A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Participants With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder

Sponsor:

Collaborators:

Information provided by (Responsible Party):

，

Tracking Information

First Submitted Date ^ICMJE

September 27, 2018

First Posted Date ^ICMJE

October 3, 2018

Last Update Posted Date

October 3, 2018

Actual Study Start Date ^ICMJE

September 10, 2018

Estimated Primary Completion Date

August 14, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Percent Change from Baseline in Motor Seizure Frequency[ Time Frame: up to 12 weeks ]

Original Primary Outcome Measures ^ICMJE

Same as current

Current Secondary Outcome Measures ^ICMJE

Percentage of Participants Considered as Treatment Responders During the Treatment Period[ Time Frame: up to 12 weeks ]
Percent Change from Baseline in Motor Seizure Frequency During the Maintenance Period[ Time Frame: up to 8 weeks ]
Percent Change from Baseline in Clinician's Global Impression of Severity and Change (CGI-S/C) Responses of Investigator[ Time Frame: up to 12 weeks ]
Correlation of TAK-935 Concentration and Plasma 24S-hydroxycholesterol (24HC) Levels[ Time Frame: up to 12 weeks ]
Percent Change from Baseline in Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family[ Time Frame: up to 12 weeks ]

Descriptive Information

Brief Title ^ICMJE

A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Participants With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder

Official Title ^ICMJE

A Multicenter, Open-label, Pilot Study of TAK-935 in Patients With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder

Brief Summary

The purpose of this study is to investigate the effect of TAK-935 (OV935) on the frequency of motor seizures for participants with 15q duplication syndrome or CDKL5 deficiency disorder throughout the Treatment Period (Titration and Maintenance).

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. The overall time to participate in this study is 22 weeks, including 4 to 6 weeks Screening/Baseline Period, 12 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 ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Allocation:
Intervention Model: Single Group Assignment
Intervention Model Description:
Masking: Interventional
Masking Description:
Primary Purpose: Treatment

Condition ^ICMJE

Intervention ^ICMJE

Drug: TAK-935
TAK-935 tablets or mini-tablets.

Study Arms

Experimental: TAK-935 (OV935)
Treatment: 2 weeks titration followed by 10 weeks maintenance period.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date

September 11, 2020

Estimated Primary Completion Date

August 14, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria: 1. Clinical diagnosis of 15q duplication syndrome or CDKL5 deficiency disorder 2. Currently taking 1 to 4 antiepileptic drugs (AEDs) at a stable dose Exclusion Criteria: 1. One or more episodes of convulsive status epilepticus per week requiring hospitalization 2. Currently receiving a study drug or participated in a clinical study involving another investigational product in the previous month

Sex/Gender

Sexes Eligible for Study:

All

Ages

2 Years and older (Adult, Older Adult)

Accepts Healthy Volunteers

Listed Location Countries ^ICMJE

United States

Removed Location Countries

Administrative Information

Has Data Monitoring Committee

Yes

U.S. FDA-regulated Product

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

IPD Sharing Statement

Plan to Share IPD:

Undecided

Responsible Party

，

Study Sponsor ^ICMJE

Takeda

Collaborators ^ICMJE

Investigators ^ICMJE

Study Director: Medical Director Takeda

PRS Account

Verification Date

October 2018

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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