Trial · NCT02823145
An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome
NCT02823145, Phase 3, Completed, Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.; results first posted 2023-09-25
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome. Trials, record NCT02823145. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02823145
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome
- NCT number
- NCT02823145
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 375 participants, actual
- Lead sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
- Conditions
- Dravet Syndrome
- Interventions
- ZX008 (Fenfluramine Hydrochloride)
- First posted
- 2016-07-06
- Last updated
- 2025-07-14
- Other registries' IDs
- eudract:2016-002804-14
- Results first posted
- 2023-09-25
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period | From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42) | Treatment-emergent adverse events (TEAE) were defined as any AEs that based on start date information occurs after the first intake of study treatment. |
| Primary | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period | From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42) | A TEAE was defined as any AE that based on start date information occurs after the first intake of study treatment. Percentage of participants with TEAEs leading to withdrawal from IMP during OLE Treatment Period were reported. |
| Primary | Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period | From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42) | Serious Adverse event (SAE) was defined as any untoward medical occurrence that at any dose: • results in death, • is life-threatening threatening, • results in initial inpatient hospitalization or prolongation of hospitalization, •results in persistent or significant disability or incapacity, • results in a congenital anomaly/birth defect, • results in any medically significant event that did not meet any of the other 5 SAE criteria, but which was judged by a physician to potentially jeopardize the participant or require medical or surgical intervention to prevent one of the above outcomes listed as an SAE criterion. |
| Secondary | Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Day 1 to End of Study (EOS) Visit (Month 42) in the OLE Treatment Period | From Day 1 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core) | Baseline (Core) was defined as Baseline prior to double-blind treatment in the core studies (ZX008-1501/ZX008-1502, and ZX008-1504 Cohort 2). Participants in 1504- Cohort 1 and de novo participants (who entered 1503 without having been in any of the core studies) did not have a Baseline (Core), and were not included in the analysis of this outcome measure. The total number of convulsive seizures from Day 1 to EOS was divided by the total number of days from Day 1 to EOS with nonmissing diary data and the result was then multiplied by 28 to get a 28-day convulsive seizure frequency (CSF). The change from Baseline for any individual participant was calculated by subtracting the Baseline (Core) from the post-baseline value. Monthly (28 day) CSF was based on electronic diary data obtained for each participant. |
| Secondary | Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period | From Month 2 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core) | Baseline (Core) was defined as Baseline prior to double-blind treatment in the core studies. Participants in 1504-Cohort 1 and de novo participants did not have a Baseline (Core), and were not included in the analysis of this outcome measure. The total number of convulsive seizures from Month 2 to EOS was divided by the total number of days from Month 2 to EOS with nonmissing diary data and the result was then multiplied by 28 to get a 28-day CSF. The change from Baseline for any individual participant was calculated by subtracting the Baseline (Core) from the post-baseline value. Monthly (28 day) CSF was based on electronic diary data obtained for each participant. |
| Secondary | Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36) | At Month 1, Month 2, Month 3, Month 4-6, Month 7-9, Month 10-12, Month 13-15, Month 16-18, Month 19-21, Month 22-24, Month 25-27, Month 28-30, Month 31-33, and Month 34-36 | Monthly (28 day) CSF was based on electronic diary data obtained for each participant. The total number of convulsive seizures in the ith interval (CSF in OLE, where, i=1, 2, 3, … , 14 ) was divided by the total number of days in the ith interval with nonmissing diary data and the result was then multiplied by 28 to get a 28-day CSF of OLE. |
| Secondary | Convulsive Seizure Frequency (CSF) by Mean Daily Dose During the Overall OLE Treatment Period | From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42) | Convulsive seizure frequency over time, reported as per 28 days was analyzed by the actual dose administered. Participants were grouped into low (0.2 to \<0.4 mg/kg), medium (0.4 to \<0.6 mg/kg), and high dose (\>0.6 mg/kg) groups depending on their mean daily doses of ZX008 during the OLE Treatment period. For each participant, the seizure frequency per 28 days was calculated as the number of seizures recorded during the period, divided by the number of days in the period and multiplied by 28. The convulsive seizure frequency was calculated from all available data collected. |
| Secondary | Percentage of Participants With Changes in Antiepileptic Drug (AED) Medications During First 6 Months of OLE Treatment Period | At Month 1, 2, 3, 4 , 5, and 6 of OLE Treatment Period | Participants in the study were required to be on stable background therapy for the first 6 months of treatment, after which background AEDs could be reduced or withdrawn so long as one background AED remained. The percentage of participants who had changes in dose or type of concomitant AED medications during the first, second, third, fourth, fifth, and sixth months were analyzed and reported. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period | From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42) | Safety (SAF) population included all enrolled participants who received at least one dose of ZX008 during the OLE. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period | From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42) | Safety (SAF) population included all enrolled participants who received at least one dose of ZX008 during the OLE. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period | From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42) | Safety (SAF) population included all enrolled participants who received at least one dose of ZX008 during the OLE. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Day 1 to End of Study (EOS) Visit (Month 42) in the OLE Treatment Period | From Day 1 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core) | Modified Intent-to-Treat (mITT) population included all enrolled participants who received at least one dose of ZX008 and had at least 1 month of valid seizure data during the OLE. | seizure frequency per 28 days | MEDIAN |
