| ID | Type | Description | Link |
|---|---|---|---|
| ZX008-1502 | Other Identifier | Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. |
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Study 1 and Study 3 are the prospective, merged analyses of 2 identical double-blind, placebo-controlled studies, ZX008-1501 and ZX008-1502, to assess the efficacy, safety, and pharmacokinetics of ZX008 when used as adjunctive therapy in pediatric and young adult subjects with Dravet syndrome. Study 1501 and Study 1502 were conducted in parallel; Study 1501 was conducted at approximately 30 study sites in North America; Study 1502 was conducted at approximately 30 study sites in Europe, Asia and Australia. Upon completion of the Baseline Period after initial Screening and Baseline charting of seizure frequency, subjects who qualified for the studies were randomized (1:1:1) in a double-blind manner to receive either 1 of 2 doses of ZX008 (0.2 mg/kg/day or 0.8 mg/kg/day; maximum dose: 30 mg/day) or placebo. Randomization was stratified by age group (< 6 years, ≥6 to 18 years) to achieve balance across treatment arms, with the target of 25% of subjects in each age group. All subjects were titrated to their randomized dose over a 14-day Titration Period. Following titration, subjects continued treatment at their randomly assigned dose over a 12-week Maintenance Period. Subjects exiting the study underwent a 2-week taper, unless they enrolled in a follow-on study. Subjects were followed for post-study safety monitoring.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ZX008 - 0.8 mg/kg/day | Experimental | ZX008 (fenfluramine HCl) is supplied as an oral solution in concentrations of 1.25, 2.5, and 5 mg/mL. ZX008 will be administered twice a day (BID) in equally divided doses with food. |
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| ZX008 - 0.2 mg/kg/day | Experimental | ZX008 (fenfluramine HCl) is supplied as an oral solution in concentrations of 1.25, 2.5, and 5 mg/mL. ZX008 will be administered twice a day (BID) in equally divided doses with food. |
|
| Matching Placebo | Placebo Comparator | Placebo will be administered twice a day (BID) in equally divided doses with food. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ZX008 (Fenfluramine Hydrochloride) | Drug | ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride buffered to pH 5. The product is sugar free and is intended to be compatible with a ketogenic diet. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Mean Convulsive Seizures Frequency (MCSF) to the Combined Titration and Maintenance Periods (T+M) in Participants Receiving ZX008 0.8 mg/kg/Day Compared to Placebo | Monthly (28 day) convulsive seizure frequency (CSF) was based on electronic diary data obtained for each participant. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). The number of convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day CSF. | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Mean Convulsive Seizures Frequency to the Combined Titration and Maintenance Period (T+M) in Participants Receiving ZX008 0.2 mg/kg/Day Compared to Placebo | Monthly (28 day) convulsive seizure frequency (CSF) was based on electronic diary data obtained for each participant. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). The number of convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day CSF. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| UCB Cares | 001 844 599 2273 | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Children's | Phoenix | Arizona | 85016 | United States | ||
| Center for Neurosciences - Tucson |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37543865 | Result | Sullivan J, Lagae L, Cross JH, Devinsky O, Guerrini R, Knupp KG, Laux L, Nikanorova M, Polster T, Talwar D, Ceulemans B, Nabbout R, Farfel GM, Galer BS, Gammaitoni AR, Lock M, Agarwal A, Scheffer IE; FAiRE DS Study Group. Fenfluramine in the treatment of Dravet syndrome: Results of a third randomized, placebo-controlled clinical trial. Epilepsia. 2023 Oct;64(10):2653-2666. doi: 10.1111/epi.17737. Epub 2023 Aug 17. | |
| 34768178 |
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Due to slow enrollment into both trials, the databases for the two trials were combined. The first 72 enrolled participants from ZX008-1501 and first 47 from ZX008-1502 were combined for an analysis and reported as Study 1, whereas the final 55 participants from ZX008- 1501 and the final 88 from ZX008-1502 were combined and reported as Study 3.
The study 1501 started to enroll participants in January 2016 and concluded in July 2020.
The study 1502 started to enroll participants in July 2016 and concluded in July 2020. The consolidated results of Study 1 and Study 3 are included in this record. The Participant Flow refers to the Randomized Population.
