A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies

NCT ID: NCT03650452

Last Updated: 2021-02-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 141 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-08-08
• Study Completion Date: 2020-07-20

Brief Summary

The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.

Detailed Description

The drug being tested in this study is called TAK-935 (OV935). This randomized, double-blind study will assess the effects of TAK-935 (OV935), compared to placebo, on efficacy, safety, and tolerability in pediatric participants with Dravet syndrome (DS) or Lennox Gastaut syndrome (LGS). This multi-center trial will be conducted worldwide and will enroll approximately 126 participants.

Participants will be randomized based on their diagnosis in 2 categories; DS or LGS. The study will consist of 2 periods: Screening Period and Treatment Period. The overall duration of Treatment Period is up to 20 weeks including 8-week Dose Optimization Period and 12-week Maintenance Period. The overall time to participants in this study is approximately 30 weeks.

Participants completing this study will have an option to enroll in the open-label extension study, under a separate protocol.

Conditions

Epilepsy; Dravet Syndrome; Lennox-Gastaut Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo

TAK-935 placebo-matching tablets, orally or via gastrostomy tube (G-tube)/percutaneous endoscopic gastrostomy (PEG), twice a day (BID) up to Week 20.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

TAK-935 placebo-matching tablets or mini-tablets.

TAK-935

TAK-935 tablets orally or via G-tube/PEG tube, BID. Participants weighing \<60 kg 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.

Group Type: EXPERIMENTAL

TAK-935

Intervention Type: DRUG

TAK-935 tablets or mini-tablets.

Interventions

TAK-935

TAK-935 tablets or mini-tablets.

Intervention Type: DRUG

Placebo

TAK-935 placebo-matching tablets or mini-tablets.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male and female participants aged greater than or equal to (>=) 2 and less than or equal to (<=) 17 years

2. Clinical diagnosis of DS or LGS

3. Weight of >=10 kilogram (kg) at the Screening visit

4. Currently taking 1 to 4 anti-epileptic drugs (AEDs) at a stable dose

5. Failed to become and remain seizure free with trials of at least 2 AEDs

Exclusion Criteria

1. Has been admitted to a medical facility and intubated for treatment of status epilepticus 2 or more times in the 3 months immediately prior to the screening visit

2. Non-epileptic events that cannot be reliably distinguished from epileptic seizures

3. Participation in a clinical study involving another study drug in the previous month

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Healx AI

INDUSTRY

Sponsor Role: collaborator

Takeda

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director Clinical Science

Role: STUDY_DIRECTOR

Takeda

Locations

Phoenix Children's Hospital

Phoenix, Arizona, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

Colorado Children's Hospital

Aurora, Colorado, United States

Nicklaus Children's Hospital

Miami, Florida, United States

Pediatric Neurology PA

Orlando, Florida, United States

Rare Disease Research, LLC

Atlanta, Georgia, United States

Center for Rare Neurological Diseases

Norcross, Georgia, United States

Ann and Robert H Lurie Childrens Hospital of Chicago

Chicago, Illinois, United States

Mayo Clinic - PPDS

Rochester, Minnesota, United States

Northeast Regional Epilepsy Group

Hackensack, New Jersey, United States

Children's Hospital at Saint Peter's University Hospital

New Brunswick, New Jersey, United States

Columbia University Medical Center

New York, New York, United States

Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

Monash Children's Hospital

Clayton, Victoria, Australia

Austin Hospital

Heidelberg West, Victoria, Australia

Hospital For Sick Children

Toronto, Ontario, Canada

Peking University First Hospital

Beijing, , China

Capital Medical University (CMU) - Beijing Children's Hospital

Beijing, , China

Beijing Children's Hospital,Capital Medical University

Beijing, , China

Xiangya Hospital Central South University

Changsha, , China

Children's Hospital of Fudan University

Shanghai, , China

Shenzhen Children's Hospital

Shenzhen, , China

Sheba Medical Center-PPDS

Tel Litwinsky, Ramat Gan, Israel

Soroka University Medical Centre

Bear Sheva, , Israel

Bnai Zion Medical Center

Haifa, , Israel

Edith Wolfson Medical Center

Holon, , Israel

Hadassah Medical Center

Jerusalem, , Israel

Schneider Childrens Medical Center of Israel

Petah Tikva, , Israel

Tel Aviv Sourasky Medical Center

Tel Aviv, , Israel

Szpital Kliniczny im. H.Swiecickiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu

Poznan, Greater Poland Voivodeship, Poland

Uniwersyteckie Centrum Kliniczne - PPDS

Gdansk, Pomeranian Voivodeship, Poland

Centrum Medyczne Plejady

Krakow, , Poland

Samodzielny Publiczny Dzieciecy Szpital Kliniczny w Warszawie

Warsaw, , Poland

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, , Poland

NZOZ Centrum Neurologii Dzieciecej i Leczenia Padaczki

Kielce, Świętokrzyskie Voivodeship, Poland

Centro Hospitalar Lisboa Central- Hospital Dona Estefania

Lisbon, , Portugal

Centro Hospitalar Lisboa Norte, E.P.E. Hospital de Santa Maria

Lisbon, , Portugal

Largo da Maternidade de Julio DinisCentro Materno Infantil do Norte

Porto, , Portugal

Clinica Universidad Navarra

Pamplona, Navarre, Spain

Hospital Vithas La Salud

Granada, , Spain

Hospital Ruber Internacional

Madrid, , Spain

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, , Spain

Countries

United States; Australia; Canada; China; Israel; Poland; Portugal; Spain

References

Hahn CD, Jiang Y, Villanueva V, Zolnowska M, Arkilo D, Hsiao S, Asgharnejad M, Dlugos D. A phase 2, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of soticlestat as adjunctive therapy in pediatric patients with Dravet syndrome or Lennox-Gastaut syndrome (ELEKTRA). Epilepsia. 2022 Oct;63(10):2671-2683. doi: 10.1111/epi.17367. Epub 2022 Aug 4.

Reference Type: DERIVED

PMID: 35841234 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

U1111-1206-5522

Identifier Type: OTHER

Identifier Source: secondary_id

2018-002484-25

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TAK-935-2002

Identifier Type: -

Identifier Source: org_study_id