| SECONDARY | Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period | From Month 2 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core) | mITT population included all enrolled participants who received at least one dose of ZX008 and had at least 1 month of valid seizure data during the OLE. Here, number of participants analyzed included those participants who were evaluable for the assessment. | seizure frequency per 28 days | MEDIAN |
| SECONDARY | Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36) | At Month 1, Month 2, Month 3, Month 4-6, Month 7-9, Month 10-12, Month 13-15, Month 16-18, Month 19-21, Month 22-24, Month 25-27, Month 28-30, Month 31-33, and Month 34-36 | mITT population included all enrolled participants who received at least one dose of ZX008 and had at least 1 month of valid seizure data during the OLE. Here, number analyzed signifies participants who were evaluable at specified time points. | seizure frequency per 28 days | MEDIAN |
| SECONDARY | Convulsive Seizure Frequency (CSF) by Mean Daily Dose During the Overall OLE Treatment Period | From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42) | mITT population included all enrolled participants who received at least one dose of ZX008 and had at least 1 month of valid seizure data during the OLE. Here, number analyzed represents the number of participants categorized by mean daily dose. | seizure frequency per 28 days | MEDIAN |
| SECONDARY | Percentage of Participants With Changes in Antiepileptic Drug (AED) Medications During First 6 Months of OLE Treatment Period | At Month 1, 2, 3, 4 , 5, and 6 of OLE Treatment Period | mITT population included all enrolled participants who received at least one dose of ZX008 and had at least 1 month of valid seizure data during the OLE. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period | Any ZX008 Open Label Dose | 98.1 | percentage of participants | ||
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period | Any ZX008 Open Label Dose | 3.5 | percentage of participants | ||
| Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period | Any ZX008 Open Label Dose | 26.5 | percentage of participants | ||
| Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Day 1 to End of Study (EOS) Visit (Month 42) in the OLE Treatment Period | Any ZX008 Open Label Dose | -6.67 | Full Range -1757.8 to 751.3 | seizure frequency per 28 days | |
| Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period | Any ZX008 Open Label Dose | -7.04 | Full Range -1986.9 to 816.4 | seizure frequency per 28 days | |
| Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36) | Any ZX008 Open Label Dose | Month 1 | 6.53 | Full Range 0.0 to 4876.9 | seizure frequency per 28 days |
| Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36) | Any ZX008 Open Label Dose | Month 2 | 4.67 | Full Range 0.0 to 3392.7 | seizure frequency per 28 days |
| Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36) | Any ZX008 Open Label Dose | Month 3 | 4.67 | Full Range 0.0 to 2593.7 | seizure frequency per 28 days |
| Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36) | Any ZX008 Open Label Dose | Month 4-6 | 4.36 | Full Range 0.0 to 1725.5 | seizure frequency per 28 days |
| Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36) | Any ZX008 Open Label Dose | Month 7-9 | 3.82 | Full Range 0.0 to 310.8 | seizure frequency per 28 days |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Enrollment | STARTED | Not Treated | 1 | |
| Enrollment | STARTED | Any ZX008 Open Label Dose | 374 | |
| Enrollment | COMPLETED | Not Treated | 0 | |
| Enrollment | COMPLETED | Any ZX008 Open Label Dose | 374 | |
| Enrollment | NOT COMPLETED | Not Treated | 1 | |
| Enrollment | NOT COMPLETED | Any ZX008 Open Label Dose | 0 | |
| Treatment | STARTED | Not Treated | 0 | |
| Treatment | STARTED | Any ZX008 Open Label Dose | 374 | |
| Treatment | COMPLETED | Not Treated | 0 | |
| Treatment | COMPLETED | Any ZX008 Open Label Dose | 49 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events119
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 | Constipation | Gastrointestinal disorders | Any ZX008 Open Label Dose | 23 | 374 | 34 |
| other | Diarrhoea | Gastrointestinal disorders | Any ZX008 Open Label Dose | 73 | 374 | 102 |
| other | Vomiting | Gastrointestinal disorders | Any ZX008 Open Label Dose | 39 | 374 | 50 |
| other | Fatigue | General disorders | Any ZX008 Open Label Dose | 34 | 374 | 49 |
| other | Pyrexia | General disorders | Any ZX008 Open Label Dose | 112 | 374 | 229 |
| other | Ear infection | Infections and infestations | Any ZX008 Open Label Dose | 39 | 374 | 56 |
| other | Gastroenteritis | Infections and infestations | Any ZX008 Open Label Dose | 32 | 374 | 34 |
| other | Gastroenteritis viral | Infections and infestations | Any ZX008 Open Label Dose | 20 | 374 | 21 |
| other | Influenza | Infections and infestations | Any ZX008 Open Label Dose | 45 | 374 | 50 |
| other | Nasopharyngitis | Infections and infestations | Any ZX008 Open Label Dose | 104 | 374 | 224 |
1 to 10 of 119Source ClinicalTrials.gov
See all 119 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Any ZX008 Open Label Dose | 3 | 374 |
| Total, other adverse events | Any ZX008 Open Label Dose | 342 | 374 |
| Total, serious adverse events | Any ZX008 Open Label Dose | 99 | 374 |
Source ClinicalTrials.gov
Papers2
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.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02823145
GET https://conductscience.org/api/trials/v1/NCT02823145/results?page=2