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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-001331-31 | EudraCT Number | ||
| U1111-1309-4338 | Other Identifier | World Health Organization (WHO) | |
| 2024-515680-61 | Registry Identifier | EU Clinical Trials |
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| Name | Class |
|---|---|
| Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. | INDUSTRY |
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This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
This is an international, multicenter, open-label, long-term safety study of ZX008 in patients with epileptic encephalopathy, including Dravet syndrome or Lennox-Gastaut syndrome. Subjects eligible for participation are those with Dravet syndrome who are currently enrolled in Study ZX008-1503, or those with Lennox-Gastaut syndrome who have successfully completed Study ZX008-1601-Part 2, and are candidates for continued treatment with ZX008 for an extended period of time, or those with Dravet syndrome, Lennox-Gastaut syndrome, or another epileptic encephalopathy who have completed participation in another Zogenix-sponsored study and have been invited to participate in this study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ZX008 (Fenfluramine Hydrochloride) | Experimental | ZX008 is supplied as an open-label oral solution.Doses will include up to 0.8 mg/kg/day divided into 2 daily doses, up to a maximum of 30 mg/day (subjects taking concomitant STP will receive up to 0.5 mg/kg/day, up to a maximum of 20 mg/day) in a concentration of 2.5 mg/mL. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ZX008 (Fenfluramine Hydrochloride) | Drug | Fenfluramine hydrochloride provided in a concentration of 2.5 mg/mL. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 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. | From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days) |
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| UCB Cares | 001 844 599 2273 | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ep0215 107 | Tucson | Arizona | 85718 | United States | ||
| Ep0215 144 |
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
The Participant Flow refers to the Safety analysis set.
The study started to enroll participants in April 2019 and concluded in May 2025.
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| ID | Title | Description |
|---|---|---|
| FG000 | Any ZX008 Open Label Dose | Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 22, 2022 | Nov 3, 2025 |
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Single Group Assisgnment
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None (open label)
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| Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator | 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. | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver | 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. | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver | 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. | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver | 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. | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator | 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. | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator | 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. | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator | 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. | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator | 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. | Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
| Los Angeles |
| California |
| 90095 |
| United States |
| Ep0215 108 | San Diego | California | 92123 | United States |
| Ep0215 101 | San Francisco | California | 94158 | United States |
| Ep0215 103 | Aurora | Colorado | 80045 | United States |
| Ep0215 115 | Gulf Breeze | Florida | 32561 | United States |
| Ep0215 104 | Miami | Florida | 33155 | United States |
| Ep0215 141 | Orlando | Florida | 32803 | United States |
| Ep0215 121 | Winter Park | Florida | 32789 | United States |
| Ep0215 117 | Atlanta | Georgia | 30328 | United States |
| Ep0215 110 | Chicago | Illinois | 60611-2605 | United States |
| Ep0215 140 | Bethesda | Maryland | 20817 | United States |
| Ep0215 112 | Boston | Massachusetts | 02114 | United States |
| Ep0215 109 | Rochester | Minnesota | 55905 | United States |
| Ep0215 132 | Roseville | Minnesota | 55113 | United States |
| Ep0215 105 | Hackensack | New Jersey | 07601 | United States |
| Ep0215 118 | Livingston | New Jersey | 07039 | United States |
| Ep0215 150 | Hawthorne | New York | 10532 | United States |
| Ep0215 142 | New York | New York | 10016 | United States |
| Ep0215 131 | Cleveland | Ohio | 44106 | United States |
| Ep0215 124 | Memphis | Tennessee | 38103 | United States |
| Ep0215 146 | Dallas | Texas | 75207 | United States |
| Ep0215 126 | Fort Worth | Texas | 76104 | United States |
| Ep0215 106 | Salt Lake City | Utah | 84108 | United States |
| Ep0215 119 | Seattle | Washington | 98105 | United States |
| Ep0215 125 | Tacoma | Washington | 98405 | United States |
| Ep0215 301 | Heidelberg | Australia |
| Ep0215 302 | South Brisbane | Australia |
| Ep0215 303 | Westmead | Australia |
| Ep0215 803 | Brussels | Belgium |
| Ep0215 801 | Edegem | Belgium |
| Ep0215 802 | Jette | Belgium |
| Ep0215 202 | Montreal | Canada |
| Ep0215 204 | Toronto | Canada |
| Ep0215 201 | Vancouver | Canada |
| Ep0215 701 | Dianalund | Denmark |
| Ep0215 1004 | Bordeaux | France |
| Ep0215 1005 | Lille | France |
| Ep0215 1007 | Marseille | France |
| Ep0215 1001 | Paris | France |
| Ep0215 1002 | Paris | France |
| Ep0215 1008 | Salouël | France |
| Ep0215 902 | Bielefeld | Germany |
| Ep0215 906 | Freiburg im Breisgau | Germany |
| Ep0215 905 | Jena | Germany |
| Ep0215 908 | Kiel | Germany |
| Ep0215 903 | Radeberg | Germany |
| Ep0215 901 | Vogtareuth | Germany |
| Ep0215 1201 | Florence | Italy |
| Ep0215 1204 | Genova | Italy |
| Ep0215 1205 | Mantua | Italy |
| Ep0215 1207 | Milan | Italy |
| Ep0215 1206 | Roma | Italy |
| Ep0215 1208 | Roma | Italy |
| Ep0215 1202 | Verona | Italy |
| Ep0215 1604 | Guadalajara | Mexico |
| Ep0215 1402 | Heeze | Netherlands |
| Ep0215 1401 | Zwolle | Netherlands |
| Ep0215 1702 | Bydgoszcz | Poland |
| Ep0215 1701 | Krakow | Poland |
| Ep0215 1105 | Barcelona | Spain |
| Ep0215 1107 | Barcelona | Spain |
| Ep0215 1103 | Esplugues de Llobregat | Spain |
| Ep0215 1101 | Madrid | Spain |
| Ep0215 1102 | Pamplona | Spain |
| Ep0215 502 | Gothenburg | Sweden |
| Ep0215 605 | Birmingham | United Kingdom |
| Ep0215 601 | Glasgow | United Kingdom |
| Ep0215 603 | Liverpool | United Kingdom |
| Ep0215 602 | London | United Kingdom |
| Ep0215 606 | London | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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Baseline Characteristics refer to the Safety analysis set which consisted of all participants who received at least one dose of ZX008 during the open label extension (OLE).
