NCT03739840

Brief Summary

The purpose of the study is to evaluate the efficacy, safety and tolerability of the 3 selected dose regimens of padsevonil (PSL) administered concomitantly with up to 3 anti-epileptic drugs (AEDs) compared with placebo for treatment of observable focal-onset seizures in subjects with drug-resistant epilepsy.

Trial Health

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Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
232

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Mar 2019

Geographic Reach
26 countries

136 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 9, 2018

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 14, 2018

Completed
4 months until next milestone

Study Start

First participant enrolled

March 6, 2019

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 28, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 28, 2020

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

October 25, 2021

Completed
Last Updated

December 21, 2022

Status Verified

December 1, 2022

Enrollment Period

1.6 years

First QC Date

November 9, 2018

Results QC Date

September 24, 2021

Last Update Submit

December 19, 2022

Conditions

Keywords

EpilepsyPadsevonil

Outcome Measures

Primary Outcomes (4)

  • Change in Log-transformed Observable Focal-onset Seizure Frequency From Baseline Over the 12-week Maintenance Period

    During the study, participants kept diaries to record daily seizure activity. Seizure frequency refers to 28-day adjusted frequency. Seizure frequency was based on investigator assessment of participants' reports of daily seizure type and frequency. Observable focal-onset seizures refer to Type IA1, IB, and IC (ILAE Classification of Epileptic Seizures, 1981). Based on ANCOVA on change in log-transformed seizure frequency from Baseline, with treatment group as the main factor, Baseline log-transformed seizure frequency as a continuous covariate, Baseline SV2A use (Yes or No) and Region (Europe, non-Europe) as categorical factors.

    From Baseline over the 12 Week Maintenance Period (up to Week 16)

  • Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)

    An Adverse Event is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. A TEAE was defined as any event not present before the initiation of the first dose of study treatment or any unresolved event already present before initiation of the first dose that worsened in intensity following exposure to the treatment.

    From Baseline until Safety Follow-Up (up to Week 23)

  • Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) Leading to Study Withdrawal

    An Adverse Event is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. A TEAE was defined as any event not present before the initiation of the first dose of study treatment or any unresolved event already present before initiation of the first dose that worsened in intensity following exposure to the treatment.

    From Baseline until Safety Follow-Up (up to Week 23)

  • Percentage of Participants With Treatment-Emergent Serious Adverse Events (SAEs)

    A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose: Results in death, is life-threatening, requires in patient hospitalization or prolongation of existing hospitalization, is a congenital anomaly or birth defect, is as infection that requires treatment parenteral antibiotics, other important medical events which based on medical or scientific judgement may jeopardize the patients, or may require medical or surgical intervention to prevent any of the above. A TEAE was defined as any event not present before the initiation of the first dose of study treatment or any unresolved event already present before initiation of the first dose that worsened in intensity following exposure to the treatment.

    From Baseline until Safety Follow-Up (up to Week 23)

Secondary Outcomes (3)

  • 75% Responder Rate From Baseline Over the 12-week Maintenance Period

    From Baseline over the 12 Week Maintenance Period (up to Week 16)

  • 50% Responder Rate From Baseline Over the 12-week Maintenance Period

    From Baseline over the 12 Week Maintenance Period (up to Week 16)

  • Percent Change in Observable Focal-onset Seizure Frequency From Baseline Over the 12-week Maintenance Period

    From Baseline over the 12 Week Maintenance Period (up to Week 16)

Study Arms (4)

Padsevonil dosing regimen 1

EXPERIMENTAL

Subjects will be randomized to receive a combination of tablets of padsevonil and placebo (as appropriate) to maintain the blinding.

Drug: PadsevonilDrug: Placebo

Padsevonil dosing regimen 2

EXPERIMENTAL

Subjects will be randomized to receive a combination of tablets of padsevonil and placebo (as appropriate) to maintain the blinding.

Drug: PadsevonilDrug: Placebo

Padsevonil dosing regimen 3

EXPERIMENTAL

Subjects will be randomized to receive a combination of tablets of padsevonil and placebo (as appropriate) to maintain the blinding.

Drug: PadsevonilDrug: Placebo

Placebo

PLACEBO COMPARATOR

Subjects randomized to the placebo group will receive a combination of several placebo tablets to maintain the blinding.

Drug: Placebo

Interventions

Padsevonil in different dosages.

Padsevonil dosing regimen 1Padsevonil dosing regimen 2Padsevonil dosing regimen 3

Placebo will be provided matching padsevonil.