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| ID | Title | Description |
|---|---|---|
| FG000 | Study 1: Placebo | Participants received matching placebo as an oral solution, twice a day (bid) in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| FG001 | Study 1: ZX008 0.2 mg/kg/Day |
| 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 | Oct 31, 2016 | Aug 19, 2022 |
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| Matching Placebo | Drug | Placebo solution for ZX008. The product is sugar free and is intended to be compatible with a ketogenic diet. |
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| From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Percentage of Participants Who Achieved Greater Than or Equal to 25% (≥25%) Reduction in Convulsive Seizure Frequency in Each ZX008 Treatment Arm Compared to Placebo From Baseline During the Titration and Maintenance Period | Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 25% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Percentage of Participants Who Achieved a ≥50% Reduction in Convulsive Seizure Frequency in Each ZX008 Treatment Arm Compared to Placebo From Baseline During the Titration and Maintenance Period | Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 50% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Percentage of Participants Who Achieved a ≥75% Reduction in Convulsive Seizure Frequency in Each ZX008 Treatment Arm Compared to Placebo From Baseline During the Titration and Maintenance Period | Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 75% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Percentage of Participants Who Achieved a 100% Reduction in Convulsive Seizure Frequency in Each ZX008 Treatment Arm Compared to Placebo From Baseline During the Titration and Maintenance Period | Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 100% reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Longest Convulsive Seizure-free Interval in Each ZX008 Treatment Arm Compared to Placebo During the Titration and Maintenance Period | The longest interval between convulsive seizures was calculated over the entire Titration and Maintenance Period and was derived as the maximum of the number of days between consecutive convulsive seizures. | During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days) |
| Number of Convulsive Seizure-free Days in Each ZX008 Treatment Arm Compared to Placebo During the Titration and Maintenance Period | A convulsive seizure free day was defined as a day for which diary data are available and no convulsive seizures were reported. Convulsive seizure free days were taken from the electronic diary data. | During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days) |
| Change From Baseline in Non-convulsive Seizure Frequency to the Combined Titration and Maintenance Period in Each ZX008 Treatment Arm Compared to Placebo | Non-convulsive seizures included focal without clear observable motor signs, absence or atypical absence, myoclonic and atonic. The number of non-convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day non-convulsive seizure frequency. | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Change From Baseline in Convulsive + Non-convulsive Seizure Frequency to the Combined Titration and Maintenance Period in Each ZX008 Treatment Arm Compared to Placebo | Total seizure frequency were defined as the combination of convulsive and non-convulsive seizures. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). Non-convulsive seizures included focal without clear observable motor signs, absence or atypical absence, myoclonic and atonic. The seizure frequency was based on electronic diary data obtained for each participant. The number of all seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day convulsive or non-convulsive seizure frequency. | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Percentage of Participants With Rescue Medication Usage in Each ZX008 Treatment Arm Compared to Placebo During the Titration and Maintenance Period | Rescue medication was administered according to each participant's usual or prescribed regimen consisting of 1 or more medications. The usage of rescue medication (number of days and number of medications used per seizure episode) was based on electronic diary data obtained for each participant. The number of days rescue medication was taken (normalized to 28 days) was calculated for each participant. Multiple medications taken on the same day were counted once for that day. | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
| Percentage of Participants With Hospitalization and Healthcare Resource Utilization to Treat Seizures in Each ZX008 Treatment Arm Compared to Placebo During Study | Participants who utilized medical center care to treat a seizure during the study were reported. | During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days) |
| Percentage of Participants With Status Epilepticus (SE) in Each ZX008 Treatment Arm Compared to Placebo During the Titration and Maintenance Period | The participants who either had SE episode recorded as an adverse event (AE) during treatment or a seizure greater than 10 minutes were reported for each treatment group. Additionally, a single participant who may had more than one episode of SE, and an episode of SE recorded as both an AE and as a seizure longer than 10 minutes was counted as a single event. | During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days) |
| Distribution of Duration of Convulsive Seizures (in Percentage) in Each ZX008 Treatment Arm Compared to Placebo at Baseline and During the Titration and Maintenance Period | Duration of single convulsive seizures during the Baseline and the duration over the Titration and Maintenance Period were reported by treatment group using categories as <2 minutes, 2 to 10 minutes and > 10 minutes as collected in the seizure diary. | At Baseline and 14 weeks of Titration (2 weeks) and Maintenance Period (12 weeks) |
| Percentage of Participants With Clinical Global Impression - Improvement (CGI-I) Rating Score, as Assessed by the Principal Investigator in Each ZX008 Treatment Arm Compared to Placebo | CGI-I scale measures improvement in the participant's clinical status from Baseline. The severity of a participant's condition was 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 and 7-very much worse. The Principal Investigator rated their global impression of the participant's condition during the study. | At Visit 6 (Day 15), 8 (Day 43), 10 (Day 71) and 12 (Day 99) |
