Clinical trial NCT03694275: 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)
Official title: A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Patients With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder (ARCADE Study)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: 15q Duplication Syndrome
CDKL5 Deficiency Disease
Keywords: 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
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Ovid Therapeutics Inc.
First posted: 2018-10-03
Enrollment: 20
Sex: ALL
Minimum age (years): 2
Maximum age (years): 55
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

1. Clinical diagnosis of Dup 15q or CDKL5 deficiency disorder.
2. Currently taking 1 to 6 antiepileptic drugs (AEDs) at a stable dose.

Exclusion Criteria:

1. Two or more episodes of convulsive status epilepticus per 3 months requiring hospitalization and intubation.
2. Currently receiving a study drug or participated in a clinical study involving another investigational product in the previous month.

Primary outcomes:
measure: Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Maintenance Period
timeFrame: Maintenance Period: Weeks 9 to 20
Secondary outcomes:
measure: Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Treatment Period
timeFrame: Treatment Period: Weeks 0 to 20
measure: Percentage of Participants Considered as Treatment Responders During the Maintenance Period
timeFrame: Maintenance Period: Weeks 9 to 20
measure: Percent Change From Baseline in Frequency of Motor Seizures Longer Than 5 Minutes in Participants With CDD
timeFrame: Treatment Period: Weeks 0 to 20
measure: Proportion of Motor Seizure-free Days in Participants During the Maintenance Period
timeFrame: Maintenance Period: Weeks 9 to 20
measure: Change From Baseline in Clinician's Global Impression of Severity (CGI-S) Responses of Investigator
timeFrame: Baseline to Week 20
measure: Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression
timeFrame: Week 20
measure: Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family
timeFrame: Week 20
measure: Change From Baseline of Plasma 24S-hydroxycholesterol (24HC) Levels
timeFrame: Baseline to Week 20
measure: Change From Baseline in Seizure Frequency in Participants Treated With TAK-935 as an Adjunctive Therapy
timeFrame: Baseline to Week 20
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA
state: California
city: Aurora
country: United States
facility: Research Institute Children's Hospital Colorado
state: Colorado
city: Norcross
country: United States
facility: Center for Rare Neurological Diseases
state: Georgia
city: Boston
country: United States
facility: Center for Rare Neurological Diseases (CRND)--Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital Translational Neuroscience Center
state: Massachusetts
city: Saint Paul
country: United States
facility: Minnesota Epilepsy Group, P.A.
state: Minnesota
city: New York
country: United States
facility: New York University (NYU)
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
Study officials:
affiliation: Takeda
name: Medical Director Clinical Science
role: STUDY_DIRECTOR
References: