Trial · NCT03790137
Treatment of Sunflower Syndrome With ZX008 (Fenfluramine Hydrochloride) in Children and Young Adults (Ages 4-25).
NCT03790137, Phase 3, Completed, Elizabeth Anne Thiele; results first posted 2023-02-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Treatment of Sunflower Syndrome With ZX008 (Fenfluramine Hydrochloride) in Children and Young Adults (Ages 4-25). Trials, record NCT03790137. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03790137
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treatment of Sunflower Syndrome With ZX008 (Fenfluramine Hydrochloride) in Children and Young Adults (Ages 4-25).
- NCT number
- NCT03790137
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 20 participants, actual
- Lead sponsor
- Elizabeth Anne Thiele
- Conditions
- Photosensitive Epilepsy
- Interventions
- Fenfluramine Hydrochloride
- First posted
- 2018-12-31
- Last updated
- 2024-11-06
- Results first posted
- 2023-02-08
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | % Change in Frequency of Handwaving Episodes | Month 3 | 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. |
| Primary | Change in Frequency of Generalized Tonic-clonic Seizures | Month 3 | 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. |
| Secondary | Change in Spike Frequency on EEG | Month 3 | 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. |
| Secondary | Number of Patients That Experienced a Photoparoxysmal Response on EEG | Month 3 | 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. |
| Secondary | Changes in Cognitive Functioning Determined by the Weschler Abbreviated Scale of Intelligence (WASI-II Subtests) | Month 3 (~84 days after the first dose of fenfluramine.) | 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 |
| Secondary | Changes in Cognitive Functioning Determined by the Weschler Intelligence Scale for Children (WISC-V) -Processing Speed Subtests. | Month 3 (approximately 84 days after the first dose of fenfluramine) | 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 |
| Secondary | Changes in Quality of Life Determined by the Quality of Life in Childhood Epilepsy Questionnaire(QOLCE-16) | Month 3 (approximately 84 days after first dose of fenfluramine) | 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. |
| Secondary | Changes in Executive Functioning Determined by The Behavioral Rating Inventory of Executive Function (BRIEF) Questionnaire. | Month 3 (approximately 84 days after the first dose of fenfluramine) | 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. |
| Secondary | Changes in Self-Concept Determined by The Beck Self Report Inventory. | Month 3 (approximately 84 days after first dose of fenfluramine) | 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 |
| Secondary | Changes in Anxiety Determined by the Beck Self Report Inventory | Month 3 | 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 |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | % Change in Frequency of Handwaving Episodes | Month 3 | One patient withdrew from the study 3 days after starting study medication. This patient developed a rash, which was not thought to be related to study medication. The patient chose not to challenge the drug at an allergists office, and instead withdrew from the study. The other 19 patients completed the core study, but only 17 provided analyzable seizure data. | % change in hand waving episodes | MEDIAN |
| PRIMARY | Change in Frequency of Generalized Tonic-clonic Seizures | Month 3 | 10 of the 20 patients reported having a generalized tonic-clonic seizure at some point in their lives. | tonic-clonic seizures/day | MEAN |
| SECONDARY | Change in Spike Frequency on EEG | Month 3 | The investigators read and interpreted pre-drug and post-drug EEGs for the first 9 patients that completed the core study. The remaining 10 patients did not undergo EEGs as part of this study, due to safety concerns during the COVID-19 pandemic. | spikes/hour | MEAN |
| SECONDARY | Number of Patients That Experienced a Photoparoxysmal Response on EEG | Month 3 | The investigators read and interpreted pre-drug and post-drug EEGs for the first 9 patients that completed the core study. The remaining 10 patients did not undergo EEGs as part of this study, due to safety concerns during the COVID-19 pandemic. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Changes in Cognitive Functioning Determined by the Weschler Abbreviated Scale of Intelligence (WASI-II Subtests) | Month 3 (~84 days after the first dose of fenfluramine.) | 9 of the first 10 patients completed the core study. The second 10 patients did not undergo neuropsychological testing as part of this study due to safety concerns during the COVID-19 pandemic. | score on a scale | MEAN |