| Percentage of Participants With Clinical Global Impression - Improvement Rating Score, as Assessed by the Parent/Caregiver in Each ZX008 Treatment Arm Compared to Placebo | CGI-I scale measures improvement in the participant's clinical status from Baseline. The severity of a participant's condition was 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 and 7-very much worse. The Parent/Caregiver rated their global impression of the participant's condition during the study. | At Visit 6 (Day 15), 8 (Day 43), 10 (Day 71) and 12 (Day 99) |
| Change From Baseline to Day 99 in the Quality of Life in Childhood Epilepsy (QOLCE) Score to Measure Quality of Life in Each ZX008 Treatment Arm Compared to Placebo | QOLCE is a low-burden parent/caregiver completed assessment that evaluates how epilepsy affects day-to day functioning of the participant in various life areas, including physical activities, well being, cognition, social activities, behavior, and general health. QOLCE scores items on 16 subscales with possible 5-point response for each, where scores of 5 was best possible response and 1 was worst possible response. Item scores were then transformed to a 0-100 scale as follows: 1-0, 2-25, 3-50, 4-75, 5-100. A score for each participant for each subscale was calculated by averaging that participant's responses to each item in the subscale. Subscale scores per participant were averaged to obtain an overall QoL score for each participant. Higher the subscale and overall QoL scores, better the response. | From Baseline to Day 99 |
| Change From Baseline to Day 99 in the Overall Quality of Life Score From the Pediatric Quality of Life Inventory™ (PedsQL) Score in Each ZX008 Treatment Arm Compared to Placebo | The Pediatric Quality of Life Inventory (PedsQL) is a pediatric modular measure of health related quality of life (QoL) completed by the parent/caregiver on behalf of the participant. It consisted of 23 items across 4 core scales that measure physical (8 items), emotional, social, and school functioning (5 items each). Each of the responses to the 23 items is initially scored on a 5-point Likert scale from 0 (Never) to 4 (Almost always). Scores are linearly transformed to a scale of 0 to 100, where 0=100, 1=75, 2=50, 3=25 and 4=0, and higher scores correspond to better health-related QoL. The Overall Quality of Life is the average of all the items over the number of items answered on all the Scales. | From Baseline to Day 99 |
| Change From Baseline to Day 99 in the Total Score From PedsQL Family Impact Module Score in Each ZX008 Treatment Arm Compared to Placebo | The PedsQL Family Impact measured the impact of pediatric chronic health conditions on parents and the family by measuring parent self-reported physical, emotional, social, and cognitive functioning, communication, worry, and family daily activities and relationships. There are a total of 36 items in the PedsQL: 6 items for Physical Functioning, 5 items each for Emotional Functioning, Cognitive Functioning and Worry, 4 for Social Functioning, 3 for Communication, 3 questions for Daily Activities, and 5 for Family Relationships. Each of the responses are initially scored on a 5-point Likert scale from 0 (Never) to 4 (Almost always) and then linearly transformed to a scale of 0 to 100, where 0=100, 1=75, 2=50, 3=25 and 4=0, and higher scores mean better health-related QoL. | From Baseline to Day 99 |
| Quality of Life (QoL) of the Parent/Caregiver Using the EQ- 5D-5L Scale in Each ZX008 Treatment Arm Compared to Placebo at Baseline and Day 99 | The EuroQOL-5 Dimensions-5 Levels scale produced by European QOL Group (EQ-5D-5L) health questionnaire is a health-related QOL instrument with 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The 5 dimensions of EQ-5D-5L health questionnaire were assessed on a Likert scale with 5 possible levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. The categories "slight problems", "moderate problems", "severe problems" and "extreme problems" are collapsed into one response category "problems. The QOL of the parent/caregiver was assessed and percentage of participants was reported for each item. | At Baseline and Day 99 |
| Change From Baseline to Day 99 in Affective Symptoms of the Parent/Caregiver Using the Hospital Anxiety and Depression Scale (HADS) in Each ZX008 Treatment Arm Compared to Placebo | The HADS is a tool that was validated to assess presence of anxiety or depression in an outpatient non-psychiatric population. The HADS a 14-item scale that generates ordinal data for 2 dimensions: 1) Anxiety (7 items), and 2) Depression (7 items). Each item has 4 possible answers rated 0 to 3, of which 0 = No distress and 3 = worst distress. All answers to the items for a dimension with their respective rating are added resulting in a range for each dimension from 0-21, out of which of 0-7 = normal; 8-10=borderline abnormal; 11-21=abnormal. Scores for the entire scale (emotional distress) range from 0 to 42, with higher scores indicating more distress. | From Baseline to Day 99 |
| Maximum Observed Concentration of ZX008 Determined Directly From the Concentration Time Profile [Cmax] at Steady State | Cmax is the maximum observed concentration determined directly from the concentration-time profile. | At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose |
| Area Under the Concentration Time Curve of ZX008 From Time Zero to Time 24 Hours [AUC0-24hours] at Steady State | AUC0-24 is the area under the concentration time curve from time zero to 24 hours. | At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose |
| Time to Maximum Concentration [Tmax] of ZX008 at Steady State | Tmax is the time to maximum concentration at steady state. | At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose |
| Elimination Half-life [t1/2 Beta] of ZX008 at Steady State | t1/2 beta is the elimination half-life. | At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose |
| Tucson |
| Arizona |
| 85718 |
| United States |
| Rady Children's Hospital San Diego | San Diego | California | 92123 | United States |
