Trial · NCT03936777
A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
NCT03936777, Phase 3, Completed, Zogenix, Inc.; results first posted 2025-11-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome. Trials, record NCT03936777. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03936777
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
- NCT number
- NCT03936777
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 412 participants, actual
- Lead sponsor
- Zogenix, Inc.
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome, Epileptic Encephalopathy
- Interventions
- ZX008 (Fenfluramine Hydrochloride)
- First posted
- 2019-05-03
- Last updated
- 2025-11-17
- Other registries' IDs
- eudract:2019-001331-31
- Results first posted
- 2025-11-17
Source ClinicalTrials.gov
Registered outcomes10
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) | From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days) | An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An AE can, therefore, be any unfavorable and unintended sign (including an abnormal, clinically significant laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment-emergent. The percentage of participants was rounded to one decimal place. |
| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The Clinical Global Impression-Improvement (CGI-I) scale is used to assess the severity of illness and global improvement in patients. Global function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The global CGI-I assessment was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported. |
| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The CGI-I scale is used to assess the severity of illness and global improvement in patients. Global function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The global CGI-I assessment was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported. |
| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The CGI-I scale is used to assess the severity of illness and global improvement in patients. Cognitive function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of cognition was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported. |
| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The CGI-I scale is used to assess the severity of illness and global improvement in patients. Behavior is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of behavior was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported. |
| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The CGI-I scale is used to assess the severity of illness and global improvement in patients. Motor abilities function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of motor abilities was conducted by the Parent/Caregiver. The number of participants in each of the 7 categories of the CGI-I for each visit is reported. |
| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The CGI-I scale is used to assess the severity of illness and global improvement in patients. Cognitive function is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of cognition was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported. |
| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The CGI-I scale is used to assess the severity of illness and global improvement in patients. Behavior is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of behavior was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported. |
| Secondary | Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The CGI-I scale is used to assess the severity of illness and global improvement in patients. Motor abilities is one of the domains assessed by the CGI-I scale. The CGI-I is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=Very much improved; 2=Much improved; 3=Minimally improved; 4=No change; 5=Minimally worse; 6=Much worse; 7=Very much worse, where higher score indicates worse outcome. The CGI-I assessment of motor abilities was conducted by the Investigator. The number of participants in each of the 7 categories of the CGI-I for each visit is reported. |
| Secondary | Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator | Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The change in seizure burden from the previous visit was assessed by the investigator using following categories: \<25%, ≥25%, ≥50%, ≥75%, 100% (i.e., seizure-free) improvement. Improvement in seizure burden at Baseline was based on comparison with the participant's last visit in the feeder studies (ZX008-1601, ZX008-1503, and ZX008-1602). Number of participants in each of the 5 categories is reported for each visit as compared to last visit. |
Source ClinicalTrials.gov
Outcomes10
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) | From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days) | Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension. | percentage of participants | NUMBER |
| SECONDARY | Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The modified Intent-to-Treat (mITT) analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the open-label extension (OLE) in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator | Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) | The mITT analysis set consisted of all participants who received at least 1 dose of ZX008 and had at least one valid efficacy assessment during the OLE in the study EP0215 (ZX008-1900). Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group433
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) | Any ZX008 Open Label Dose | 75.5 | percentage of participants | ||
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 6: Very much improved | 39 | Participants | |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 6: Much improved | 88 | Participants | |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 6: Minimally improved | 87 | Participants | |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 6: No change | 124 | Participants | |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 6: Minimally worse | 23 | Participants | |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 6: Much worse | 7 | Participants | |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 6: Very much worse | 3 | Participants | |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 12: Very much improved | 42 | Participants | |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | Any ZX008 Open Label Dose | Month 12: Much improved | 65 | Participants |
1 to 10 of 433Source ClinicalTrials.gov
See all 433 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Any ZX008 Open Label Dose | 412 | |
| Overall Study | COMPLETED | Any ZX008 Open Label Dose | 360 | |
| Overall Study | NOT COMPLETED | Any ZX008 Open Label Dose | 52 |
Source ClinicalTrials.gov
Adverse events76
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 | Diarrhoea | Gastrointestinal disorders | Any ZX008 Open Label Dose | 29 | 412 | 36 |
| other | Pyrexia | General disorders | Any ZX008 Open Label Dose | 53 | 412 | 86 |
| other | Corona virus infection | Infections and infestations | Any ZX008 Open Label Dose | 84 | 412 | 99 |
| other | Nasopharyngitis | Infections and infestations | Any ZX008 Open Label Dose | 49 | 412 | 69 |
| other | Upper respiratory tract infection | Infections and infestations | Any ZX008 Open Label Dose | 23 | 412 | 28 |
| other | Decreased appetite | Metabolism and nutrition disorders | Any ZX008 Open Label Dose | 25 | 412 | 30 |
| other | Seizure | Nervous system disorders | Any ZX008 Open Label Dose | 52 | 412 | 69 |
| serious | Thrombocytopenia | Blood and lymphatic system disorders | Any ZX008 Open Label Dose | 1 | 412 | 3 |
| serious | Cardiac arrest | Cardiac disorders | Any ZX008 Open Label Dose | 1 | 412 | 1 |
| serious | Non-compaction cardiomyopathy | Congenital, familial and genetic disorders | Any ZX008 Open Label Dose | 1 | 412 | 1 |
1 to 10 of 76Source ClinicalTrials.gov
See all 76 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 | 412 |
| Total, other adverse events | Any ZX008 Open Label Dose | 204 | 412 |
| Total, serious adverse events | Any ZX008 Open Label Dose | 67 | 412 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT03936777
GET https://conductscience.org/api/trials/v1/NCT03936777/results?page=2