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| ID | Title | Description |
|---|---|---|
| BG000 | Any ZX008 Open Label Dose | Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Age, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 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. | Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension. | Posted | Number | percentage of participants | From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days) |
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| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver | 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. | 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. | Posted | Count of Participants | Participants | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator | 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. | 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. | Posted | Count of Participants | Participants | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver | 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. | 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. | Posted | Count of Participants | Participants | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver | 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. | 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. | Posted | Count of Participants | Participants | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver | 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. | 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. | Posted | Count of Participants | Participants | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator | 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. | 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. | Posted | Count of Participants | Participants | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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| Secondary | Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator | 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. | 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. | Posted | Count of Participants | Participants | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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| Secondary | Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator | 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. | 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. | Posted | Count of Participants | Participants | Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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| Secondary | Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator | 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. | 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. | Posted | Count of Participants | Participants | Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49) |
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From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
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. Safety analysis set consisted of participants who received at least one dose of ZX008 during the open label extension.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Any ZX008 Open Label Dose | Participants continued to receive ZX008 dose prescribed at last visit in ZX008-1503/EP0212 (NCT02823145), ZX008-1601/EP0214 Part 2 (NCT03355209), or ZX008-1602/EP0207 (NCT03467113) and had volume adjusted according to weight. In this Open Label Extension (OLE) study, participants who were not receiving concomitant Stiripentol (STP), dose could be titrated in increments of 0.2 mg/kg/day (milligram per kilogram per day) up to maximum of 0.8 mg/kg/day (not exceeding 30 mg/day). For participants receiving concomitant STP, dose could be adjusted to 0.4 mg/kg/day initially and increased to 0.5 mg/kg/day (not exceeding 20 mg/day). Participants continued ZX008 until one of following occurred: approval of ZX008 obtained from regulatory authorities for participants indication; managed access program established as per country-specific requirements, legal and regulatory guidelines in participants country of residence; or investigational product development for participants indication stopped by Sponsor. | 3 | 412 | 67 | 412 | 204 | 412 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 21.1 | Non-systematic Assessment |
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| Cardiac arrest | Cardiac disorders | MedDRA 21.1 | Non-systematic Assessment |
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| Non-compaction cardiomyopathy | Congenital, familial and genetic disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pyloric stenosis | Congenital, familial and genetic disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Complication associated with device | General disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 21.1 | Non-systematic Assessment |
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| Gait inability | General disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pneumonia viral | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Cellulitis of male external genital organ | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Dengue haemorrhagic fever | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Legionella infection | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Nasal abscess | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pneumonia mycoplasmal | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Animal bite | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Craniocerebral injury | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Craniofacial fracture | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Extradural haematoma | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Jaw fracture | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Spinal column injury | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Traumatic renal injury | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 21.1 | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Diabetic ketoacidosis | Metabolism and nutrition disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Hypophagia | Metabolism and nutrition disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Status epilepticus | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Seizure cluster | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Altered state of consciousness | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Parkinsonism | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Petit mal epilepsy | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Thalamus haemorrhage | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Aggression | Psychiatric disorders | MedDRA 21.1 | Non-systematic Assessment |
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| Agitation | Psychiatric disorders | MedDRA 21.1 | Non-systematic Assessment |
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| Hallucination | Psychiatric disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Intentional self-injury | Psychiatric disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Psychotic disorder | Psychiatric disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.1 | Non-systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 21.1 | Non-systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 21.1 | Non-systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| UCB | Cares | 001 844 599 2273 | UCBCares@ucb.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 27, 2023 | Nov 3, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D004831 | Epilepsies, Myoclonic |
| D065768 | Lennox Gastaut Syndrome |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| D005277 | Fenfluramine |
| ID | Term |
|---|---|
| D010627 | Phenethylamines |
| D005021 | Ethylamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
Not provided
Not provided
| 18 - <65 years |
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| Black or African American |
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| Native Hawaiian or Other Pacific Islander |
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| White |
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| Other or Mixed |
|
| Not Reported |
|
| Unknown |
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| Not Reported |
|
| Unknown |
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