| University of California San Francisco | San Francisco | California | 94143 | United States |
| The Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| NW FL Clinical Research Group, LLC | Gulf Breeze | Florida | 32561 | United States |
| Miami Children's Hospital Brain Institute | Miami | Florida | 33155 | United States |
| Neurology and Epilepsy Research Center | Orlando | Florida | 32819 | United States |
| Panda Neurology | Atlanta | Georgia | 30328 | United States |
| Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02467 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Saint Barnabas Medical Center | Livingston | New Jersey | 07039 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76087 | United States |
| University of Utah | Salt Lake City | Utah | 84113 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| MultiCare Institute for Research & Innovation | Tacoma | Washington | 98405 | United States |
| Melbourne Brain Centre Austin Hospital | Melbourne | Australia |
| Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital | South Brisbane | Australia |
| The Children's Hospital Westmead Dept. of Neurology and Neurosurgery | Westmead | Australia |
| Universitair Ziekenhuis Antwerpen | Antwerp | Belgium |
| British Columbia Children's Hospital BCCH | Vancouver | British Columbia | V6H3V4 | Canada |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | H3T 1C5 | Canada |
| Danish National Epilepsy Centre | Dianalund | Denmark |
| French Ref centre Necker Hospital Paris | Paris | France |
| Epilepsiezentrum / Neuropädiatrie Hedwig-von-Rittberg-Zentrum Für Kinder und Jugendliche | Berlin | Germany |
| Krankenhaus Mara Epilepsie-Zentrum Bethel | Bielefeld | Germany |
| Epilepsiezentrum Freiburg | Freiburg im Breisgau | Germany |
| Universitaetsklinikum Jena Klinik fuer Kinder- und Jugendmedizin Neuropaediatrie | Jena | Germany |
| Klinik für Neuropädiatrie Universitätsklinikum Schleswig Holstein Campus Kiel | Kiel | Germany |
| Kleinwachau Saechsisches Epilepsiezentrum Radeberggemeinnuetzige GmbH | Radeberg | Germany |
| Universitaetsklinik fuer Kinder- und Jugendmedizin Abteilung III | Tübingen | Germany |
| Schoen Klinik Vogtareuth Neuropaediatrie und Neurologische Rehabilitation, Epilepsiezentrum fuer Kinder und Jugendlische,Tagesklinik fuer Neuropaediatrie | Vogtareuth | Germany |
| AOU Anna Meyer | Florence | 50139 | Italy |
| Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia | Genova | Italy |
| A.O Carlo Poma | Mantua | 46100 | Italy |
| Instituto Neurologica Carlo Besta | Milan | 20133 | Italy |
| Ospedale Fatebenefratelli e Oftalmico | Milan | Italy |
| U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù, IRCS | Roma | 00165 | Italy |
| Ospedal Policlinico Giambattista Rossi diBorga Roma | Verona | 37134 | Italy |
| Okayama University Hospital | Okayama | Okayama-ken | Japan |
| Saitama Children's Medical Center | Saitama-shi | Saitama | Japan |
| National Epilepsy Center Shizuoka Institute | Shizuoka | Shizuoka | Japan |
| Tokyo Women's Medical University Hospital | Shinjuku-ku | Tokyo | Japan |
| Hospital Sant Joande Déu | Barcelona | Spain |
| Hospital Ruber Internacional Primera Planta Servicio de Neurologia | Madrid | Spain |
| Clinica Universitaria de Navarra Fase 4. Segunda planta, Consulta de Pediatria | Pamplona | Spain |
| Birmingham Children Hospital | Birmingham | United Kingdom |
| Institute of Neurosciences Queens Elizabeth University Hospital | Glasgow | United Kingdom |
| Alder Hey Hospital | Liverpool | United Kingdom |
| Evelina Hospital | London | United Kingdom |
| Great Ormonnd Street Hospital for Children NHS Foundation Trust | London | United Kingdom |
| Sheffield Children's Hospital | Sheffield | United Kingdom |
| Derived |
| Cross JH, Galer BS, Gil-Nagel A, Devinsky O, Ceulemans B, Lagae L, Schoonjans AS, Donner E, Wirrell E, Kothare S, Agarwal A, Lock M, Gammaitoni AR. Impact of fenfluramine on the expected SUDEP mortality rates in patients with Dravet syndrome. Seizure. 2021 Dec;93:154-159. doi: 10.1016/j.seizure.2021.10.024. Epub 2021 Nov 2. |
| 34676542 | Derived | Sullivan J, Specchio N, Devinsky O, Auvin S, Perry MS, Strzelczyk A, Gil-Nagel A, Dai D, Galer BS, Gammaitoni AR. Fenfluramine significantly reduces day-to-day seizure burden by increasing number of seizure-free days and time between seizures in patients with Dravet syndrome: A time-to-event analysis. Epilepsia. 2022 Jan;63(1):130-138. doi: 10.1111/epi.17106. Epub 2021 Oct 22. |
| 33540241 | Derived | Sullivan J, Perry MS, Wheless JW, Galer B, Gammaitoni A. Fenfluramine responder analyses and numbers needed to treat: Translating epilepsy trial data into clinical practice. Eur J Paediatr Neurol. 2021 Mar;31:10-14. doi: 10.1016/j.ejpn.2021.01.005. Epub 2021 Jan 22. |
| 31862249 | Derived | Lagae L, Sullivan J, Knupp K, Laux L, Polster T, Nikanorova M, Devinsky O, Cross JH, Guerrini R, Talwar D, Miller I, Farfel G, Galer BS, Gammaitoni A, Mistry A, Morrison G, Lock M, Agarwal A, Lai WW, Ceulemans B; FAiRE DS Study Group. Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: a randomised, double-blind, placebo-controlled trial. Lancet. 2019 Dec 21;394(10216):2243-2254. doi: 10.1016/S0140-6736(19)32500-0. Epub 2019 Dec 17. |
Participants received ZX008 0.2 milligram per kilogram per day (mg/kg/day) as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| FG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| FG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| FG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| FG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| COMPLETED |
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| NOT COMPLETED |
