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)

NCT03694275 | Completed Phase 2 Results DMC FDA Drug

• 3 other identifiers: TAK-935-18-002 (OV935)U1111-1219-57872022-001315-44
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 8

Brief Summary

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.

Conditions

15q Duplication Syndrome; CDKL5 Deficiency Disease

Keywords

Drug Therapy; 15q Duplication Syndrome (Dup15q); CDKL5 Deficiency Disorder (CDD); Brain Diseases; Epilepsy; Central Nervous System Diseases; Autism; Cholesterol 24S-hydroxylase inhibitor; Anti-epilepsy drug; Anticonvulsants

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Maintenance Period

  Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during maintenance period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.

  Maintenance Period: Weeks 9 to 20

Secondary Outcomes (9)

• Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Treatment Period

  Treatment Period: Weeks 0 to 20

• Percentage of Participants Considered as Treatment Responders During the Maintenance Period

  Maintenance Period: Weeks 9 to 20

• Percent Change From Baseline in Frequency of Motor Seizures Longer Than 5 Minutes in Participants With CDD

  Treatment Period: Weeks 0 to 20

• Proportion of Motor Seizure-free Days in Participants During the Maintenance Period

  Maintenance Period: Weeks 9 to 20

• Change From Baseline in Clinician's Global Impression of Severity (CGI-S) Responses of Investigator

  Baseline to Week 20

Study Arms (2)

Soticlestat Dup 15q

EXPERIMENTAL

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.

Drug: Soticlestat

Soticlestat CDD

EXPERIMENTAL

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.

Drug: Soticlestat

Interventions

Soticlestat DRUG

TAK-935 tablets

Also known as: TAK-935

Soticlestat CDD; Soticlestat Dup 15q

Eligibility Criteria

• Age: 2 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Clinical diagnosis of Dup 15q or CDKL5 deficiency disorder.
• Currently taking 1 to 6 antiepileptic drugs (AEDs) at a stable dose.

You may not qualify if:

• Two or more episodes of convulsive status epilepticus per 3 months requiring hospitalization and intubation.
• Currently receiving a study drug or participated in a clinical study involving another investigational product in the previous month.

Sponsors & Collaborators

• Takeda: lead
• Healx AI: collaborator

Study Sites (8)

UCLA

Los Angeles, California, 90095, United States

Location

Research Institute Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Center for Rare Neurological Diseases

Norcross, Georgia, 30093, United States

Location

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

Boston, Massachusetts, 02114, United States

Location

Boston Children's Hospital Translational Neuroscience Center

Boston, Massachusetts, 02115, United States

Location

Minnesota Epilepsy Group, P.A.

Saint Paul, Minnesota, 55102, United States

Location

New York University (NYU)

New York, New York, 10017, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Related Publications (1)

• 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.

  PMID: 37011526 DERIVED

MeSH Terms

Conditions

Polyposis Syndrome, Hereditary Mixed, 1; CDKL5 deficiency disorder; Brain Diseases; Epilepsy; Central Nervous System Diseases; Autistic Disorder

Interventions

soticlestat

Condition Hierarchy (Ancestors)

Nervous System Diseases; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Medical Director
• Organization: Takeda

trialdisclosures@takeda.com

+1-877-825-3327

Study Officials

• Medical Director Clinical Science

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 27, 2018
• First Posted: October 3, 2018
• Study Start: September 10, 2018
• Primary Completion: July 13, 2020
• Study Completion: July 31, 2020
• Last Updated: May 26, 2022
• Results First Posted: April 20, 2021

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

US (8)