| SECONDARY | Changes in Cognitive Functioning Determined by the Weschler Intelligence Scale for Children (WISC-V) -Processing Speed Subtests. | Month 3 (approximately 84 days after the first dose of fenfluramine) | 9 of the first 10 patients underwent neuropsychological testing as part of the core study. The remaining patients did not undergo this testing due to safety concerns during the COVID-19 pandemic. | score on a scale | MEAN |
| SECONDARY | Changes in Quality of Life Determined by the Quality of Life in Childhood Epilepsy Questionnaire(QOLCE-16) | Month 3 (approximately 84 days after first dose of fenfluramine) | 8 of the first 9 patients completed this testing as part of the core study. The remaining patients did not undergo this testing due to safety concerns during the COVID-19 pandemic. | score on a scale | MEAN |
| SECONDARY | Changes in Executive Functioning Determined by The Behavioral Rating Inventory of Executive Function (BRIEF) Questionnaire. | Month 3 (approximately 84 days after the first dose of fenfluramine) | 7 of the first 9 patients provided pre- and post-treatment responses to the BRIEF questionnaire. The remaining patients did not undergo this testing due to safety concerns during the COVID-19 pandemic. | T Score | MEAN |
| SECONDARY | Changes in Self-Concept Determined by The Beck Self Report Inventory. | Month 3 (approximately 84 days after first dose of fenfluramine) | 7 of the first 9 patients provided pre- and post-treatment responses to the Beck Self Report Inventory. The remaining patients did not undergo this testing due to safety concerns during the COVID-19 pandemic. | T-Score | MEAN |
| SECONDARY | Changes in Anxiety Determined by the Beck Self Report Inventory | Month 3 | T score | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| % Change in Frequency of Handwaving Episodes | Treatment Group | -66.2 | 95% Confidence Interval -78.7 to -31.3 | % change in hand waving episodes | |
| Change in Frequency of Generalized Tonic-clonic Seizures | Treatment Group | 0 | Full Range 0 to 0 | tonic-clonic seizures/day | |
| Change in Spike Frequency on EEG | Treatment Group | Pre-Treatment | 73.9 | Full Range 0 to 277.2 | spikes/hour |
| Change in Spike Frequency on EEG | Treatment Group | Post-Treatment | 50.4 | Full Range 0 to 187.2 | spikes/hour |
| Number of Patients That Experienced a Photoparoxysmal Response on EEG | Treatment Group | Baseline EEG (pre-treatment) | 5 | Participants | |
| Number of Patients That Experienced a Photoparoxysmal Response on EEG | Treatment Group | Month 3 EEG (post-treatment) | 3 | Participants | |
| Changes in Cognitive Functioning Determined by the Weschler Abbreviated Scale of Intelligence (WASI-II Subtests) | Treatment Group | Baseline | 97.89 | Standard Deviation 13.53 | score on a scale |
| Changes in Cognitive Functioning Determined by the Weschler Abbreviated Scale of Intelligence (WASI-II Subtests) | Treatment Group | Month 3 | 106.22 | Standard Deviation 11.64 | score on a scale |
| Changes in Cognitive Functioning Determined by the Weschler Intelligence Scale for Children (WISC-V) -Processing Speed Subtests. | Treatment Group | Baseline | 89.63 | Standard Deviation 9.12 | score on a scale |
| Changes in Cognitive Functioning Determined by the Weschler Intelligence Scale for Children (WISC-V) -Processing Speed Subtests. | Treatment Group | Month 3 | 92.14 | Standard Deviation 10.48 | score on a scale |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Treatment Group | 20 | |
| Overall Study | COMPLETED | Treatment Group | 19 | |
| Overall Study | NOT COMPLETED | Treatment Group | 1 |
Source ClinicalTrials.gov
Adverse events14
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Fatigue | General disorders | Treatment Group | 7 | 19 | |
| other | Reduced Appetite | General disorders | Treatment Group | 8 | 19 | |
| other | Nocturnal Enuresis | General disorders | Treatment Group | 2 | 19 | |
| other | Viral Illness | General disorders | Treatment Group | 4 | 19 | |
| other | Diarrhea | Gastrointestinal disorders | Treatment Group | 2 | 19 | |
| other | Shortness of Breath | General disorders | Treatment Group | 2 | 19 | |
| other | Chest Pain | General disorders | Treatment Group | 1 | 19 | |
| other | Seasonal Allergies | General disorders | Treatment Group | 2 | 19 | |
| other | Rhinorrhea | General disorders | Treatment Group | 1 | 19 | |
| other | irritability | General disorders | Treatment Group | 1 | 19 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment Group | 0 | 19 |
| Total, other adverse events | Treatment Group | 16 | 19 |
| Total, serious adverse events | Treatment Group | 0 | 19 |
Source ClinicalTrials.gov
Papers9
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests2
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as | Source |
|---|---|---|
| Matrix Reasoning | Secondary outcome | ConductCognition |
| Symbol Scan Task | Secondary outcome | ConductCognition |
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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