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Baseline characteristics refers to the Randomized population which included all participants randomized to receive study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Study 1: Placebo | Participants received matching placebo as an oral solution, twice a day (bid) in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| BG001 | Study 1: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 milligram per kilogram per day (mg/kg/day) as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| BG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| BG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| BG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| BG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| BG006 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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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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| 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 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Change From Baseline in the Mean Convulsive Seizures Frequency (MCSF) to the Combined Titration and Maintenance Periods (T+M) in Participants Receiving ZX008 0.8 mg/kg/Day Compared to Placebo | Monthly (28 day) convulsive seizure frequency (CSF) was based on electronic diary data obtained for each participant. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). The number of convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day CSF. | The modified intent-to-treat (mITT) population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Mean | Standard Deviation | seizure frequency per 28 days | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Change From Baseline in the Mean Convulsive Seizures Frequency to the Combined Titration and Maintenance Period (T+M) in Participants Receiving ZX008 0.2 mg/kg/Day Compared to Placebo | Monthly (28 day) convulsive seizure frequency (CSF) was based on electronic diary data obtained for each participant. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). The number of convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day CSF. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Mean | Standard Deviation | seizure frequency per 28 days | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Percentage of Participants Who Achieved Greater Than or Equal to 25% (≥25%) Reduction in Convulsive Seizure Frequency in Each ZX008 Treatment Arm Compared to Placebo From Baseline During the Titration and Maintenance Period | Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 25% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Percentage of Participants Who Achieved a ≥50% Reduction in Convulsive Seizure Frequency in Each ZX008 Treatment Arm Compared to Placebo From Baseline During the Titration and Maintenance Period | Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 50% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Percentage of Participants Who Achieved a ≥75% Reduction in Convulsive Seizure Frequency in Each ZX008 Treatment Arm Compared to Placebo From Baseline During the Titration and Maintenance Period | Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 75% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Percentage of Participants Who Achieved a 100% Reduction in Convulsive Seizure Frequency in Each ZX008 Treatment Arm Compared to Placebo From Baseline During the Titration and Maintenance Period | Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 100% reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Longest Convulsive Seizure-free Interval in Each ZX008 Treatment Arm Compared to Placebo During the Titration and Maintenance Period | The longest interval between convulsive seizures was calculated over the entire Titration and Maintenance Period and was derived as the maximum of the number of days between consecutive convulsive seizures. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Median | Full Range | days | During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days) |
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| Secondary | Number of Convulsive Seizure-free Days in Each ZX008 Treatment Arm Compared to Placebo During the Titration and Maintenance Period | A convulsive seizure free day was defined as a day for which diary data are available and no convulsive seizures were reported. Convulsive seizure free days were taken from the electronic diary data. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Median | Full Range | seizure free days | During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days) |
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| Secondary | Change From Baseline in Non-convulsive Seizure Frequency to the Combined Titration and Maintenance Period in Each ZX008 Treatment Arm Compared to Placebo | Non-convulsive seizures included focal without clear observable motor signs, absence or atypical absence, myoclonic and atonic. The number of non-convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day non-convulsive seizure frequency. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. Here, number of participants analyzed included those participants who were evaluable for the assessment. | Posted | Median | Full Range | seizure frequency per 28 days | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Change From Baseline in Convulsive + Non-convulsive Seizure Frequency to the Combined Titration and Maintenance Period in Each ZX008 Treatment Arm Compared to Placebo | Total seizure frequency were defined as the combination of convulsive and non-convulsive seizures. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). Non-convulsive seizures included focal without clear observable motor signs, absence or atypical absence, myoclonic and atonic. The seizure frequency was based on electronic diary data obtained for each participant. The number of all seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day convulsive or non-convulsive seizure frequency. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Median | Full Range | seizure frequency per 28 days | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Percentage of Participants With Rescue Medication Usage in Each ZX008 Treatment Arm Compared to Placebo During the Titration and Maintenance Period | Rescue medication was administered according to each participant's usual or prescribed regimen consisting of 1 or more medications. The usage of rescue medication (number of days and number of medications used per seizure episode) was based on electronic diary data obtained for each participant. The number of days rescue medication was taken (normalized to 28 days) was calculated for each participant. Multiple medications taken on the same day were counted once for that day. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)] |
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| Secondary | Percentage of Participants With Hospitalization and Healthcare Resource Utilization to Treat Seizures in Each ZX008 Treatment Arm Compared to Placebo During Study | Participants who utilized medical center care to treat a seizure during the study were reported. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days) |
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| Secondary | Percentage of Participants With Status Epilepticus (SE) in Each ZX008 Treatment Arm Compared to Placebo During the Titration and Maintenance Period | The participants who either had SE episode recorded as an adverse event (AE) during treatment or a seizure greater than 10 minutes were reported for each treatment group. Additionally, a single participant who may had more than one episode of SE, and an episode of SE recorded as both an AE and as a seizure longer than 10 minutes was counted as a single event. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days) |
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| Secondary | Distribution of Duration of Convulsive Seizures (in Percentage) in Each ZX008 Treatment Arm Compared to Placebo at Baseline and During the Titration and Maintenance Period | Duration of single convulsive seizures during the Baseline and the duration over the Titration and Maintenance Period were reported by treatment group using categories as <2 minutes, 2 to 10 minutes and > 10 minutes as collected in the seizure diary. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of seizures | At Baseline and 14 weeks of Titration (2 weeks) and Maintenance Period (12 weeks) |
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| Secondary | Percentage of Participants With Clinical Global Impression - Improvement (CGI-I) Rating Score, as Assessed by the Principal Investigator in Each ZX008 Treatment Arm Compared to Placebo | CGI-I scale measures improvement in the participant's clinical status from Baseline. The severity of a participant's condition was 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 and 7-very much worse. The Principal Investigator rated their global impression of the participant's condition during the study. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | At Visit 6 (Day 15), 8 (Day 43), 10 (Day 71) and 12 (Day 99) |
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| Secondary | Percentage of Participants With Clinical Global Impression - Improvement Rating Score, as Assessed by the Parent/Caregiver in Each ZX008 Treatment Arm Compared to Placebo | CGI-I scale measures improvement in the participant's clinical status from Baseline. The severity of a participant's condition was 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 and 7-very much worse. The Parent/Caregiver rated their global impression of the participant's condition during the study. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Number | percentage of participants | At Visit 6 (Day 15), 8 (Day 43), 10 (Day 71) and 12 (Day 99) |
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| Secondary | Change From Baseline to Day 99 in the Quality of Life in Childhood Epilepsy (QOLCE) Score to Measure Quality of Life in Each ZX008 Treatment Arm Compared to Placebo | QOLCE is a low-burden parent/caregiver completed assessment that evaluates how epilepsy affects day-to day functioning of the participant in various life areas, including physical activities, well being, cognition, social activities, behavior, and general health. QOLCE scores items on 16 subscales with possible 5-point response for each, where scores of 5 was best possible response and 1 was worst possible response. Item scores were then transformed to a 0-100 scale as follows: 1-0, 2-25, 3-50, 4-75, 5-100. A score for each participant for each subscale was calculated by averaging that participant's responses to each item in the subscale. Subscale scores per participant were averaged to obtain an overall QoL score for each participant. Higher the subscale and overall QoL scores, better the response. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Mean | Standard Deviation | score on a scale | From Baseline to Day 99 |
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| Secondary | Change From Baseline to Day 99 in the Overall Quality of Life Score From the Pediatric Quality of Life Inventory™ (PedsQL) Score in Each ZX008 Treatment Arm Compared to Placebo | The Pediatric Quality of Life Inventory (PedsQL) is a pediatric modular measure of health related quality of life (QoL) completed by the parent/caregiver on behalf of the participant. It consisted of 23 items across 4 core scales that measure physical (8 items), emotional, social, and school functioning (5 items each). Each of the responses to the 23 items is initially scored on a 5-point Likert scale from 0 (Never) to 4 (Almost always). Scores are linearly transformed to a scale of 0 to 100, where 0=100, 1=75, 2=50, 3=25 and 4=0, and higher scores correspond to better health-related QoL. The Overall Quality of Life is the average of all the items over the number of items answered on all the Scales. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. | Posted | Mean | Standard Deviation | score on a scale | From Baseline to Day 99 |
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| Secondary | Change From Baseline to Day 99 in the Total Score From PedsQL Family Impact Module Score in Each ZX008 Treatment Arm Compared to Placebo | The PedsQL Family Impact measured the impact of pediatric chronic health conditions on parents and the family by measuring parent self-reported physical, emotional, social, and cognitive functioning, communication, worry, and family daily activities and relationships. There are a total of 36 items in the PedsQL: 6 items for Physical Functioning, 5 items each for Emotional Functioning, Cognitive Functioning and Worry, 4 for Social Functioning, 3 for Communication, 3 questions for Daily Activities, and 5 for Family Relationships. Each of the responses are initially scored on a 5-point Likert scale from 0 (Never) to 4 (Almost always) and then linearly transformed to a scale of 0 to 100, where 0=100, 1=75, 2=50, 3=25 and 4=0, and higher scores mean better health-related QoL. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. Here, number of participants analyzed included those participants who were evaluable for the assessment. | Posted | Mean | Standard Deviation | score on a scale | From Baseline to Day 99 |
