A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Participants With 15Q Duplication Syndrome or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)

• ClinicalTrials.gov Identifier: NCT03694275
• Recruitment Status: Completed
• First Posted: October 3, 2018
• Last Update Posted: August 14, 2020
• Sponsor: Takeda
• Collaborator: Ovid Therapeutics Inc.
• Information provided by (Responsible Party): Takeda

Study Description

Brief Summary:

The purpose of this study is to investigate the effect of TAK-935 on the frequency of motor seizures for participants with 15q duplication syndrome or CDKL5 deficiency disorder during the Maintenance Period.

Condition or disease	Intervention/treatment	Phase
15q Duplication Syndrome; CDKL5 Deficiency Disease	Drug: TAK-935	Phase 2

Detailed Description:

The drug being tested in this study is called TAK-935 (OV935). TAK-935 is being tested to treat people with 15q duplication syndrome or CDKL5 deficiency disorder. 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: 15q duplication syndrome or CDKL5 deficiency disorder.

All participants will be asked to take TAK-935 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 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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 20 participants
• Allocation: N/A
• 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

Arms and Interventions

Arm	Intervention/treatment
Experimental: TAK-935 (OV935); Treatment: 8 weeks dose optimization followed by 12 weeks maintenance period.	Drug: TAK-935; TAK-935 tablets or mini-tablets.

Outcome Measures

Primary Outcome Measures :

1. Percent Change from Baseline in Motor Seizure Frequency During the Maintenance Period [ Time Frame: up to 12 weeks ]

Secondary Outcome Measures :

1. Percentage of Participants Considered as Treatment Responders During the Maintenance Period [ Time Frame: up to 12 weeks ]
2. Percent Change from Baseline in Motor Seizure Frequency During the Treatment Period [ Time Frame: up to 20 weeks ]
3. Change from Baseline in Clinician's Global Impression of Severity and Change (CGI-S/C) Responses of Investigator [ Time Frame: up to 20 weeks ]
4. Correlation of plasma 24S-hydroxycholesterol (24HC) Levels and change in motor seizures [ Time Frame: up to 20 weeks ]
5. Change from Baseline in Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family [ Time Frame: up to 20 weeks ]

Eligibility Criteria

• Ages Eligible for Study: 2 Years to 55 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. Clinical diagnosis of 15q duplication syndrome 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

Contacts and Locations

Locations

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

Sponsors and Collaborators

Takeda

Ovid Therapeutics Inc.

Investigators

• Study Director: Medical Director; Takeda

More Information

• Responsible Party: Takeda
• ClinicalTrials.gov Identifier: NCT03694275
• Other Study ID Numbers: TAK-935-18-002 (OV935); U1111-1219-5787 ( Registry Identifier: WHO )
• First Posted: October 3, 2018
• Last Update Posted: August 14, 2020
• Last Verified: August 2020

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Takeda makes patient-level, de-identified data sets and associated documents available for all interventional studies after applicable marketing approvals and commercial availability have been received (or program is completely terminated), an opportunity for the primary publication of the research and final report development has been allowed, and other criteria have been met as set forth in Takeda's Data Sharing Policy (see www.TakedaClinicalTrials.com for details). To obtain access, researchers must submit a legitimate academic research proposal for adjudication by an independent review panel, who will review the scientific merit of the research and the requestor's qualifications and conflict of interest that can result in potential bias. Once approved, qualified researchers who sign a data sharing agreement are provided access to these data in a secure research environment.

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Takeda:

Drug Therapy; 15q Duplication Syndrome; CDKL5 Deficiency Disease; Brain Diseases; Epilepsy; Central Nervous System Diseases; Autism; Cholesterol 24S-hydroxylase inhibitor; Anti-epilepsy drug; Anticonvulsants; Cyclin-dependent kinase-like 5 deficiency disorder; Chromosome 15q

Additional relevant MeSH terms:

Deficiency Diseases; Syndrome; Disease; Pathologic Processes; Malnutrition; Nutrition Disorders