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NCT Number: NCT06356233

Phenotyping and Identification of Biological Markers in STXBP1 Encephalopathy

This is a prospective observational study to evaluate the phenotype of 10 patients under 10 years of age with developmental epileptic encephalopathy due to mutation of the STXBP1 gene. The study will consist of a clinical and neurodevelopmental evaluation, magnetic resonance imaging, prolonged electroencephalogram, cardiological study, and analysis of biomarkers in cerebrospinal fluid. These patients will be followed up for 3 years. The aim of the study is, knowing the baseline phenotype, to analyse the response to commonly used drugs and to anticipate the response to different drugs available on the market in this group of patients based on clinical and biomarker assessment (EEG, MRI and study of specific proteins and neurotransmitters in plasma, urine and CSF).

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Key information

Age range

1 month–10 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital Ruber Internacional

Madrid, 28034, Spain

Location contact

Alvaro Beltran Corbellini, MD

CONTACT

[email protected]

+34913875250

Antonio Gil-Nagel, MD

PRINCIPAL_INVESTIGATOR

Antonio Gil-Nagel, MD, PhD

CONTACT

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients under 10 years of age with confirmed mutation for STXBP1. In cases where the diagnostic technique for the mutation is not optimal, a trio exome will be performed to confirm the mutation.

Exclusion criteria

  • Presence of functional disability that prevents the neuropsychological study from being carried out and absence of a reliable informant for the patient.

Treatment and study plan

No intervention will be performed

Other

No intervention will be performed

Primary outcomes

  1. CSF biomarkers

    Time frame: Baseline, 1 year and 2 years

  2. EEG markers

    Time frame: Baseline, 1 year and 2 years

  3. MRI markers

    Time frame: Baseline, 1 year and 2 years

Secondary outcomes

  1. Clinical phenotype

    Time frame: Baseline, 1 year and 2 years

Sponsors and collaborators

Lead sponsor

Fundación Iniciativa para las Neurociencias (FINCE)

Other

Registry information

Acronym: FIMBEX

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Apr 10, 2024
Registry last updated
Apr 10, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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