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| Secondary | Quality of Life (QoL) of the Parent/Caregiver Using the EQ- 5D-5L Scale in Each ZX008 Treatment Arm Compared to Placebo at Baseline and Day 99 | The EuroQOL-5 Dimensions-5 Levels scale produced by European QOL Group (EQ-5D-5L) health questionnaire is a health-related QOL instrument with 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The 5 dimensions of EQ-5D-5L health questionnaire were assessed on a Likert scale with 5 possible levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. The categories "slight problems", "moderate problems", "severe problems" and "extreme problems" are collapsed into one response category "problems. The QOL of the parent/caregiver was assessed and percentage of participants was reported for each item. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Number | percentage of participants | At Baseline and Day 99 |
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| Secondary | Change From Baseline to Day 99 in Affective Symptoms of the Parent/Caregiver Using the Hospital Anxiety and Depression Scale (HADS) in Each ZX008 Treatment Arm Compared to Placebo | The HADS is a tool that was validated to assess presence of anxiety or depression in an outpatient non-psychiatric population. The HADS a 14-item scale that generates ordinal data for 2 dimensions: 1) Anxiety (7 items), and 2) Depression (7 items). Each item has 4 possible answers rated 0 to 3, of which 0 = No distress and 3 = worst distress. All answers to the items for a dimension with their respective rating are added resulting in a range for each dimension from 0-21, out of which of 0-7 = normal; 8-10=borderline abnormal; 11-21=abnormal. Scores for the entire scale (emotional distress) range from 0 to 42, with higher scores indicating more distress. | The mITT population included all randomized participants who received at least 1 dose of ZX008 or placebo and for whom at least 1 week of diary data were available. Here, number of participants analyzed included those participants who were evaluable for the assessment. | Posted | Mean | Standard Deviation | score on a scale | From Baseline to Day 99 |
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| Secondary | Maximum Observed Concentration of ZX008 Determined Directly From the Concentration Time Profile [Cmax] at Steady State | Cmax is the maximum observed concentration determined directly from the concentration-time profile. | PK population for each study represents participants randomized to ZX008 and provided concentrations for use in the population PK analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanograms per milliliter (ng/mL) | At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose |
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| Secondary | Area Under the Concentration Time Curve of ZX008 From Time Zero to Time 24 Hours [AUC0-24hours] at Steady State | AUC0-24 is the area under the concentration time curve from time zero to 24 hours. | PK population for each study represents participants randomized to ZX008 and provided concentrations for use in the population PK analysis. PK samples at Visit 8 (Day 43) taken pre-dose to 6 hours post-dose were used to develop a population PK model. The model was utilized to generate plasma concentration-time curve over 24 hours at steady-state in study participants. AUC0-24 was calculated by numerical integration of the individual predicted concentration-time curve. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanogram*hour per milliliter (ng*h/mL) | At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose |
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| Secondary | Time to Maximum Concentration [Tmax] of ZX008 at Steady State | Tmax is the time to maximum concentration at steady state. | PK population for each study represents participants randomized to ZX008 and provided concentrations for use in the population PK analysis. | Posted | Median | Full Range | hours (h) | At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose |
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| Secondary | Elimination Half-life [t1/2 Beta] of ZX008 at Steady State | t1/2 beta is the elimination half-life. | PK population for each study represents participants randomized to ZX008 and provided concentrations for use in the population PK analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours (h) | At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose |
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From Titration Period until the Safety Follow-up Visit (up to Day 113)
A Treatment emergent adverse event (TEAE) was defined as any AE that based on start date information occurred after the first dose of study drug. The safety population included all randomized participants who received at least 1 dose of ZX008 or placebo.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
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| EG000 | Study 1: Placebo | Participants received matching placebo as an oral solution, twice a day (bid) in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. | 0 | 40 | 4 | 40 | 22 | 40 |
| EG001 | Study 1: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 milligram per kilogram per day (mg/kg/day) as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. | 0 | 39 | 4 | 39 | 35 | 39 |
| EG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. | 0 | 40 | 5 | 40 | 36 | 40 |
| EG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. | 1 | 48 | 2 | 48 | 37 | 48 |
| EG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. | 0 | 46 | 3 | 46 | 41 | 46 |
| EG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. | 0 | 48 | 3 | 48 | 41 | 48 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Adverse drug reaction | General disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Sudden unexplained death in epilepsy | General disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Gastroenteritis viral | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Lower respiratory tract infection | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Varicella | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Head injury | Injury, poisoning and procedural complications | MedDRA v 19.0 | Non-systematic Assessment |
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| Skull fracture | Injury, poisoning and procedural complications | MedDRA v 19.0 | Non-systematic Assessment |