Padsevonil dosing regimen 1Padsevonil dosing regimen 2Padsevonil dosing regimen 3Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of focal epilepsy per 1989 International League Against Epilepsy (ILAE) criteria at least 3 years before study entry
  • Subject has failed to achieve seizure control with \>=4 tolerated and appropriately chosen prior antiepileptic drugs (AED), including past and ongoing treatment, that were individually optimized for adequate dose and duration. Prior discontinued AED treatment would need to be assessed by the Investigator considering the patient medical records and patient and/or caregiver interview. 'Prior AED' is defined as all past and ongoing AED treatments with a start date before the Screening Visit (Visit 1)
  • Average of \>= 4 spontaneous and observable focal seizures (type IA1 (i.e. focal aware), IB (i.e. focal impaired awareness), IC (i.e. focal to bilateral tonic-clonic)) per month
  • Current treatment with an individually optimized and stable dose of at least 1 and up to 3 AEDs for the 8 weeks prior to the Screening Visit with or without additional Vagus Nerve Stimulation (VNS) or other neurostimulation treatments

You may not qualify if:

  • Subject has a history of or signs of generalized or combined generalized and focal epilepsy
  • Cluster seizures which are uncountable in the previous 8 weeks before study entry and during 4 weeks prospective baseline
  • Current treatment with carbamazepine, phenytoin, primidone, phenobarbital
  • Current treatment/ use of (non-AED) prescription, nonprescription, dietary (eg, grapefruit or passion fruit), or herbal products that are potent inducers or inhibitors of the CYP3A4 or 2C19 pathway for 2 weeks (or 5 half-lives, whichever is longer) prior to the Baseline Visit
  • Subjects taking sensitive substrates of CYP2C19 for 2 weeks (or 5 half-lives, whichever is longer) prior to the Baseline Visit
  • Subject has been taking vigabatrin less than 2 years at study entry
  • Subject has been taking felbamate for less than 12 months
  • Subject taking retigabine for less than 4 years
  • Current treatment with benzodiazepines (i.e. GABA-A-ergic drugs like zolpidem, zaleplon, or zopiclone, excluding GABA-A-ergic AEDs) \<3 times per week for emergencies
  • Subject has a current medical condition that occurred within the last 12 months which, in the opinion of the investigator, could compromise his/her safety or ability to participate in this study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (141)

Ep0092 839

Chandler, Arizona, 85226, United States

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Tucson, Arizona, 85724, United States

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Carlsbad, California, 92011, United States

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Ep0092 629

Orange, California, 92868, United States

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Washington D.C., District of Columbia, 20037, United States

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Bradenton, Florida, 34209, United States

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Ep0092 640

Hialeah, Florida, 33016, United States

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Ep0092 641

Jacksonville, Florida, 32209, United States

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Ep0092 823

Orlando, Florida, 32806, United States

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Ep0092 803

Honolulu, Hawaii, 96817, United States

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Ep0092 637

Urbana, Illinois, 61801, United States

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Ep0092 880

Anderson, Indiana, 46011, United States

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Ep0092 638

Fort Wayne, Indiana, 46804, United States

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Ep0092 630

Ames, Iowa, 50010, United States

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Ep0092 707

Lexington, Kentucky, 40536, United States

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Baltimore, Maryland, 21287, United States

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Bethesda, Maryland, 20817, United States

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Boston, Massachusetts, 02215, United States

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Ep0092 645

Golden Valley, Minnesota, 55422, United States

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Minneapolis, Minnesota, 55416, United States

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Brooklyn, New York, 11203, United States

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New York, New York, 10075, United States

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Syracuse, New York, 13210, United States

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The Bronx, New York, 10467, United States

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Chapel Hill, North Carolina, 27599, United States

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Charlotte, North Carolina, 28204, United States

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Columbus, Ohio, 43210, United States

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Ep0092 647

Oklahoma City, Oklahoma, 73106, United States

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Portland, Oregon, 97225, United States

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Philadelphia, Pennsylvania, 19107, United States

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Charlottesville, Virginia, 22903, United States

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Ep0092 639

Renton, Washington, 98055, United States

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Box Hill, Australia

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Camperdown, Australia

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Fitzroy, Australia

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Heidelberg, Australia

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Melbourne, Australia

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Brussels, Belgium

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Ottignies, Belgium

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Bihać, Bosnia and Herzegovina

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Mostar, Bosnia and Herzegovina

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Sarajevo, Bosnia and Herzegovina

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Tuzla, Bosnia and Herzegovina

