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)

NCT ID: NCT03694275

Last Updated: 2022-05-26

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-09-10

Study Completion Date

2020-07-31

Brief Summary

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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

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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.

Conditions

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15q Duplication Syndrome CDKL5 Deficiency Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Soticlestat Dup 15q

Soticlestat tablets twice daily (BID) orally or via gastrostomy tube (G-tube)/ percutaneous endoscopic gastrostomy (PEG) tube, BID. Participants with Dup 15q weighing \<60 kg at Baseline 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

Soticlestat

Intervention Type DRUG

TAK-935 tablets

Soticlestat CDD

Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing \<60 kg at Baseline 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

Soticlestat

Intervention Type DRUG

TAK-935 tablets

Interventions

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Soticlestat

TAK-935 tablets

Intervention Type DRUG

Other Intervention Names

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TAK-935

Eligibility Criteria

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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.
Minimum Eligible Age

2 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Healx AI

INDUSTRY

Sponsor Role collaborator

Takeda

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director Clinical Science

Role: STUDY_DIRECTOR

Takeda

Locations

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UCLA

Los Angeles, California, United States

Site Status

Research Institute Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Center for Rare Neurological Diseases

Norcross, Georgia, United States

Site Status

Center for Rare Neurological Diseases (CRND)--Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Boston Children's Hospital Translational Neuroscience Center

Boston, Massachusetts, United States

Site Status

Minnesota Epilepsy Group, P.A.

Saint Paul, Minnesota, United States

Site Status

New York University (NYU)

New York, New York, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

Countries

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United States

References

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Demarest S, Jeste S, Agarwal N, Arkilo D, Asgharnejad M, Hsiao S, Thibert R. Efficacy, safety, and tolerability of soticlestat as adjunctive therapy for the treatment of seizures in patients with Dup15q syndrome or CDKL5 deficiency disorder in an open-label signal-finding phase II study (ARCADE). Epilepsy Behav. 2023 May;142:109173. doi: 10.1016/j.yebeh.2023.109173. Epub 2023 Apr 1.

Reference Type DERIVED
PMID: 37011526 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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U1111-1219-5787

Identifier Type: REGISTRY

Identifier Source: secondary_id

2022-001315-44

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TAK-935-18-002 (OV935)

Identifier Type: -

Identifier Source: org_study_id

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