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| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA v 19.0 | Non-systematic Assessment |
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| Hepatic enzyme increased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
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| Weight decreased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Febrile convulsion | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Lethargy | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Seizure | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Somnolence | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Status epilepticus | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Fatigue | General disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Pyrexia | General disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Blood glucose decreased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
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| Blood pressure diastolic increased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
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| Echocardiogram abnormal | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Lethargy | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Somnolence | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Salivary hypersecretion | Gastrointestinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Asthenia | General disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Gait disturbance | General disorders | MedDRA v 19.0 | Non-systematic Assessment |
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| Croup infectious | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
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| Influenza | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Blood pressure systolic increased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Blood prolactin increased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Heart rate increased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Ataxia | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Balance disorder | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Drooling | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Hypotonia | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Sedation | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Seizure cluster | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Status epilepticus | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Abnormal behaviour | Psychiatric disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Enuresis | Psychiatric disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Negativism | Psychiatric disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA v 19.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v 19.0 | Non-systematic Assessment |
|
The full results posting consists of the pooled analysis 1 and 2 of the studies with study ID ZX008-1501 and ZX008-1502. Pooled analysis 1 is referenced as Study 1 in the respective statistical analysis plan (SAP) and corresponding clinical study report (CSR). Pooled analysis 2 is referenced as Study 2 in the respective SAP and, due to the timing of regulatory submissions, Study 3 in the corresponding CSR and in this results posting.
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 | Jun 9, 2020 | Aug 19, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D004831 | Epilepsies, Myoclonic |
| D004827 | Epilepsy |
| D012640 | Seizures |
| D001927 | Brain Diseases |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| D005277 | Fenfluramine |
| ID | Term |
|---|---|
| D010627 | Phenethylamines |
| D005021 | Ethylamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
Not provided
Not provided
| 12 - < 18 Years |
|
| 18 - < 65 Years |
|
| Male |
|
| Black or African American |
|
| Asian |
|
| American Indian or Alaska Native |
|
| Other |
|
| Not Reported |
|
| Unknown |
|
| Percentage difference from Placebo |
| 64.75 |
| 2-Sided |
| 95 |
| 51.85 |
| 74.19 |
Estimate was obtained from the LSMeans on the log scale as follows: 100 x [1 - exp(LS mean active - LS mean placebo).](streamdown:incomplete-link) |
| Superiority |
| OG002 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies.
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
|
Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies.
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
Participants received ZX008 0.2 milligram per kilogram per day (mg/kg/day) as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 |
| Study 1: ZX008 0.8 mg/kg/Day |
Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
Participants received ZX008 0.2 milligram per kilogram per day (mg/kg/day) as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
Participants received ZX008 0.2 milligram per kilogram per day (mg/kg/day) as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
Participants received ZX008 0.2 milligram per kilogram per day (mg/kg/day) as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
Participants received ZX008 0.2 milligram per kilogram per day (mg/kg/day) as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies.
| OG002 | Study 1: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 1 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: Placebo | Participants received matching placebo as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG004 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG005 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG003 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
|
|
| OG002 | Study 3: ZX008 0.2 mg/kg/Day | Participants received ZX008 0.2 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
| OG003 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
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| OG003 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
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| OG003 | Study 3: ZX008 0.8 mg/kg/Day | Participants received ZX008 0.8 mg/kg/day as an oral solution, bid, in equally divided doses with food for up to approximately 16 weeks. Study 3 is the merged analysis of 2 identical (ZX008-1501 and ZX008-1502) studies. |
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