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Ep0092 150

Blagoevgrad, Bulgaria

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Pleven, Bulgaria

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Pleven, Bulgaria

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Ep0092 154

Sofia, Bulgaria

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Zagreb, Croatia

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Zagreb, Croatia

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Zagreb, Croatia

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Zagreb, Croatia

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Ep0092 254

Brno, Czechia

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Ostrava, Czechia

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Prague, Czechia

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Prague, Czechia

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Ep0092 016

Aarhus, Denmark

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Ep0092 015

Odense, Denmark

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Ep0092 276

Tallinn, Estonia

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Tallinn, Estonia

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Tartu, Estonia

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Tampere, Finland

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Lyon, France

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Paris, France

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Strasbourg, France

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Berlin, Germany

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Bernau, Germany

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Bielefeld, Germany

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Frankfurt, Germany

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Jena, Germany

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Leipzig, Germany

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Regensburg, Germany

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Ioannina, Greece

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Thessaloniki, Greece

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Thessaloniki, Greece

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Thessaloniki, Greece

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Budapest, Hungary

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Debrecen, Hungary

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Pécs, Hungary

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Cork, Ireland

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Dublin, Ireland

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Milan, Italy

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Asahikawa, Japan

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Asaka, Japan

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Bunkyō City, Japan

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Bunkyō City, Japan

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Hamamatsu, Japan

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Hiroshima, Japan

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Hōfu, Japan

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Itami, Japan

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Izumi, Japan

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Kumamoto, Japan

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Kure, Japan

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Kyoto, Japan

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Nagakute, Japan

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Ōmura, Japan

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Ōsaka-sayama, Japan

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Ep0092 515

Saitama, Japan

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Ep0092 508

Sapporo, Japan

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Ep0092 527

Shinagawa-Ku, Japan

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Ep0092 509

Shizuoka, Japan

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Ep0092 529

Yonago, Japan

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Ep0092 775

Sandvika, Norway

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Ep0092 605

Katowice, Poland

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Ep0092 616

Katowice, Poland

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Ep0092 603

Krakow, Poland

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Ep0092 614

Krakow, Poland

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Lodz, Poland

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Lublin, Poland

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Lublin, Poland

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Nowa Sól, Poland

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Warsaw, Poland

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Wroclaw, Poland

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Zamość, Poland

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Zgierz, Poland

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Aveiro, Portugal

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Matosinhos Municipality, Portugal

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Bucharest, Romania

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Bucharest, Romania

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Târgu Mureş, Romania

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Ep0092 327

Belgrade, Serbia

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Novi Sad, Serbia

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Bardejov, Slovakia

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Alicante, Spain

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Barcelona, Spain

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Barcelona, Spain

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Barcelona, Spain

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Madrid, Spain

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Valencia, Spain

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Zaragoza, Spain

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Ep0092 576

Gothenburg, Sweden

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Ep0092 575

Linköping, Sweden

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Ep0092 053

Zurich, Switzerland

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Ep0092 913

Ankara, Turkey (Türkiye)

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Ep0092 915

Antalya, Turkey (Türkiye)

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Ep0092 900

Istanbul, Turkey (Türkiye)

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Ep0092 906

Istanbul, Turkey (Türkiye)

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Ep0092 909

Istanbul, Turkey (Türkiye)

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Ep0092 908

Trabzon, Turkey (Türkiye)

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Ep0092 766

Brighton, United Kingdom

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Ep0092 750

Manchester, United Kingdom

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Ep0092 764

Swansea, United Kingdom

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Related Publications (1)

  • Rademacher M, Toledo M, Van Paesschen W, Liow KK, Milanov IG, Esch ML, Wang N, MacPherson M, Byrnes WJ, Minh TDC, Webster E, Werhahn KJ. Efficacy and safety of adjunctive padsevonil in adults with drug-resistant focal epilepsy: Results from two double-blind, randomized, placebo-controlled trials. Epilepsia Open. 2022 Dec;7(4):758-770. doi: 10.1002/epi4.12656. Epub 2022 Oct 22.

MeSH Terms

Conditions

Drug Resistant EpilepsySeizuresEpilepsy

Interventions

padsevonil

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System DiseasesNervous System DiseasesNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Results Point of Contact

Title
UCB
Organization
Cares

Study Officials

  • UCB Cares

    001 844 599 2273 (UCB)

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
GT60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 9, 2018

First Posted

November 14, 2018

Study Start

March 6, 2019

Primary Completion

September 28, 2020

Study Completion

September 28, 2020

Last Updated

December 21, 2022

Results First Posted

October 25, 2021

Record last verified: 2022-12

Data Sharing

IPD Sharing
Will share

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 prespecified 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.

Shared Documents
STUDY PROTOCOL, SAP, CSR
Time Frame
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.
Access Criteria
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.
More information

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