for Youth (BANI-Y) score.
    • Add scores from Items 81-100 for the total Beck Disruptive Inventory for Youth (BDBI-Y) score.

    Raw scores will be translated into T-scores.

    T-scores for BYI are as follows:

    • 55 or less = average
    • 55-59 = mildly elevated
    • 60-69 = moderately elevated
    • 70+ = extremely elevated

    Measured: Month 3

  9. Changes in Depression Determined by the Beck Self Report Inventory

    The Beck Self Report Inventory will be administered at baseline, and 84 days after the first dose (Month 3; Visit 5). This questionnaire assesses self-concept, depression, anxiety, anger and disruptive behavior through a 100-item self-report.

    The raw scores for subscales are calculated in the following manner:

    • Add scores from Items 1-20 for the total Beck Self Concept Inventory for Youth (BSCI-Y) score.
    • Add scores from Items 21-40 for the total Beck Anxiety Inventory for Youth (BAI-Y) score.
    • Add scores from Items 41-60 for the total Beck Depression Inventory for Youth (BDI-Y) score.
    • Add scores from Items 61-80 for the total Beck Anger Inventory for Youth (BANI-Y) score.
    • Add scores from Items 81-100 for the total Beck Disruptive Inventory for Youth (BDBI-Y) score.

    Raw scores will be translated into T-scores.

    T-scores for BYI are as follows:

    • 55 or less = average
    • 55-59 = mildly elevated
    • 60-69 = moderately elevated
    • 70+ = extremely elevated

    Measured: Month 3

  10. Changes in Anger Determined by the Beck Self Report Inventory

    The Beck Self Report Inventory will be administered at baseline, and 84 days after the first dose (Month 3; Visit 5). This questionnaire assesses self-concept, depression, anxiety, anger and disruptive behavior through a 100-item self-report.

    The raw scores for subscales are calculated in the following manner:

    • Add scores from Items 1-20 for the total Beck Self Concept Inventory for Youth (BSCI-Y) score.
    • Add scores from Items 21-40 for the total Beck Anxiety Inventory for Youth (BAI-Y) score.
    • Add scores from Items 41-60 for the total Beck Depression Inventory for Youth (BDI-Y) score.
    • Add scores from Items 61-80 for the total Beck Anger Inventory for Youth (BANI-Y) score.
    • Add scores from Items 81-100 for the total Beck Disruptive Inventory for Youth (BDBI-Y) score.

    Raw scores will be translated into T-scores.

    T-scores for BYI are as follows:

    • 55 or less = average
    • 55-59 = mildly elevated
    • 60-69 = moderately elevated
    • 70+ = extremely elevated

    Measured: Month 3

  11. Changes in Disruptive Behavior Determined by the Beck Self Report Inventory

    The Beck Self Report Inventory will be administered at baseline, and 84 days after the first dose (Month 3; Visit 5). This questionnaire assesses self-concept, depression, anxiety, anger and disruptive behavior through a 100-item self-report.

    The raw scores for subscales are calculated in the following manner:

    • Add scores from Items 1-20 for the total Beck Self Concept Inventory for Youth (BSCI-Y) score.
    • Add scores from Items 21-40 for the total Beck Anxiety Inventory for Youth (BAI-Y) score.
    • Add scores from Items 41-60 for the total Beck Depression Inventory for Youth (BDI-Y) score.
    • Add scores from Items 61-80 for the total Beck Anger Inventory for Youth (BANI-Y) score.
    • Add scores from Items 81-100 for the total Beck Disruptive Inventory for Youth (BDBI-Y) score.

    Raw scores will be translated into T-scores.

    T-scores for BYI are as follows:

    • 55 or less = average
    • 55-59 = mildly elevated
    • 60-69 = moderately elevated
    • 70+ = extremely elevated

    Measured: Month 3

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Dec 2018
  2. Study start31 May 2019
  3. Primary completion25 Nov 2021
  4. Study completion19 Jul 2024

Resources

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 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03790137
Sponsor's protocol ID
12112018
Status last verified
Oct 2024
First submitted
12 Dec 2018
Last updated
6 Nov 2024
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: Individual participant data (IPD) will not be made available to other researchers.

Publications

  • Aicardi J, Gastaut H. Treatment of self-induced photosensitive epilepsy with fenfluramine. N Engl J Med. 1985 Nov 28;313(22):1419. doi: 10.1056/NEJM198511283132218. No abstract available.

    PubMed 3932858

  • Ames FR. "Self-induction" in photosensitive epilepsy. Brain. 1971;94(4):781-98. doi: 10.1093/brain/94.4.781. No abstract available.

    PubMed 5132972

  • Ames FR, Saffer D. The sunflower syndrome. A new look at "self-induced" photosensitive epilepsy. J Neurol Sci. 1983 Apr;59(1):1-11. doi: 10.1016/0022-510x(83)90076-x.

    PubMed 6854340

  • Belcastro V, Striano P. Self-induction seizures in sunflower epilepsy: a video-EEG report. Epileptic Disord. 2014 Mar;16(1):93-5. doi: 10.1684/epd.2014.0630.

    PubMed 24556582

  • Boel M, Casaer P. Add-on therapy of fenfluramine in intractable self-induced epilepsy. Neuropediatrics. 1996 Aug;27(4):171-3. doi: 10.1055/s-2007-973781.

    PubMed 8892363

  • Chieffo D, Battaglia D, Lettori D, Del Re M, Brogna C, Dravet C, Mercuri E, Guzzetta F. Neuropsychological development in children with Dravet syndrome. Epilepsy Res. 2011 Jun;95(1-2):86-93. doi: 10.1016/j.eplepsyres.2011.03.005. Epub 2011 Apr 6.

    PubMed 21474289

  • Fuller RW, Snoddy HD, Robertson DW. Mechanisms of effects of d-fenfluramine on brain serotonin metabolism in rats: uptake inhibition versus release. Pharmacol Biochem Behav. 1988 Jul;30(3):715-21. doi: 10.1016/0091-3057(88)90089-5.

    PubMed 2463643

  • LIVINGSTON S, TORRES IC. PHOTIC EPILEPSY: REPORT OF AN UNUSUAL CASE AND REVIEW OF THE LITERATURE. Clin Pediatr (Phila). 1964 May;3:304-7. doi: 10.1177/000992286400300511. No abstract available.

    PubMed 14124883

  • Patel S, Geenen KR, Dowless D, Bruno PL, Thiele EA. Follow-up to low-dose fenfluramine for Sunflower syndrome: A non-randomized controlled trial. Dev Med Child Neurol. 2023 Jul;65(7):961-967. doi: 10.1111/dmcn.15492. Epub 2022 Dec 23.

    PubMed 36562419

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Fenfluramine Hydrochloride in people with Photosensitive Epilepsy, run by Elizabeth Anne Thiele 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 8 inclusion and 24 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: finished, as of the sponsor's last update on 6 Nov 2024. 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 8 Feb 2023). 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 in Frequency of Handwaving Episodes (time frame: Month 3). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03790137, which also lists 9 linked publications.

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 6 Nov 2024.

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