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)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03694275|
Recruitment Status : Active, not recruiting
First Posted : October 3, 2018
Last Update Posted : June 26, 2020
|Condition or disease||Intervention/treatment||Phase|
|15q Duplication Syndrome CDKL5 Deficiency Disease||Drug: TAK-935||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Estimated Primary Completion Date :||July 14, 2020|
|Estimated Study Completion Date :||August 30, 2020|
Experimental: TAK-935 (OV935)
Treatment: 8 weeks dose optimization followed by 12 weeks maintenance period.
TAK-935 tablets or mini-tablets.
- Percent Change from Baseline in Motor Seizure Frequency During the Maintenance Period [ Time Frame: up to 12 weeks ]
- Percentage of Participants Considered as Treatment Responders During the Maintenance Period [ Time Frame: up to 12 weeks ]
- Percent Change from Baseline in Motor Seizure Frequency During the Treatment Period [ Time Frame: up to 20 weeks ]
- Change from Baseline in Clinician's Global Impression of Severity and Change (CGI-S/C) Responses of Investigator [ Time Frame: up to 20 weeks ]
- Correlation of plasma 24S-hydroxycholesterol (24HC) Levels and change in motor seizures [ Time Frame: up to 20 weeks ]
- Change from Baseline in Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family [ Time Frame: up to 20 weeks ]
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|
|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||Takeda|