A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)
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ClinicalTrials.gov Identifier: NCT03694275 |
Recruitment Status :
Completed
First Posted : October 3, 2018
Results First Posted : April 20, 2021
Last Update Posted : May 26, 2022
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Condition or disease | Intervention/treatment | Phase |
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15q Duplication Syndrome CDKL5 Deficiency Disease | Drug: Soticlestat | Phase 2 |
The drug being tested in this study is called soticlestat. Soticlestat is being tested to treat people with Dup 15q or CDD. This study will assess the effects of TAK-935 on seizure frequency, safety.
The study will enroll approximately 30 participants. Participants will be enrolled into 2 groups based on their diagnosis as: Dup 15q or CDD.
All participants will be asked to take soticlestat tablets twice daily with or without food.
The study comprises of 2 periods: Screening/Baseline Period and Treatment Period (Dose Optimization and Maintenance). The overall time to participate in this study is approximately 30 weeks, including 4 to 6 weeks Screening/Baseline Period, 20 weeks Treatment Period, 2 weeks Taper, and 2 weeks safety follow up period. Participants completing this study will have an option to enroll in the open-label extension (OLE) study, under a separate protocol.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 20 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Patients With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder (ARCADE Study) |
Actual Study Start Date : | September 10, 2018 |
Actual Primary Completion Date : | July 13, 2020 |
Actual Study Completion Date : | July 31, 2020 |

Arm | Intervention/treatment |
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Experimental: Soticlestat Dup 15q
Soticlestat tablets twice daily (BID) orally or via gastrostomy tube (G-tube)/ percutaneous endoscopic gastrostomy (PEG) tube, BID. Participants with Dup 15q weighing <60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
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Drug: Soticlestat
TAK-935 tablets
Other Name: TAK-935 |
Experimental: Soticlestat CDD
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing <60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
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Drug: Soticlestat
TAK-935 tablets
Other Name: TAK-935 |
- Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Maintenance Period [ Time Frame: Maintenance Period: Weeks 9 to 20 ]Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during maintenance period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.
- Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Treatment Period [ Time Frame: Treatment Period: Weeks 0 to 20 ]Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during the treatment period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.
- Percentage of Participants Considered as Treatment Responders During the Maintenance Period [ Time Frame: Maintenance Period: Weeks 9 to 20 ]Responders are defined as having over 50% motor seizure reduction compared to Baseline. Percent reduction from Baseline (%) is defined as [(Maintenance Period motor Seizure Frequency - Baseline Period motor Seizure Frequency) divided by Baseline motor Seizure Frequency] multiplied by 100. Data is reported as reduction of 25%, 50%, 75% and 100% or more in motor seizures from Baseline.
- Percent Change From Baseline in Frequency of Motor Seizures Longer Than 5 Minutes in Participants With CDD [ Time Frame: Treatment Period: Weeks 0 to 20 ]Seizure frequency is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period. Percent Change from Baseline is defined as (frequency of seizures during Treatment period - frequency of seizures at Baseline) divided by frequency of seizures at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease. The data is reported only for CDD participants.
- Proportion of Motor Seizure-free Days in Participants During the Maintenance Period [ Time Frame: Maintenance Period: Weeks 9 to 20 ]Seizure-free days is defined as number of days with zero motor seizure during the period the Maintenance Period divided by number of days participant was in the Maintenance Period.
- Change From Baseline in Clinician's Global Impression of Severity (CGI-S) Responses of Investigator [ Time Frame: Baseline to Week 20 ]The CGI-S focuses on clinician's observations of the participant's cognitive, functional, and behavioral performance since the beginning of the study. The CGI-S is rated on a 7-point scale, with the severity of illness scale using a range of responses where, 1= normal, not at all ill, 2= borderline mentally ill, 3= mildly ill, 4= moderately ill, 5= markedly ill, 6= severely ill and 7=amongst the most extremely ill participants. Negative change from Baseline indicates improvement.
- Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression [ Time Frame: Week 20 ]CGI-Change (CGI-C) treatment response ratings should take account of both therapeutic efficacy and treatment-related AEs. Each component of the CGI is rated separately; the instrument does not yield a global score. The CGI-C is rated on a 5-point scale, where, 0 = marked improvement and no side-effects, 1 = marked improvement and minimal side-effects, 2 = no change, 3 = minimal improvement and marked side-effects and 4 = unchanged or worse and side-effects outweigh the therapeutic effect. Lower scores indicated improvement.
- Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family [ Time Frame: Week 20 ]CGI-Change (CGI-C) treatment response ratings should take account of both therapeutic efficacy and treatment-related AEs. Each component of the CGI is rated separately; the instrument does not yield a global score. The CGI-C is rated on a 7-point scale where, 1 = very much improved, 2 = much improved, 3 = slightly improved, 4= no change, 5= slightly worse, 6= much worse and 7= very much worse and marked side-effects. Lower scores indicated improvement.
- Change From Baseline of Plasma 24S-hydroxycholesterol (24HC) Levels [ Time Frame: Baseline to Week 20 ]
- Change From Baseline in Seizure Frequency in Participants Treated With TAK-935 as an Adjunctive Therapy [ Time Frame: Baseline to Week 20 ]Seizure Frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure during the period seizures were assessed multiplied by 28. Positive change from Baseline indicates seizure increase and negative change from Baseline indicates seizure decrease.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 2 Years to 55 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Clinical diagnosis of Dup 15q or CDKL5 deficiency disorder.
- Currently taking 1 to 6 antiepileptic drugs (AEDs) at a stable dose.
Exclusion Criteria:
- Two or more episodes of convulsive status epilepticus per 3 months requiring hospitalization and intubation.
- Currently receiving a study drug or participated in a clinical study involving another investigational product in the previous month.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03694275
United States, California | |
UCLA | |
Los Angeles, California, United States, 90095 | |
United States, Colorado | |
Research Institute Children's Hospital Colorado | |
Aurora, Colorado, United States, 80045 | |
United States, Georgia | |
Center for Rare Neurological Diseases | |
Norcross, Georgia, United States, 30093 | |
United States, Massachusetts | |
Center for Rare Neurological Diseases (CRND)--Massachusetts General Hospital | |
Boston, Massachusetts, United States, 02114 | |
Boston Children's Hospital Translational Neuroscience Center | |
Boston, Massachusetts, United States, 02115 | |
United States, Minnesota | |
Minnesota Epilepsy Group, P.A. | |
Saint Paul, Minnesota, United States, 55102 | |
United States, New York | |
New York University (NYU) | |
New York, New York, United States, 10017 | |
Columbia University Medical Center | |
New York, New York, United States, 10032 |
Study Director: | Medical Director Clinical Science | Takeda |
Documents provided by Takeda:
Responsible Party: | Takeda |
ClinicalTrials.gov Identifier: | NCT03694275 |
Other Study ID Numbers: |
TAK-935-18-002 (OV935) U1111-1219-5787 ( Registry Identifier: WHO ) 2022-001315-44 ( EudraCT Number ) |
First Posted: | October 3, 2018 Key Record Dates |
Results First Posted: | April 20, 2021 |
Last Update Posted: | May 26, 2022 |
Last Verified: | May 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement. |
Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) Informed Consent Form (ICF) |
Access Criteria: | IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement. |
URL: | https://vivli.org/ourmember/takeda/ |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Drug Therapy 15q Duplication Syndrome (Dup15q) CDKL5 Deficiency Disorder (CDD) Brain Diseases Epilepsy |
Central Nervous System Diseases Autism Cholesterol 24S-hydroxylase inhibitor Anti-epilepsy drug Anticonvulsants |
Deficiency Diseases Syndrome Disease |
Pathologic Processes Malnutrition Nutrition Disorders |